Identification

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Name
Sirolimus
Accession Number
DB00877  (APRD00178, DB02439)
Type
Small Molecule
Groups
Approved, Investigational
Description

A macrolide compound obtained from Streptomyces hygroscopicus that acts by selectively blocking the transcriptional activation of cytokines thereby inhibiting cytokine production. It is bioactive only when bound to immunophilins. Sirolimus is a potent immunosuppressant and possesses both antifungal and antineoplastic properties.

Structure
Thumb
Synonyms
  • (-)-Rapamycin
  • Rapamycin
  • Sirolimús
  • Sirolimus
  • Sirolimusum
External IDs
AY-22989 / WY-090217
Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing End
RapamuneTablet, sugar coated2 mg/1OralWyeth Pharmaceuticals Inc.2006-06-072006-09-21Us
RapamuneTablet, coated2 mgOralPfizer2001-03-13Not applicableEu
RapamuneTablet, sugar coated2 mg/1OralWyeth Pharmaceuticals Llc, a Subsidiary of Pfizer Inc.2001-07-01Not applicableUs
RapamuneTablet, sugar coated1 mg/1OralWyeth Pharmaceuticals Inc.2006-06-072006-09-21Us
RapamuneTablet, coated2 mgOralPfizer2001-03-13Not applicableEu
RapamuneTablet5 mgOralPfizer Canada UlcNot applicableNot applicableCanada
RapamuneTablet, sugar coated1 mg/1OralWyeth Pharmaceuticals Llc, a Subsidiary of Pfizer Inc.2001-07-01Not applicableUs
RapamuneTablet, sugar coated1 mg/1OralCardinal Health2001-07-012011-08-31Us
RapamuneTablet, coated1 mgOralPfizer2001-03-13Not applicableEu
RapamuneTablet2 mgOralPfizer Canada UlcNot applicableNot applicableCanada
Additional Data Available
  • Application Number
    Application Number

    A unique ID assigned by the FDA when a product is submitted for approval by the labeller.

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  • Product Code
    Product Code

    A governmentally-recognized ID which uniquely identifies the product within its regulatory market.

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Generic Prescription Products
NameDosageStrengthRouteLabellerMarketing StartMarketing End
Gd-sirolimusSolutionOralGenmed A Division Of Pfizer Canada UlcNot applicableNot applicableCanada
Gd-sirolimusTabletOralGenmed A Division Of Pfizer Canada UlcNot applicableNot applicableCanada
Gd-sirolimusTabletOralGenmed A Division Of Pfizer Canada UlcNot applicableNot applicableCanada
Gd-sirolimusTabletOralGenmed A Division Of Pfizer Canada UlcNot applicableNot applicableCanada
SirolimusTablet, film coated0.5 mg/1OralZydus Pharmaceuticals (USA) Inc.2014-01-15Not applicableUs
SirolimusTablet1 mg/1OralAvPAK2018-04-23Not applicableUs
SirolimusTablet1 mg/1OralAmerican Health Packaging2015-11-05Not applicableUs
SirolimusTablet, film coated0.5 mg/1OralCadila Healthcare Limited2014-01-15Not applicableUs
SirolimusTablet, film coated2 mg/1OralCadila Healthcare Limited2014-01-072014-01-03Us
SirolimusTablet, sugar coated2 mg/1OralGreenstone LLC2014-10-27Not applicableUs
Additional Data Available
  • Application Number
    Application Number

    A unique ID assigned by the FDA when a product is submitted for approval by the labeller.

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  • Product Code
    Product Code

    A governmentally-recognized ID which uniquely identifies the product within its regulatory market.

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Categories
UNII
W36ZG6FT64
CAS number
53123-88-9
Weight
Average: 914.187
Monoisotopic: 913.555141608
Chemical Formula
C51H79NO13
InChI Key
QFJCIRLUMZQUOT-HPLJOQBZSA-N
InChI
InChI=1S/C51H79NO13/c1-30-16-12-11-13-17-31(2)42(61-8)28-38-21-19-36(7)51(60,65-38)48(57)49(58)52-23-15-14-18-39(52)50(59)64-43(33(4)26-37-20-22-40(53)44(27-37)62-9)29-41(54)32(3)25-35(6)46(56)47(63-10)45(55)34(5)24-30/h11-13,16-17,25,30,32-34,36-40,42-44,46-47,53,56,60H,14-15,18-24,26-29H2,1-10H3/b13-11+,16-12+,31-17+,35-25+/t30-,32-,33-,34-,36-,37+,38+,39+,40-,42+,43+,44-,46-,47+,51-/m1/s1
IUPAC Name
(1R,9S,12S,15R,16E,18R,19R,21R,23S,24E,26E,28E,30S,32S,35R)-1,18-dihydroxy-12-[(2R)-1-[(1S,3R,4R)-4-hydroxy-3-methoxycyclohexyl]propan-2-yl]-19,30-dimethoxy-15,17,21,23,29,35-hexamethyl-11,36-dioxa-4-azatricyclo[30.3.1.0^{4,9}]hexatriaconta-16,24,26,28-tetraene-2,3,10,14,20-pentone
SMILES
[H][C@@]1(C[C@@H](C)[C@]2([H])CC(=O)[C@H](C)\C=C(C)\[C@@H](O)[C@@H](OC)C(=O)[C@H](C)C[C@H](C)\C=C\C=C\C=C(C)\[C@H](C[C@]3([H])CC[C@@H](C)[C@@](O)(O3)C(=O)C(=O)N3CCCC[C@@]3([H])C(=O)O2)OC)CC[C@@H](O)[C@@H](C1)OC

Pharmacology

Indication

For the prophylaxis of organ rejection in patients receiving renal transplants.

Associated Conditions
Pharmacodynamics

Sirolimus, a macrocyclic lactone produced by Streptomyces hygroscopicus, is an immunosuppressive agent indicated for the prophylaxis of organ rejection in patients receiving renal transplants. It is recommended that sirolimus be used in a regimen with cyclosporine and corticosteroids.

Mechanism of action

Sirolimus inhibits T lymphocyte activation and proliferation that occurs in response to antigenic and cytokine (Interleukin IL-2, IL-4, and IL-15) stimulation by a mechanism that is distinct from that of other immunosuppressants. Sirolimus also inhibits antibody production. In cells, sirolimus binds to the immunophilin, FK Binding Protein-12 (FKBP-12), to generate an immunosuppressive complex. The sirolimus:FKBP-12 complex has no effect on calcineurin activity. This complex binds to and inhibits the activation of the mammalian Target Of Rapamycin (mTOR), a key regulatory kinase. This inhibition suppresses cytokine-driven T-cell proliferation, inhibiting the progression from the G1 to the S phase of the cell cycle.

TargetActionsOrganism
ASerine/threonine-protein kinase mTOR
inhibitor
Humans
APeptidyl-prolyl cis-trans isomerase FKBP1A
other
Humans
AFibroblast growth factor 2
other/unknown
Humans
Additional Data Available
Adverse Effects

Comprehensive structured data on known drug adverse effects with statistical prevalence. MedDRA and ICD10 ids are provided for adverse effect conditions and symptoms.

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Additional Data Available
Contraindications

Structured data covering drug contraindications. Each contraindication describes a scenario in which the drug is not to be used. Includes restrictions on co-administration, contraindicated populations, and more.

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Additional Data Available
Blackbox Warnings

Structured data representing warnings from the black box section of drug labels. These warnings cover important and dangerous risks, contraindications, or adverse effects.

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Absorption
Not Available
Volume of distribution
Not Available
Protein binding

92%

Metabolism
Route of elimination
Not Available
Half life

57-63 hours

Clearance
Not Available
Toxicity
Not Available
Affected organisms
  • Humans and other mammals
Pathways
Not Available
Pharmacogenomic Effects/ADRs
Not Available

Interactions

Drug Interactions
This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.
DrugInteraction
(R)-warfarinThe therapeutic efficacy of (R)-warfarin can be increased when used in combination with Sirolimus.
(S)-WarfarinThe therapeutic efficacy of (S)-Warfarin can be increased when used in combination with Sirolimus.
2-MethoxyethanolThe risk or severity of adverse effects can be increased when Sirolimus is combined with 2-Methoxyethanol.
2,4-thiazolidinedioneThe therapeutic efficacy of 2,4-thiazolidinedione can be decreased when used in combination with Sirolimus.
3-isobutyl-1-methyl-7H-xanthineThe metabolism of 3-isobutyl-1-methyl-7H-xanthine can be decreased when combined with Sirolimus.
4-hydroxycoumarinThe therapeutic efficacy of 4-hydroxycoumarin can be increased when used in combination with Sirolimus.
6-Deoxyerythronolide BThe metabolism of Sirolimus can be decreased when combined with 6-Deoxyerythronolide B.
6-O-benzylguanineThe metabolism of 6-O-benzylguanine can be decreased when combined with Sirolimus.
7-DeazaguanineThe metabolism of 7-Deazaguanine can be decreased when combined with Sirolimus.
7-ethyl-10-hydroxycamptothecinThe metabolism of Sirolimus can be decreased when combined with 7-ethyl-10-hydroxycamptothecin.
Additional Data Available
  • Extended Description
    Extended Description

    Extended description of the mechanism of action and particular properties of each drug interaction.

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  • Severity
    Severity

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  • Evidence Level
    Evidence Level

    A rating for the strength of the evidence supporting each drug interaction.

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  • Action
    Action

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Food Interactions
Not Available

References

Synthesis Reference

Madhup K. Dhaon, Chi-nung Hsiao, Subhash R. Patel, Peter J. Bonk, Sanjay R. Chemburkar, Yong Y. Chen, "One pot synthesis of tetrazole derivatives of sirolimus." U.S. Patent US20080167335, issued July 10, 2008.

US20080167335
General References
  1. Pritchard DI: Sourcing a chemical succession for cyclosporin from parasites and human pathogens. Drug Discov Today. 2005 May 15;10(10):688-91. [PubMed:15896681]
  2. Shuchman M: Trading restenosis for thrombosis? New questions about drug-eluting stents. N Engl J Med. 2006 Nov 9;355(19):1949-52. [PubMed:17093244]
  3. Sun SY, Rosenberg LM, Wang X, Zhou Z, Yue P, Fu H, Khuri FR: Activation of Akt and eIF4E survival pathways by rapamycin-mediated mammalian target of rapamycin inhibition. Cancer Res. 2005 Aug 15;65(16):7052-8. [PubMed:16103051]
  4. Chan S: Targeting the mammalian target of rapamycin (mTOR): a new approach to treating cancer. Br J Cancer. 2004 Oct 18;91(8):1420-4. [PubMed:15365568]
  5. Graziani EI: Recent advances in the chemistry, biosynthesis and pharmacology of rapamycin analogs. Nat Prod Rep. 2009 May;26(5):602-9. doi: 10.1039/b804602f. Epub 2009 Mar 5. [PubMed:19387497]
External Links
Human Metabolome Database
HMDB15015
KEGG Drug
D00753
KEGG Compound
C07909
PubChem Compound
5284616
PubChem Substance
46505675
ChemSpider
10482078
BindingDB
36609
ChEBI
9168
ChEMBL
CHEMBL413
Therapeutic Targets Database
DNC001197
PharmGKB
PA451365
HET
RAP
RxList
RxList Drug Page
Drugs.com
Drugs.com Drug Page
Wikipedia
Sirolimus
ATC Codes
S01XA23 — SirolimusL04AA10 — Sirolimus
AHFS Codes
  • 92:44.00 — Immunosuppressive Agents
PDB Entries
1c9h / 1fap / 1fkb / 1fkl / 1pbk / 1z58 / 2dg3 / 2dg4 / 2dg9 / 2vcd
show 9 more
FDA label
Download (480 KB)

Clinical Trials

Clinical Trials
PhaseStatusPurposeConditionsCount
0Active Not RecruitingTreatmentAdult Acute Megakaryoblastic Leukemia (M7) / Adult Acute Monoblastic Leukemia (M5a) / Adult Acute Monocytic Leukemia (M5b) / Adult Acute Myeloblastic Leukemia With Maturation (M2) / Adult Acute Myeloblastic Leukemia Without Maturation (M1) / Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities / Adult Acute Myeloid Leukemia With Del(5q) / Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) / Adult Acute Myeloid Leukemia With T(16;16)(p13;q22) / Adult Acute Myeloid Leukemia With T(8;21)(q22;q22) / Adult Acute Myelomonocytic Leukemia (M4) / Adult Erythroleukemia (M6a) / Adult Pure Erythroid Leukemia (M6b) / Untreated Adult Acute Myeloid Leukemia1
0Active Not RecruitingTreatmentBladder Cancers1
0CompletedTreatmentAcute Lymphoblastic Leukaemias (ALL) / Adult T-Cell Leukemia/Lymphoma / Burkitt's Lymphoma / Lymphoid Malignancies (New or Relapsed) / Lymphoma, Lymphoblastic / Mantle Cell Lymphoma (MCL)1
0CompletedTreatmentAdult Solid Neoplasm / Advanced Malignant Neoplasm / Cervical Squamous Cell Carcinoma / Endometrial Carcinoma / Malignant Uterine Neoplasm / Recurrent Bladder Carcinoma / Recurrent Breast Carcinoma / Recurrent Cervical Carcinoma / Recurrent Head and Neck Carcinoma / Recurrent Malignant Neoplasm / Recurrent Ovarian Carcinoma / Recurrent Prostate Carcinoma / Recurrent Uterine Corpus Carcinoma / Renal Cell Carcinoma Recurrent / Sarcomas / Solid Neoplasms / Stage III Bladder Cancer / Stage III Prostate Cancer / Stage III Renal Cell Cancer / Stage IIIA Breast Cancer / Stage IIIA Cervical Cancer / Stage IIIA Ovarian Cancer / Stage IIIA Uterine Corpus Cancer / Stage IIIB Breast Cancer / Stage IIIB Cervical Cancer / Stage IIIB Ovarian Cancer / Stage IIIB Uterine Corpus Cancer / Stage IIIC Breast Cancer / Stage IIIC Ovarian Cancer / Stage IIIC Uterine Corpus Cancer / Stage IIIC1 Uterine Corpus Cancer / Stage IIIC2 Uterine Corpus Cancer / Stage IV Breast Cancer / Stage IV Ovarian Cancer / Stage IV Prostate Cancer / Stage IV Renal Cell Cancer / Stage IVA Bladder Cancer / Stage IVA Cervical Cancer / Stage IVA Uterine Corpus Cancer / Stage IVB Bladder Cancer / Stage IVB Cervical Cancer / Stage IVB Uterine Corpus Cancer1
0CompletedTreatmentInherited Immune Deficiencies1
0CompletedTreatmentInvasive Bladder Cancer Stage II1
0CompletedTreatmentPlasma Cell Myeloma1
0CompletedTreatmentTransplantation, Stem Cell1
0CompletedTreatmentTumors, Solid1
0Not Yet RecruitingTreatmentDry Eye1
0Not Yet RecruitingTreatmentKeloid Scars1
0RecruitingTreatmentChronic Granulomatous Disease (CGD)1
0RecruitingTreatmentSickle Cell Disorders1
0TerminatedTreatmentAcute Kidney Injury (AKI) / Renal Transplant Recipient Patients1
0TerminatedTreatmentPemphigus1
1Active Not RecruitingOtherBioavailability Study1
1Active Not RecruitingPreventionAcute Leukemia in Remission / Chronic Myeloid Leukemia (CML) / Chronic Myelomonocytic Leukemia / Lymphoma, Hodgkins / Multiple Myeloma (MM) / Myelodysplastic Syndromes / Non-Hodgkin's Lymphoma (NHL) / Primary Myelofibrosis1
1Active Not RecruitingTreatmentAdult Solid Neoplasm1
1Active Not RecruitingTreatmentAdvanced Cancers1
1Active Not RecruitingTreatmentAstrocytomas / Atypical Teratoid/Rhabdoid Tumor / Clear Cell Sarcoma of Soft Tissue / Ependymomas / Ewing's Sarcoma (ES) / Germ Cell Tumors / Gliomas / Hepatoblastomas / Medulloblastomas / Neuroblastomas / Renal Cell Adenocarcinoma / Retinoblastoma / Rhabdoid Tumors / Rhabdomyosarcomas / Sarcoma, Osteogenic / Wilms' tumor1
1Active Not RecruitingTreatmentAutologous Hematopoietic Stem Cell Transplant Recipient / Leukemia, Plasma Cell / Loss of Chromosome 17p / Recurrent Plasma Cell Myeloma / Refractory Multiple Myeloma / Refractory Plasma Cell Myeloma / Stage I Multiple Myeloma / Stage II Multiple Myeloma1
1Active Not RecruitingTreatmentCancer, Advanced1
1Active Not RecruitingTreatmentClear Cell Carcinoma / Ewing's Sarcoma (ES) / Gastrointestinal Tumors / Germ Cell Tumors / Hepatic Tumors / Malignant Lymphomas / Melanoma / Neuroblastomas / Non-rhabdomyosarcoma / Renal Cell Adenocarcinoma / Rhabdoid Tumors / Rhabdomyosarcomas / Wilms' tumor1
1Active Not RecruitingTreatmentRecurrent Fallopian Tube Cancer / Recurrent Ovarian Epithelial Cancer / Recurrent Primary Peritoneal Cavity Cancer / Stage IIA Fallopian Tube Cancer / Stage IIA Ovarian Epithelial Cancer / Stage IIA Primary Peritoneal Cavity Cancer / Stage IIB Fallopian Tube Cancer / Stage IIB Ovarian Epithelial Cancer / Stage IIB Primary Peritoneal Cavity Cancer / Stage IIC Fallopian Tube Cancer / Stage IIC Ovarian Epithelial Cancer / Stage IIC Primary Peritoneal Cavity Cancer / Stage IIIA Fallopian Tube Cancer / Stage IIIA Ovarian Epithelial Cancer / Stage IIIA Primary Peritoneal Cavity Cancer / Stage IIIB Fallopian Tube Cancer / Stage IIIB Ovarian Epithelial Cancer / Stage IIIB Primary Peritoneal Cavity Cancer / Stage IIIC Fallopian Tube Cancer / Stage IIIC Ovarian Epithelial Cancer / Stage IIIC Primary Peritoneal Cavity Cancer / Stage IV Fallopian Tube Cancer / Stage IV Ovarian Epithelial Cancer / Stage IV Primary Peritoneal Cavity Cancer1
1CompletedNot AvailableAcute Lymphoblastic Leukaemias (ALL)1
1CompletedNot AvailableAdvanced Solid Tumors / Refractory to Standard Therapies1
1CompletedNot AvailableHealthy Volunteers / Pharmacokinetics of Isavuconazole / Pharmacokinetics of Sirolimus1
1CompletedBasic ScienceAging1
1CompletedBasic ScienceDrug Drug Interaction (DDI) / High Cholesterol / Immunosuppression / Pharmacokinetics1
1CompletedBasic ScienceSarcopenia1
1CompletedOtherBioequivalence Study1
1CompletedPreventionChronic Renal Failure (CRF) / Immunosuppression / Transplantation, Kidney1
1CompletedPreventionNeoplastic Syndrome / Non-Melanomatous Skin Cancer1
1CompletedTreatmentAcinar Cell Adenocarcinoma of the Pancreas / Duct Cell Adenocarcinoma of the Pancreas / Recurrent Pancreatic Cancer / Stage IV Pancreatic Cancer / Unspecified Adult Solid Tumor, Protocol Specific1
1CompletedTreatmentAdult Solid Neoplasm1
1CompletedTreatmentAdult Solid Neoplasm / Lung Carcinoid Tumor / Recurrent Digestive System Neuroendocrine Tumor G1 / Recurrent Uterine Corpus Sarcoma / Renal Cell Carcinoma Recurrent / Stage III Renal Cell Cancer / Stage IIIB Uterine Sarcoma / Stage IIIC Uterine Sarcoma / Stage IV Renal Cell Cancer / Stage IVA Uterine Sarcoma / Stage IVB Uterine Sarcoma1
1CompletedTreatmentAdvanced Cancers1
1CompletedTreatmentAdvanced Solid Tumors1
1CompletedTreatmentAging / Coronary Artery Disease1
1CompletedTreatmentAllogeneic Stem Cell Transplantation / Malignancies, Hematologic1
1CompletedTreatmentAnaplastic Astrocytoma (AA) / Anaplastic Oligoastrocytoma / Anaplastic Oligodendroglioma (AO) / Estrogen Receptor Negative / Estrogen Receptor Positive / Glioblastomas / Hormone-Resistant Prostate Cancer / Metastatic Hormone Refractory Prostate Cancer / Metastatic Renal Cell Cancer / Recurrent Adult Brain Neoplasm / Recurrent Bladder Carcinoma / Recurrent Breast Carcinoma / Recurrent Colorectal Carcinoma / Recurrent Esophageal Carcinoma / Recurrent Gastric Carcinoma / Recurrent Hepatocellular Carcinoma / Recurrent Lung Carcinoma / Recurrent Melanoma / Recurrent Ovarian Carcinoma / Recurrent Prostate Carcinoma / Recurrent Uterine Corpus Carcinoma / Renal Cell Carcinoma Recurrent / Resectable Hepatocellular Carcinoma / Sarcomas / Stage IA Breast Cancer / Stage IA Ovarian Cancer / Stage IA Uterine Corpus Cancer / Stage IB Breast Cancer / Stage IB Ovarian Cancer / Stage IB Uterine Corpus Cancer / Stage IC Ovarian Cancer / Stage II Uterine Corpus Cancer / Stage IIA Breast Cancer / Stage IIA Lung Carcinoma / Stage IIA Ovarian Cancer / Stage IIB Breast Cancer / Stage IIB Esophageal Cancer / Stage IIB Lung Carcinoma / Stage IIB Ovarian Cancer / Stage IIB Skin Melanoma / Stage IIC Ovarian Cancer / Stage IIC Skin Melanoma / Stage IIIA Breast Cancer / Stage IIIA Esophageal Cancer / Stage IIIA Lung Carcinoma / Stage IIIA Ovarian Cancer / Stage IIIA Skin Melanoma / Stage IIIA Uterine Corpus Cancer / Stage IIIB Breast Cancer / Stage IIIB Esophageal Cancer / Stage IIIB Ovarian Cancer / Stage IIIB Skin Melanoma / Stage IIIB Uterine Corpus Cancer / Stage IIIC Breast Cancer / Stage IIIC Esophageal Cancer / Stage IIIC Ovarian Cancer / Stage IIIC Skin Melanoma / Stage IIIC Uterine Corpus Cancer / Stage IV Bladder Urothelial Carcinoma / Stage IV Esophageal Cancer / Stage IV Ovarian Cancer / Stage IV Prostate Cancer / Stage IV Skin Melanoma / Stage IVA Uterine Corpus Cancer / Stage IVB Uterine Corpus Cancer1
1CompletedTreatmentAngiofibromas / Neurofibromas / Neurofibromatoses / Tuberous Sclerosis1
1CompletedTreatmentAnterior Uveitis (AU)1
1CompletedTreatmentBrain and Central Nervous System Tumors1
1CompletedTreatmentCML / Leukemia Acute Myeloid Leukemia (AML) / Leukemias / Myeloid Leukemias1
1CompletedTreatmentCancer of Kidney / Cancer of Liver / Malignant Lymphomas / Neoplasms, Breast / Neoplasms, Lung1
1CompletedTreatmentCancer, Advanced4
1CompletedTreatmentCancer, Advanced / Metastatic Cancers1
1CompletedTreatmentChildhood B Acute Lymphoblastic Leukemia / Childhood T Acute Lymphoblastic Leukemia / Mature T-Cell and NK-Cell Non-Hodgkin Lymphoma / Recurrent Childhood Acute Lymphoblastic Leukemia / Recurrent Childhood Lymphoblastic Lymphoma1
1CompletedTreatmentChildhood Solid Tumors / Malignant Solid Tumours1
1CompletedTreatmentChorioretinitis / Panuveitis / Uveitis / Uveitis, Intermediate1
1CompletedTreatmentDiabetic Macular Edema (DME) / Retinopathy, Diabetic1
1CompletedTreatmentDrug/Agent Toxicity by Tissue/Organ / Multiple Myeloma and Plasma Cell Neoplasm1
1CompletedTreatmentExtensive Stage Small Cell Lung Cancer / Hereditary Paraganglioma / Male Breast Cancer / Metastatic Gastrointestinal Carcinoid Tumor / Metastatic Pheochromocytoma / Non-Small Cell Lung Cancer Recurrent / Pancreatic Polypeptide Tumor / Paraganglion neoplasm malignant / Recurrent Breast Cancer / Recurrent Cervical Cancer / Recurrent Endometrial Carcinoma / Recurrent Gastrointestinal Carcinoid Tumor / Recurrent Islet Cell Carcinoma / Recurrent Neuroendocrine Carcinoma of the Skin / Recurrent Ovarian Epithelial Cancer / Recurrent Ovarian Germ Cell Tumor / Recurrent Pheochromocytoma / Recurrent Prostate Cancer / Recurrent Uterine Sarcoma / Refractory Small cell lung cancer / Regional Gastrointestinal Carcinoid Tumor / Regional Pheochromocytoma / Renal Cell Cancer, Recurrent / Stage III Cervical Cancer / Stage III Endometrial Carcinoma / Stage III Neuroendocrine Carcinoma of the Skin / Stage III Ovarian Epithelial Cancer / Stage III Ovarian Germ Cell Tumor / Stage III Prostate Cancer / Stage III Renal Cell Cancer / Stage III Uterine Sarcoma / Stage IIIA Breast Cancer / Stage IIIA Non-Small Cell Lung Cancer / Stage IIIB Breast Cancer / Stage IIIb Non-small Cell Lung Cancer / Stage IIIC Breast Cancer / Stage IV Breast Cancer / Stage IV Endometrial Carcinoma / Stage IV Neuroendocrine Carcinoma of the Skin / Stage IV Non-Small Cell Lung Cancer / Stage IV Ovarian Epithelial Cancer / Stage IV Ovarian Germ Cell Tumor / Stage IV Prostate Cancer / Stage IV Renal Cell Cancer / Stage IV Uterine Sarcoma / Stage IVA Cervical Cancer / Stage IVB Cervical Cancer / Thyroid Gland Medullary Carcinoma1
1CompletedTreatmentFacial Angiofibroma1
1CompletedTreatmentGlioblastomas1
1CompletedTreatmentGraft Versus Host Disease (GVHD) / Peripheral Blood Stem Cell Transplantation / Transplantation, Homologous1
1CompletedTreatmentHealthy Volunteers2
1CompletedTreatmentHepatic Complications1
1CompletedTreatmentHepatocellular,Carcinoma / Rapamycin1
1CompletedTreatmentKidney Diseases1
1CompletedTreatmentLeukemia Acute Myeloid Leukemia (AML)1
1CompletedTreatmentLeukemias / Malignant Lymphomas / Multiple Myeloma (MM) / Myelodysplastic Syndromes / Plasma Cell Neoplasms1
1CompletedTreatmentLiver Cancer1
1CompletedTreatmentLow-Grade Gliomas1
1CompletedTreatmentLung Cancer Non-Small Cell Cancer (NSCLC)2
1CompletedTreatmentLymphangioleiomyomatosis1
1CompletedTreatmentNeoplasms Metastasis / Tumors1
1CompletedTreatmentNeoplasms, Malignant1
1CompletedTreatmentPachyonychia Congenita1
1CompletedTreatmentPort Wine Stains2
1CompletedTreatmentRecurrent Adult Hodgkin's Lymphoma1
1CompletedTreatmentRecurrent Plasma Cell Myeloma / Refractory Plasma Cell Myeloma1
1CompletedTreatmentRejection, Transplant / Transplantation, Renal1
1CompletedTreatmentSarcomas1
1CompletedTreatmentTumors of the Central Nervous System / Tumors, Solid1
1CompletedTreatmentTumors, Solid1
1CompletedTreatmentType 1 Insulin-Dependent Diabetes Mellitus1
1CompletedTreatmentUnspecified Adult Solid Tumor, Protocol Specific1
1CompletedTreatmentUnspecified Adult Solid Tumor, Protocol Specific / Unspecified Childhood Solid Tumor, Protocol Specific1
1Not Yet RecruitingSupportive CareAllogeneic Hematopoietic Stem Cell Transplant Recipient / Hematopoietic and Lymphoid Cell Neoplasm1
1Not Yet RecruitingTreatmentVascular Anomalies1
1RecruitingTreatmentAcute Myelogenous Leukaemia (AML) / Breast Cancer / Malignant Neoplasm of Pancreas / Prostate Cancer1
1RecruitingTreatmentAdvanced Cancers / Advanced Malignant Neoplasm / Castleman's Disease / Digestive System Carcinoma / Erdheim-Chester Disease (ECD) / Lip and Oral Cavity Carcinoma / Lymphangioleiomyomatosis / Malignant Endocrine Neoplasm / Malignant Female Reproductive System Neoplasm / Malignant Male Reproductive System Neoplasm / Malignant respiratory tract neoplasm / Malignant Thoracic Neoplasm / Malignant Urinary System Neoplasm / Mesothelial Neoplasm / Metastatic Malignant Neoplasm / Neoplasms, Malignant / Neurofibromatosis Type 2 / Recurrent Adult Soft Tissue Sarcoma / Recurrent Breast Carcinoma / Recurrent Childhood Soft Tissue Sarcoma / Recurrent Digestive System Carcinoma / Recurrent Female Reproductive System Carcinoma / Recurrent Male Reproductive System Carcinoma / Recurrent Malignant Neoplasm / Recurrent Pharyngeal Carcinoma / Recurrent Thyroid Gland Carcinoma / Refractory Malignant Neoplasm / Soft Tissue Neoplasms / Stage III Breast Cancer AJCC V7 / Stage III Pharyngeal Cancer / Stage IIIA Breast Cancer AJCC v7 / Stage IIIB Breast Cancer AJCC v7 / Stage IIIC Breast Cancer AJCC v7 / Stage IV Breast Cancer AJCC v6 and v7 / Stage IV Pharyngeal Cancer / Stage IVA Pharyngeal Cancer / Stage IVB Pharyngeal Cancer / Stage IVC Pharyngeal Cancer / Thyroid Gland Neoplasm / Urothelial carcinoma ureter metastatic1
1RecruitingTreatmentAdvanced Malignant Solid Neoplasm / Recurrent Brain Neoplasm / Recurrent Malignant Solid Neoplasm / Refractory Brain Neoplasm / Tumors, Solid1
1RecruitingTreatmentAdvanced Solid Tumors / Lung Cancer Non-Small Cell Cancer (NSCLC)1
1RecruitingTreatmentAlcohol Dependence / Alcohol Use Disorder (AUD)1
1RecruitingTreatmentChildhood Solid Neoplasm / Recurrent Central Nervous System Neoplasm / Recurrent Malignant Solid Neoplasm / Recurrent Solid Neoplasm / Refractory Central Nervous System Neoplasm / Refractory Malignant Solid Neoplasm1
1RecruitingTreatmentDiffuse Intrinsic Pontine Glioma (DIPG)1
1RecruitingTreatmentGraft Versus Host Disease (GVHD) / Sickle Cell Disorders1
1RecruitingTreatmentPompe's Disease1
1RecruitingTreatmentPrimary Myelofibrosis / Secondary Myelofibrosis1
1RecruitingTreatmentProstate Cancer1
1RecruitingTreatmentPulmonary Hypertension (PH)1
1RecruitingTreatmentRenal Transplant Recipient Patients / Transplantation, Kidney / Transplantation, Renal1
1RecruitingTreatmentSarcoma, Osteogenic / Tumors, Solid1
1RecruitingTreatmentSickle Cell Disorders1
1RecruitingTreatmentSickle Cell-thalassemia Disease / Thalassaemic disorders1
1TerminatedPreventionAdult Hodgkin Lymphoma / Adult Myelodysplastic Syndrome / Agnogenic Myeloid Metaplasia / Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive / Childhood Chronic Myelogenous Leukemia, BCR-ABL1 Positive / Childhood Hodgkin Lymphoma / Childhood Myelodysplastic Syndrome / Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive / Primary Myelofibrosis / Recurrent Adult Acute Lymphoblastic Leukemia / Recurrent Adult Acute Myeloid Leukemia / Recurrent Adult Non-Hodgkin Lymphoma / Recurrent Childhood Acute Lymphoblastic Leukemia / Recurrent Childhood Acute Myeloid Leukemia / Recurrent Childhood Non-Hodgkin Lymphoma / Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive / Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive / Refractory Non-Hodgkin's lymphoma1
1TerminatedTreatmentAdenocarcinoma of the Prostate / Hormone-Resistant Prostate Cancer / Prostate Cancer / Recurrent Prostate Cancer / Stage IV Prostate Cancer1
1TerminatedTreatmentAdvanced Solid Cancers1
1TerminatedTreatmentEosinophilic Gastroenteritis / Oesophagitis, Eosinophilic1
1TerminatedTreatmentLeukemias / Lymphoma, Hodgkins / Multiple Myeloma (MM) / Non-Hodgkin's Lymphoma (NHL) / Smith-Magenis Syndrome1
1TerminatedTreatmentLeukemias / Malignant Lymphomas1
1TerminatedTreatmentLeukemias / Malignant Lymphomas / Plasma Cell Myeloma1
1TerminatedTreatmentTumors1
1Unknown StatusTreatmentCardiac Hypertrophy / Immunosuppression1
1Unknown StatusTreatmentRefractory Solid Tumors in Children1
1WithdrawnSupportive CareAcute myeloid leukaemia (in remission) / Primary Myelofibrosis / Primary Myelofibrosis, Prefibrotic Stage / Secondary Acute Myeloid Leukemia (Secondary AML, sAML) / Secondary Myelofibrosis1
1WithdrawnSupportive CareHematopoietic and Lymphoid Cell Neoplasm / Hematopoietic Cell Transplantation Recipient1
1WithdrawnTreatmentAcute Lymphoblastic Leukaemias (ALL)1
1WithdrawnTreatmentMultiple Myeloma (MM)1
1WithdrawnTreatmentNon-Small Cell Lung Cancer Recurrent / Squamous Cell Carcinoma of Lung / Stage IIIA Non-Small Cell Lung Cancer / Stage IIIb Non-small Cell Lung Cancer / Stage IV Non-Small Cell Lung Cancer / Unspecified Adult Solid Tumor, Protocol Specific1
1WithdrawnTreatmentNon-Small Cell Lung Cancer Recurrent / Unspecified Adult Solid Tumor, Protocol Specific1
1WithdrawnTreatmentRecurrent Fallopian Tube Carcinoma / Recurrent Ovarian Carcinoma / Recurrent Primary Peritoneal Carcinoma / Stage IIA Fallopian Tube Cancer / Stage IIA Ovarian Cancer / Stage IIB Fallopian Tube Cancer / Stage IIB Ovarian Cancer / Stage IIC Fallopian Tube Cancer / Stage IIC Ovarian Cancer / Stage IIIA Fallopian Tube Cancer / Stage IIIA Ovarian Cancer / Stage IIIA Primary Peritoneal Cancer / Stage IIIB Fallopian Tube Cancer / Stage IIIB Ovarian Cancer / Stage IIIB Primary Peritoneal Cancer / Stage IIIC Fallopian Tube Cancer / Stage IIIC Ovarian Cancer / Stage IIIC Primary Peritoneal Cancer / Stage IV Fallopian Tube Cancer / Stage IV Ovarian Cancer / Stage IV Primary Peritoneal Cancer1
1, 2Active Not RecruitingTreatmentAlemtuzumab (Campath ) / Graft Versus Host Disease (GVHD) / Peripheral Blood Stem Cell Transplantation / Sickle Cell Anemia / Sirolimus (Rapamune )1
1, 2Active Not RecruitingTreatmentChronic Granulomatous Disease (CGD)1
1, 2Active Not RecruitingTreatmentCognitive Side Effects of Cancer Therapy / Fatigue / Neurotoxicity Syndrome / Recurrent Grade 1 Follicular Lymphoma / Recurrent Grade 2 Follicular Lymphoma / Recurrent Grade 3 Follicular Lymphoma / Recurrent Mantle Cell Lymphoma / Recurrent Marginal Zone Lymphoma / Recurrent Small Lymphocytic Lymphoma / Therapy-Related Toxicity / Waldenström's Macroglobulinemia (WM)1
1, 2Active Not RecruitingTreatmentDesmoid Tumors1
1, 2Active Not RecruitingTreatmentLeukemias / Malignant Lymphomas1
1, 2Active Not RecruitingTreatmentPancreatic Adenocarcinoma Metastatic1
1, 2CompletedSupportive CareChronic Myeloproliferative Disorders / Graft Versus Host Disease (GVHD) / Leukemias / Malignant Lymphomas / Multiple Myeloma and Plasma Cell Neoplasm / Myelodysplastic Syndromes / Myelodysplastic/Myeloproliferative Diseases1
1, 2CompletedTreatmentAIDS-Related Hodgkin Lymphoma / Recurrent Adult Diffuse Large Cell Lymphoma / Recurrent Adult Hodgkin's Lymphoma / Recurrent Follicular Lymphoma / Recurrent Lymphoplasmacytic Lymphoma / Recurrent Marginal Zone Lymphoma / Recurrent Non-Hodgkin Lymphoma / Recurrent T-Cell Non-Hodgkin Lymphoma / Waldenström's Macroglobulinemia (WM)1
1, 2CompletedTreatmentAdenocarcinoma, Prostate / Hormone-Resistant Prostate Cancer / Recurrent Prostate Carcinoma / Stage IV Prostate Cancer1
1, 2CompletedTreatmentAdult Giant Cell Glioblastoma / Adult Glioblastoma / Adult Gliosarcoma / Recurrent Adult Brain Neoplasm / Recurrent Adult Brain Tumor1
1, 2CompletedTreatmentAdvanced Soft Tissue Sarcoma1
1, 2CompletedTreatmentAge-Related Macular Degeneration (ARMD)1
1, 2CompletedTreatmentAge-Related Macular Degeneration (ARMD) / Dry Macular Degeneration1
1, 2CompletedTreatmentAnemia, Hemolytic, Autoimmune / Autoimmune Lymphoproliferative Syndrome (ALPS) / Autoimmune Neutropenia / Autoimmune Pancytopenia / Evans Syndrome / Idiopathic Thrombocytopenic Purpura / Inflammatory Bowel Diseases (IBD) / Rheumatoid Arthritis / Systemic Lupus Erythematosus (SLE)1
1, 2CompletedTreatmentBrain and Central Nervous System Tumors1
1, 2CompletedTreatmentCNI Side Effects1
1, 2CompletedTreatmentClear Cell Renal Cell Carcinoma / Renal Cell Cancer, Recurrent / Stage IV Renal Cell Cancer1
1, 2CompletedTreatmentGraft Versus Host Disease (GVHD)1
1, 2CompletedTreatmentGraft Versus Host Disease (GVHD) / Prevention of GvHD in Patients With Hematological Malignancies Undergoing AHSCT1
1, 2CompletedTreatmentHuman Immunodeficiency Virus Type 1 (HIV-1) Infection1
1, 2CompletedTreatmentKidney Diseases / Renal Failure / Transplantation, Kidney1
1, 2CompletedTreatmentKidney Diseases / Transplantation, Kidney1
1, 2CompletedTreatmentMale Breast Cancer / Male Breast Carcinoma / Recurrent Breast Cancer / Recurrent Breast Carcinoma / Stage IV Breast Cancer / Stage IV Breast Cancer AJCC v6 and v71
1, 2CompletedTreatmentMalignancies, Hematologic / Multiple Myeloma (MM)1
1, 2CompletedTreatmentMalignant Peripheral Nerve Sheath Tumour (MPNST) / Sarcomas1
1, 2CompletedTreatmentMetastatic Bladder Cancer / Recurrent Bladder Carcinoma / Stage II Bladder Cancer / Stage III Bladder Cancer1
1, 2CompletedTreatmentMouth Neoplasms / Neoplasms, Head and Neck / Squamous Cell Carcinoma (SCC) / Tongue Neoplasms1
1, 2CompletedTreatmentPolycystic Kidney Diseases1
1, 2CompletedTreatmentProstate Cancer2
1, 2CompletedTreatmentRectum Cancer1
1, 2CompletedTreatmentRetinopathy, Diabetic1
1, 2CompletedTreatmentScleritis1
1, 2CompletedTreatmentSclerosis, Progressive Systemic1
1, 2CompletedTreatmentType 1 Insulin-Dependent Diabetes Mellitus2
1, 2Enrolling by InvitationTreatmentHutchinson-Gilford Progeria Syndrome1
1, 2Not Yet RecruitingTreatmentDonor / Non-Malignant Neoplasm1
1, 2RecruitingPreventionAcute GvHD / Graft Versus Host Disease (GVHD)1
1, 2RecruitingPreventionGraft Versus Host Disease (GVHD) / Lung Cancers1
1, 2RecruitingTreatmentActivated PI3K-delta Syndrome / Congenital Immunodeficiency1
1, 2RecruitingTreatmentAcute Lymphoblastic Leukaemia Recurrent / Acute Lymphoblastic Leukemia in Remission / Acute myeloid leukaemia (in remission) / Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome / Acute, recurrent Myeloid Leukemia / CD45-Positive Neoplastic Cells Present / Chronic Myelomonocytic Leukemia / High Risk Myelodysplastic Syndrome / Myelodysplastic Syndrome With Excess Blasts / Myeloproliferative Neoplasm, Unclassifiable / Myeloproliferative Neoplasms / Refractory Acute Lymphoblastic Leukemia / Refractory Acute Myeloid Leukemia1
1, 2RecruitingTreatmentAcute Lymphoblastic Leukaemia Recurrent / Acute Lymphoblastic Leukemia in Remission / Acute myeloid leukaemia (in remission) / Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome / Acute, recurrent Myeloid Leukemia / CD45-Positive Neoplastic Cells Present / Chronic Myelomonocytic Leukemia / High Risk Myelodysplastic Syndrome / Myelodysplastic Syndrome With Excess Blasts / Recurrent Adult Acute Lymphoblastic Leukemia / Recurrent Adult Acute Myeloid Leukemia / Recurrent Mixed Phenotype Acute Leukemia / Refractory Acute Lymphoblastic Leukemia / Refractory Acute Myeloid Leukemia / Refractory Adult Acute Lymphoblastic Leukemia / Refractory Mixed Phenotype Acute Leukemia1
1, 2RecruitingTreatmentAdenocarcinoma of the Lung / Extensive Stage Small Cell Lung Carcinoma / Recurrent Non-Small Cell Lung Carcinoma / Recurrent Small Cell Lung Carcinoma / Squamous Cell Lung Carcinoma / Stage IIIA Non-Small Cell Lung Cancer / Stage IIIb Non-small Cell Lung Cancer / Stage IV Non-Small Cell Lung Cancer1
1, 2RecruitingTreatmentCastration Levels of Testosterone / Castration-Resistant Prostate Carcinoma / Hormone-Resistant Prostate Cancer / Metastatic Hormone Refractory Prostate Cancer / Prostate Carcinoma Metastatic in the Bone / PSA Level Greater Than or Equal to Two / PSA Progression / Recurrent Prostate Carcinoma / Stage IV Prostate Cancer / Stage IV Prostate Cancer AJCC v71
1, 2RecruitingTreatmentChondrosarcomas / Ewing's Sarcoma (ES) / Liposarcoma / Sarcoma, Osteogenic / Undifferentiated Pleomorphic Sarcoma1
1, 2RecruitingTreatmentChronic Granulomatous Disease Transplant / X-Linked Chronic Granulomatious Disease1
1, 2RecruitingTreatmentCongenital Hemolytic Anemia / Congenital Hypoplastic Anemia / Sickle Cell Disorders1
1, 2RecruitingTreatmentDermal Atrophy1
1, 2RecruitingTreatmentGraft Versus Host Disease (GVHD) / Neoplasms, Hematologic1
1, 2RecruitingTreatmentHematopoietic Stem Cell Transplantation (HSCT) / Multiple Myeloma (MM)1
1, 2RecruitingTreatmentMalignancies, Hematologic1
1, 2RecruitingTreatmentMalignant Neoplasm of Pancreas1
1, 2RecruitingTreatmentMalignant Peripheral Nerve Sheath Tumour (MPNST) / Sarcomas1
1, 2RecruitingTreatmentMetastatic Colorectal Cancer (MCRC)1
1, 2RecruitingTreatmentNon-muscle Invasive Bladder Cancer (NMIBC)1
1, 2RecruitingTreatmentSickle Cell Disorders2
1, 2SuspendedTreatmentLocally Advanced Soft Tissue Sarcoma, Excluding Liposarcoma / Metastatic Soft Tissue Sarcoma, Excluding Liposarcoma / Soft Tissue Sarcoma (STS)1
1, 2TerminatedTreatmentAdult Anaplastic Astrocytoma / Adult Anaplastic Oligodendroglioma / Adult Diffuse Astrocytoma / Adult Giant Cell Glioblastoma / Adult Glioblastoma / Adult Gliosarcoma / Adult Mixed Glioma / Adult Oligodendroglioma / Recurrent Adult Brain Neoplasm1
1, 2TerminatedTreatmentAge-Related Macular Degeneration (ARMD) / Subfoveal Choroidal Neovascularization1
1, 2TerminatedTreatmentAll / Burkitt's Lymphoma / CML / Leukaemia, Lymphoblastic1
1, 2TerminatedTreatmentDisseminated Sclerosis / Relapsing Remitting Multiple Sclerosis (RRMS)1
1, 2TerminatedTreatmentGraft Versus Host Disease (GVHD)1
1, 2TerminatedTreatmentHypoglycemia / Type 1 Insulin-Dependent Diabetes Mellitus1
1, 2TerminatedTreatmentLung Cancer Non-Small Cell Cancer (NSCLC)1
1, 2TerminatedTreatmentMultiple Myeloma (MM)1
1, 2TerminatedTreatmentMultiple Myeloma (MM) / Myeloma, Plasma-Cell / Myeloma-Multiple / Plasma Cell Myeloma1
1, 2Unknown StatusTreatmentAplastic Anemia1
1, 2Unknown StatusTreatmentEnd-Stage Renal Disease (ESRD) / Renal Failure1
1, 2WithdrawnSupportive CareGraft Versus Host Disease (GVHD) / Leukemias / Myelodysplastic Syndromes1
1, 2WithdrawnTreatmentHyperinsulinism1
1, 2WithdrawnTreatmentLung Cancers1
1, 2WithdrawnTreatmentMultiple Myeloma (MM) / Refractory Multiple Myeloma1
2Active Not RecruitingOtherDepression1
2Active Not RecruitingPreventionGraft Versus Host Disease (GVHD)2
2Active Not RecruitingTreatmentAcute Biphenotypic Leukemia (ABL) / Acute Lymphoblastic Leukemia (ALL)/T Lymphoblastic Lymphoma / Acute Myelogenous Leukaemia (AML) / Acute Undifferentiated Leukemia (AUL) / Chemotherapy-sensitive Lymphoma / Chronic Lymphocytic Leukaemia (CLL) / Myelodysplastic Syndromes (MDS)1
2Active Not RecruitingTreatmentAcute GvHD1
2Active Not RecruitingTreatmentAcute Lymphoblastic Leukaemias (ALL) / Adult Acute Lymphoblastic Leukemia in Remission / Adult B Acute Lymphoblastic Leukemia / Adult B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1 / Adult B Acute Lymphoblastic Leukemia With t(9;22)(q34;q11.2); BCR-ABL1 / Adult L1 Acute Lymphoblastic Leukemia / Adult L2 Acute Lymphoblastic Leukemia / Adult T Acute Lymphoblastic Leukemia / Recurrent Adult Acute Lymphoblastic Leukemia / Untreated Adult Acute Lymphoblastic Leukemia1
2Active Not RecruitingTreatmentAcute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome / Adult Acute Lymphoblastic Leukemia in Remission / Adult Acute Megakaryoblastic Leukemia (M7) / Adult Acute Minimally Differentiated Myeloid Leukemia (M0) / Adult Acute Monoblastic Leukemia (M5a) / Adult Acute Monocytic Leukemia (M5b) / Adult Acute Myeloblastic Leukemia With Maturation (M2) / Adult Acute Myeloblastic Leukemia Without Maturation (M1) / Adult Acute Myeloid Leukemia in Remission / Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities / Adult Acute Myeloid Leukemia With Del(5q) / Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) / Adult Acute Myeloid Leukemia With T(16;16)(p13;q22) / Adult Acute Myeloid Leukemia With T(8;21)(q22;q22) / Adult Acute Myelomonocytic Leukemia (M4) / Adult Erythroleukemia (M6a) / Adult Pure Erythroid Leukemia (M6b) / Chronic Myelomonocytic Leukemia / Myelodysplastic Syndrome / Previously Treated Myelodysplastic Syndromes / Secondary Acute Myeloid Leukemia (Secondary AML, sAML) / Secondary Acute Myeloid Leukemia in Remission / Secondary Myelodysplastic Syndromes / Therapy-Related Myelodysplastic Syndrome1
2Active Not RecruitingTreatmentAdult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities / Adult Acute Myeloid Leukemia With Del(5q) / Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) / Adult Acute Myeloid Leukemia With T(15;17)(q22;q12) / Adult Acute Myeloid Leukemia With T(16;16)(p13;q22) / Adult Acute Myeloid Leukemia With T(8;21)(q22;q22) / De Novo Myelodysplastic Syndromes / Myelodysplastic Syndrome With Isolated Del(5q) / Previously Treated Myelodysplastic Syndromes / Recurrent Adult Acute Myeloid Leukemia1
2Active Not RecruitingTreatmentAplastic Anemia / Donor / Immunodeficiency Syndrome / Non-Cancer Diagnosis / Severe Aplastic Anemia (SAA)1
2Active Not RecruitingTreatmentCapillary Lymphatic Arterial Venous Malformations / Capillary Venous Lymphatic Malformation / Kaposiform Hemangioendotheliomas / Lymphangiectasia Syndromes / Microcystic Lymphatic Malformation / Mucocutaneous Lymphangiomatosis and Thrombocytopenia / PTEN Overgrowth Syndrome With Vascular Anomaly / Tufted Angioma / Venous Lymphatic Malformation1
2Active Not RecruitingTreatmentChronic Lymphocytic Leukaemia (CLL) / Lymphoma, Hodgkins / Lymphoma, Large B-Cell, Diffuse (DLBCL) / Lymphoma, Low-Grade / Mantle Cell Lymphoma (MCL) / Small Lymphocytic Lymphoma / T-Cell Lymphomas1
2Active Not RecruitingTreatmentChronic Myeloproliferative Disorders / Graft Versus Host Disease (GVHD) / Leukemias / Malignant Lymphomas / Multiple Myeloma and Plasma Cell Neoplasm / Myelodysplastic Syndromes / Myelodysplastic/Myeloproliferative Diseases / Precancerous/Nonmalignant Condition1
2Active Not RecruitingTreatmentEndometrial Adenocarcinomas / Endometrial Adenosquamous Carcinoma / Endometrial Clear Cell Adenocarcinoma / Endometrial Serous Adenocarcinoma / Recurrent Uterine Corpus Carcinoma / Stage IIIA Uterine Corpus Cancer / Stage IIIA Uterine Corpus Cancer AJCC v7 / Stage IIIB Uterine Corpus Cancer / Stage IIIB Uterine Corpus Cancer AJCC v7 / Stage IIIC Uterine Corpus Cancer / Stage IIIC Uterine Corpus Cancer AJCC v7 / Stage IVA Uterine Corpus Cancer / Stage IVA Uterine Corpus Cancer AJCC v7 / Stage IVB Uterine Corpus Cancer / Stage IVB Uterine Corpus Cancer AJCC v71
2Active Not RecruitingTreatmentEpidermolysis Bullosa Simplex / Epidermolysis Bullosa Simplex Kobner / Weber-Cockayne Syndrome1
2Active Not RecruitingTreatmentEstrogen Receptor Positive / HER2/Neu Positive / Male Breast Carcinoma / Progesterone Receptor Positive / Recurrent Breast Carcinoma / Stage III Breast Cancer AJCC V7 / Stage IIIA Breast Cancer / Stage IIIA Breast Cancer AJCC v7 / Stage IIIB Breast Cancer / Stage IIIB Breast Cancer AJCC v7 / Stage IIIC Breast Cancer / Stage IIIC Breast Cancer AJCC v7 / Stage IV Breast Cancer / Stage IV Breast Cancer AJCC v6 and v71
2Active Not RecruitingTreatmentGraft Versus Host Disease (GVHD)1
2Active Not RecruitingTreatmentGraft Versus Host Disease (GVHD) / Leukemias / Malignant Lymphomas1
2Active Not RecruitingTreatmentHaemangioma-thrombocytopenia syndrome / Kaposiform Hemangioendothelioma (KHE) / Tufted Angioma1
2Active Not RecruitingTreatmentLeukemias / Malignant Lymphomas1
2Active Not RecruitingTreatmentLeukemias / Malignant Lymphomas / Myelodysplastic Syndromes / Myeloproliferative Disorders1
2Active Not RecruitingTreatmentOvarian Serous Tumor / Recurrent Ovarian Carcinoma1
2CompletedNot AvailableSturge - Weber Syndrome (SWS)1
2CompletedBasic ScienceAging1
2CompletedPreventionAcute Lymphoblastic Leukaemias (ALL) / Acute Myelogenous Leukaemia (AML) / Chronic Chronic myelogenous leukemia / Graft Versus Host Disease (GVHD) / Hodgkins Disease (HD) / Myelodysplastic Syndromes / Non-Hodgkin's Lymphoma (NHL)1
2CompletedPreventionAcute Lymphoblastic Leukaemias (ALL) / Hodgkins Disease (HD) / Malignancies, Hematologic / Myelodysplastic Syndrome / Myelogenous Leukemia / Non-Hodgkin's Lymphoma (NHL)1
2CompletedPreventionBlood and Marrow Transplant (BMT) / Leukemias / Non-Hodgkin's Lymphoma (NHL)1
2CompletedPreventionChronic Renal Failure (CRF) / Immunosuppression / Transplantation, Kidney1
2CompletedPreventionGraft Versus Host Disease, Acute1
2CompletedPreventionTransplant, Kidney1
2CompletedPreventionType 1 Insulin-Dependent Diabetes Mellitus1
2CompletedSupportive CareChronic Myeloproliferative Disorders / Graft Versus Host Disease (GVHD) / Infection NOS / Leukemias / Malignant Lymphomas / Multiple Myeloma and Plasma Cell Neoplasm / Myelodysplastic Syndromes / Myelodysplastic/Myeloproliferative Neoplasms / Precancerous Conditions / Secondary Myelofibrosis / Small Intestine Cancer1
2CompletedSupportive CareGraft Versus Host Disease (GVHD)1
2CompletedTreatmentAccelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive / Adult Acute Lymphoblastic Leukemia in Remission / Adult Acute Myeloid Leukemia in Remission / Adult Acute Myeloid Leukemia With Inv(16)(p13.1q22); CBFB-MYH11 / Adult Acute Myeloid Leukemia With t(8;21)(q22;q22); RUNX1-RUNX1T1 / Adult Acute Myeloid Leukemia With t(9;11)(p22;q23); MLLT3- / Adult Acute Myeloid Leukemia With t(9;11)(p22;q23); MLLT3-MLL / Adult Acute Promyelocytic Leukemia With t(15;17)(q22;q12); PML-RARA / Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative / Blast Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive / Childhood Acute Lymphoblastic Leukemia in Remission / Childhood Acute Myeloid Leukemia in Remission / Childhood Burkitt Lymphoma / Childhood Chronic Myelogenous Leukemia, BCR-ABL1 Positive / Childhood Diffuse Large Cell Lymphoma / Childhood Immunoblastic Lymphoma / Childhood Myelodysplastic Syndrome / Chronic Lymphocytic Leukemia (CLL) - Refractory / Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable / Previously Treated Myelodysplastic Syndromes / Recurrent Adult Acute Lymphoblastic Leukemia / Recurrent Adult Acute Myeloid Leukemia / Recurrent Adult Burkitt Lymphoma / Recurrent Adult Diffuse Large Cell Lymphoma / Recurrent Adult Diffuse Mixed Cell Lymphoma / Recurrent Adult Diffuse Small Cleaved Cell Lymphoma / Recurrent Adult Hodgkin's Lymphoma / Recurrent Adult Immunoblastic Lymphoma / Recurrent Adult Lymphoblastic Lymphoma / Recurrent Childhood Acute Lymphoblastic Leukemia / Recurrent Childhood Acute Myeloid Leukemia / Recurrent Childhood Anaplastic Large Cell Lymphoma / Recurrent Childhood Burkitt Lymphoma / Recurrent Childhood Hodgkin Lymphoma / Recurrent Childhood Large Cell Lymphoma / Recurrent Childhood Lymphoblastic Lymphoma / Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive / Recurrent Grade 1 Follicular Lymphoma / Recurrent Grade 2 Follicular Lymphoma / Recurrent Grade 3 Follicular Lymphoma / Recurrent Mantle Cell Lymphoma / Recurrent Marginal Zone Lymphoma / Recurrent Small Lymphocytic Lymphoma / Refractory Plasma Cell Myeloma / Secondary Myelodysplastic Syndromes / Stage I Adult Burkitt Lymphoma / Stage I Adult Diffuse Large Cell Lymphoma / Stage I Adult Diffuse Mixed Cell Lymphoma / Stage I Adult Immunoblastic Lymphoma / Stage I Adult Lymphoblastic Lymphoma / Stage I Childhood Anaplastic Large Cell Lymphoma / Stage I Childhood Burkitt Lymphoma / Stage I Childhood Large Cell Lymphoma / Stage I Childhood Lymphoblastic Lymphoma / Stage I Grade 1 Follicular Lymphoma / Stage I Grade 2 Follicular Lymphoma / Stage I Grade 3 Follicular Lymphoma / Stage I Mantle Cell Lymphoma / Stage I Marginal Zone Lymphoma / Stage I Small Lymphocytic Lymphoma / Stage II Adult Contiguous Immunoblastic Lymphoma / Stage II Adult Non-Contiguous Immunoblastic Lymphoma / Stage II Childhood Anaplastic Large Cell Lymphoma / Stage II Childhood Burkitt Lymphoma / Stage II Childhood Lymphoblastic Lymphoma / Stage II Contiguous Adult Burkitt Lymphoma / Stage II Contiguous Adult Diffuse Large Cell Lymphoma / Stage II Contiguous Adult Diffuse Mixed Cell Lymphoma / Stage II Contiguous Adult Diffuse Small Cleaved Cell Lymphoma / Stage II Contiguous Adult Lymphoblastic Lymphoma / Stage II Contiguous Mantle Cell Lymphoma / Stage II Grade 1 Contiguous Follicular Lymphoma / Stage II Grade 1 Non-Contiguous Follicular Lymphoma / Stage II Grade 2 Contiguous Follicular Lymphoma / Stage II Grade 2 Non-Contiguous Follicular Lymphoma / Stage II Grade 3 Contiguous Follicular Lymphoma / Stage II Grade 3 Non-Contiguous Follicular Lymphoma / Stage II Non-Contiguous Adult Burkitt Lymphoma / Stage II Non-Contiguous Adult Diffuse Large Cell Lymphoma / Stage II Non-Contiguous Adult Diffuse Mixed Cell Lymphoma / Stage II Non-Contiguous Adult Diffuse Small Cleaved Cell Lymphoma / Stage II Non-Contiguous Adult Lymphoblastic Lymphoma / Stage II Non-Contiguous Mantle Cell Lymphoma / Stage II Small Lymphocytic Lymphoma / Stage III Adult Burkitt Lymphoma / Stage III Adult Diffuse Large Cell Lymphoma / Stage III Adult Diffuse Mixed Cell Lymphoma / Stage III Adult Diffuse Small Cleaved Cell Lymphoma / Stage III Adult Immunoblastic Lymphoma / Stage III Adult Lymphoblastic Lymphoma / Stage III Childhood Anaplastic Large Cell Lymphoma / Stage III Childhood Burkitt Lymphoma / Stage III Childhood Large Cell Lymphoma / Stage III Childhood Lymphoblastic Lymphoma / Stage III Grade 1 Follicular Lymphoma / Stage III Grade 2 Follicular Lymphoma / Stage III Grade 3 Follicular Lymphoma / Stage III Mantle Cell Lymphoma / Stage III Marginal Zone Lymphoma / Stage III Small Lymphocytic Lymphoma / Stage IV Adult Burkitt Lymphoma / Stage IV Adult Diffuse Large Cell Lymphoma / Stage IV Adult Diffuse Mixed Cell Lymphoma / Stage IV Adult Diffuse Small Cleaved Cell Lymphoma / Stage IV Adult Immunoblastic Lymphoma / Stage IV Adult Lymphoblastic Lymphoma / Stage IV Childhood Anaplastic Large Cell Lymphoma / Stage IV Childhood Burkitt Lymphoma / Stage IV Childhood Large Cell Lymphoma / Stage IV Childhood Lymphoblastic Lymphoma / Stage IV Grade 1 Follicular Lymphoma / Stage IV Grade 2 Follicular Lymphoma / Stage IV Grade 3 Follicular Lymphoma / Stage IV Mantle Cell Lymphoma / Stage IV Marginal Zone Lymphoma / Stage IV Small Lymphocytic Lymphoma / Waldenström's Macroglobulinemia (WM)1
2CompletedTreatmentAcute Lymphoblastic Leukaemias (ALL)1
2CompletedTreatmentAdenomatous Polyposis Coli1
2CompletedTreatmentAdult Acute Lymphoblastic Leukemia / Adult Acute Myeloid Leukemia / Adult Diffuse Large B-Cell Lymphoma / Adult Myelodysplastic Syndrome / Adult Non-Hodgkin's Lymphoma / Aggressive Non-Hodgkin Lymphoma / Childhood Acute Lymphoblastic Leukemia / Childhood Acute Myeloid Leukemia / Childhood Diffuse Large B -Cell Lymphoma / Childhood Diffuse Large B-Cell Lymphoma / Childhood Myelodysplastic Syndrome / Childhood Non-Hodgkin's Lymphoma / Chronic Lymphocytic Leukaemia (CLL) / Chronic Lymphocytic Leukemia (CLL) - Refractory / Chronic Lymphocytic Leukemia in Remission / Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive / Chronic, recurrent Lymphocytic Leukemia / Hematopoietic and Lymphoid Cell Neoplasm / Leukemia, Prolymphocytic / Mantle Cell Lymphoma (MCL) / Plasma Cell Myeloma / Recurrent Diffuse Large B-Cell Lymphoma / Recurrent Hodgkin Lymphoma / T-cell chronic lymphocytic leukaemia / T-Cell Prolymphocytic Leukemia / Waldenström's Macroglobulinemia (WM)1
2CompletedTreatmentAdult Acute Megakaryoblastic Leukemia (M7) / Adult Acute Minimally Differentiated Myeloid Leukemia (M0) / Adult Acute Monoblastic Leukemia (M5a) / Adult Acute Monocytic Leukemia (M5b) / Adult Acute Myeloblastic Leukemia With Maturation (M2) / Adult Acute Myeloblastic Leukemia Without Maturation (M1) / Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities / Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) / Adult Acute Myeloid Leukemia With T(16;16)(p13;q22) / Adult Acute Myeloid Leukemia With T(8;21)(q22;q22) / Adult Acute Myelomonocytic Leukemia (M4) / Adult Erythroleukemia (M6a) / Adult Pure Erythroid Leukemia (M6b) / Recurrent Adult Acute Myeloid Leukemia1
2CompletedTreatmentAdult Hepatocellular Carcinoma / Advanced Adult Hepatocellular Carcinoma / Endometrial Serous Adenocarcinoma / Localized Non-Resectable Adult Liver Carcinoma / Lung Carcinoid Tumor / Malignant Ovarian Mixed Epithelial Tumor / Malignant Pancreatic Gastrinoma / Malignant Pancreatic Glucagonoma / Malignant Pancreatic Insulinoma / Malignant Pancreatic Somatostatinoma / Metastatic Digestive System Neuroendocrine Tumor G1 / Ovarian Carcinosarcoma / Ovarian Endometrioid Adenocarcinoma / Ovarian Seromucinous Carcinoma / Ovarian Serous Surface Papillary Adenocarcinoma / Pancreatic Alpha Cell Adenoma / Pancreatic Beta Cell Adenoma / Pancreatic Delta Cell Adenoma / Pancreatic G-Cell Adenoma / Pancreatic Polypeptide Tumor / Recurrent Adult Liver Carcinoma / Recurrent Digestive System Neuroendocrine Tumor G1 / Recurrent Fallopian Tube Carcinoma / Recurrent Ovarian Carcinoma / Recurrent Pancreatic Neuroendocrine Carcinoma / Recurrent Primary Peritoneal Carcinoma / Recurrent Uterine Corpus Carcinoma / Regional Digestive System Neuroendocrine Tumor G1 / Stage IIIA Fallopian Tube Cancer / Stage IIIA Ovarian Cancer / Stage IIIA Primary Peritoneal Cancer / Stage IIIA Uterine Corpus Cancer / Stage IIIB Fallopian Tube Cancer / Stage IIIB Ovarian Cancer / Stage IIIB Primary Peritoneal Cancer / Stage IIIB Uterine Corpus Cancer / Stage IIIC Fallopian Tube Cancer / Stage IIIC Ovarian Cancer / Stage IIIC Primary Peritoneal Cancer / Stage IIIC Uterine Corpus Cancer / Stage IV Fallopian Tube Cancer / Stage IV Ovarian Cancer / Stage IV Primary Peritoneal Cancer / Stage IVA Uterine Corpus Cancer / Stage IVB Uterine Corpus Cancer / Uterine Carcinosarcoma1
2CompletedTreatmentAdult Rhabdomyosarcoma / Childhood Alveolar Rhabdomyosarcoma / Childhood Pleomorphic Rhabdomyosarcoma / Childhood Rhabdomyosarcoma With Mixed Embryonal and Alveolar Features / Previously Treated Childhood Rhabdomyosarcoma / Recurrent Adult Soft Tissue Sarcoma / Recurrent Childhood Rhabdomyosarcoma1
2CompletedTreatmentAge-Related Macular Degeneration (ARMD)1
2CompletedTreatmentAge-Related Macular Degeneration (ARMD) / Subfoveal Choroidal Neovascularization1
2CompletedTreatmentAngiofibromas / Tuberous Sclerosis1
2CompletedTreatmentAutism, Early Infantile / Self Injurious Behaviour / Tuberous Sclerosis Complex (TSC)1
2CompletedTreatmentBlood and Marrow Transplant (BMT) / Graft Versus Host Disease (GVHD)1
2CompletedTreatmentBreast Cancer1
2CompletedTreatmentChildhood Alveolar Soft Part Sarcoma / Childhood Alveolar Soft-part Sarcoma / Childhood Angiosarcoma / Childhood Epithelioid Sarcoma / Childhood Fibrosarcoma / Childhood Gliosarcoma / Childhood Leiomyosarcoma / Childhood Liposarcoma / Childhood Malignant Peripheral Nerve Sheath Tumor / Childhood Neurofibrosarcoma / Childhood Synovial Sarcoma / Previously Treated Childhood Rhabdomyosarcoma / Recurrent Childhood Rhabdomyosarcoma / Recurrent Childhood Soft Tissue Sarcoma / Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor / Recurrent Osteosarcoma / Rhabdomyosarcomas1
2CompletedTreatmentClear Cell Renal Cell Carcinoma / Renal Cell Cancer, Recurrent / Renal Cell Carcinoma Recurrent / Stage IV Renal Cell Cancer / Stage IV Renal Cell Cancer AJCC V71
2CompletedTreatmentCowden's Disease / Hamartoma Syndrome, Multiple1
2CompletedTreatmentDiabetes Mellitus, Insulin-Dependent1
2CompletedTreatmentDiabetic Macular Edema (DME)1
2CompletedTreatmentDisorder Related to Renal Transplantation1
2CompletedTreatmentDry Eye1
2CompletedTreatmentEndometrial Carcinoma / Recurrent Uterine Corpus Carcinoma / Stage IIIA Uterine Corpus Cancer / Stage IIIB Uterine Corpus Cancer / Stage IIIC1 Uterine Corpus Cancer / Stage IIIC2 Uterine Corpus Cancer / Stage IVA Uterine Corpus Cancer / Stage IVB Uterine Corpus Cancer1
2CompletedTreatmentEngraftment Syndrome / Graft Versus Host Disease (GVHD) / Renal Cell Adenocarcinoma1
2CompletedTreatmentExudative Age Related Macular Degeneration1
2CompletedTreatmentGanglioneuroblastoma / Recurrent Neuroblastoma1
2CompletedTreatmentGenetics / Growth Disorders / PIK3CA-Related Overgrowth Spectrum (PROS)1
2CompletedTreatmentGlioblastomas / Gliosarcoma1
2CompletedTreatmentGlomerulonephritis, Membranous / Lupus Membranous Nepropathy1
2CompletedTreatmentGlomerulosclerosis, Focal Segmental1
2CompletedTreatmentGout Chronic / Hyperuricemia1
2CompletedTreatmentGraft Versus Host Disease (GVHD)2
2CompletedTreatmentGraft Versus Host Disease (GVHD) / Malignancies, Hematologic1
2CompletedTreatmentH1N1 Pneumonia / Hypoxemia1
2CompletedTreatmentHepatocellular,Carcinoma1
2CompletedTreatmentHodgkins Disease (HD) / Leukaemia, Lymphoblastic / Multiple Myeloma (MM) / Myelogenous Leukemia / Non-Hodgkin's Lymphoma (NHL)1
2CompletedTreatmentHypoglycemia / Metabolic Diseases / Type 1 Insulin-Dependent Diabetes Mellitus1
2CompletedTreatmentIgA Glomerulonephritis / IgA Nephropathy1
2CompletedTreatmentKidney, Polycystic1
2CompletedTreatmentLeukemia Acute Myeloid Leukemia (AML)1
2CompletedTreatmentLeukemias / Malignancies / Malignant Lymphomas1
2CompletedTreatmentLeukemias / Malignant Lymphomas / Myelodysplastic Syndromes1
2CompletedTreatmentLymphangioleiomyomatosis / Tuberous Sclerosis1
2CompletedTreatmentMalignant Neoplasm of Pancreas1
2CompletedTreatmentMetastatic Bone Sarcomas / Recurrent Adult Soft Tissue Sarcoma / Recurrent Osteosarcoma / Stage III Adult Soft Tissue Sarcoma / Stage IV Adult Soft Tissue Sarcoma1
2CompletedTreatmentNeurofibromatosis Type 11
2CompletedTreatmentOvarian Clear Cell Cystadenocarcinoma / Stage III Ovarian Cancer / Stage IV Ovarian Cancer1
2CompletedTreatmentRecurrent Adult Soft Tissue Sarcoma / Stage III Adult Soft Tissue Sarcoma / Stage IV Adult Soft Tissue Sarcoma1
2CompletedTreatmentRecurrent Hypopharyngeal Squamous Cell Carcinoma / Recurrent Laryngeal Squamous Cell Carcinoma / Recurrent Laryngeal Verrucous Carcinoma / Recurrent Lip and Oral Cavity Squamous Cell Carcinoma / Recurrent Metastatic Squamous Cell Carcinoma in the Neck With Occult Primary / Recurrent Nasal Cavity and Paranasal Sinus Squamous Cell Carcinoma / Recurrent Nasopharyngeal Keratinizing Squamous Cell Carcinoma / Recurrent Oral Cavity Verrucous Carcinoma / Recurrent Oropharyngeal Squamous Cell Carcinoma / Recurrent Salivary Gland Carcinoma / Salivary Gland Squamous Cell Carcinoma / Squamous Cell Carcinoma Metastatic in the Neck With Occult Primary / Stage IV Hypopharyngeal Squamous Cell Carcinoma / Stage IV Nasopharyngeal Keratinizing Squamous Cell Carcinoma / Stage IVA Laryngeal Squamous Cell Carcinoma / Stage IVA Laryngeal Verrucous Carcinoma / Stage IVA Lip and Oral Cavity Squamous Cell Carcinoma / Stage IVA Major Salivary Gland Carcinoma / Stage IVA Nasal Cavity and Paranasal Sinus Squamous Cell Carcinoma / Stage IVA Oral Cavity Verrucous Carcinoma / Stage IVA Oropharyngeal Squamous Cell Carcinoma / Stage IVB Laryngeal Squamous Cell Carcinoma / Stage IVB Laryngeal Verrucous Carcinoma / Stage IVB Lip and Oral Cavity Squamous Cell Carcinoma / Stage IVB Major Salivary Gland Carcinoma / Stage IVB Nasal Cavity and Paranasal Sinus Squamous Cell Carcinoma / Stage IVB Oral Cavity Verrucous Carcinoma / Stage IVB Oropharyngeal Squamous Cell Carcinoma / Stage IVC Laryngeal Squamous Cell Carcinoma / Stage IVC Laryngeal Verrucous Carcinoma / Stage IVC Lip and Oral Cavity Squamous Cell Carcinoma / Stage IVC Major Salivary Gland Carcinoma / Stage IVC Nasal Cavity and Paranasal Sinus Squamous Cell Carcinoma / Stage IVC Oral Cavity Verrucous Carcinoma / Stage IVC Oropharyngeal Squamous Cell Carcinoma / Tongue Carcinoma1
2CompletedTreatmentRecurrent Implantation Failure (RIF)1
2CompletedTreatmentRecurrent Melanoma / Stage IIIA Skin Melanoma / Stage IIIB Skin Melanoma / Stage IIIC Skin Melanoma / Stage IV Skin Melanoma1
2CompletedTreatmentRenal Cell Adenocarcinoma1
2CompletedTreatmentRenal Failure1
2CompletedTreatmentSarcoma, Osteogenic2
2CompletedTreatmentSarcomas1
2CompletedTreatmentSegmental Overgrowth Disorders Due to PIK3CA1
2CompletedTreatmentSystemic Lupus Erythematosus (SLE)1
2CompletedTreatmentTransplantation, Kidney4
2CompletedTreatmentType 1 Insulin-Dependent Diabetes Mellitus3
2CompletedTreatmentVascular Malformations1
2Enrolling by InvitationTreatmentHeart Transplantation1
2Not Yet RecruitingBasic ScienceCerebral Aneurysms1
2Not Yet RecruitingTreatmentEwing's Sarcoma (ES) / Sarcomas1
2Not Yet RecruitingTreatmentLingual Microcystic Lymphatic Malformations1
2Not Yet RecruitingTreatmentSclerosis, Progressive Systemic1
2RecruitingBasic ScienceCancer of the Breast1
2RecruitingBasic ScienceRecurrent Adult Acute Myeloid Leukemia / Secondary Acute Myeloid Leukemia (Secondary AML, sAML) / Untreated Adult Acute Myeloid Leukemia1
2RecruitingPreventionAcute Graft-Versus-Host Disease / Chronic Graft Versus Host Disease / Steroid Refractory Graft-Versus-Host Disease1
2RecruitingPreventionAcute myeloid leukaemia (in remission) / Adult Acute Lymphoblastic Leukemia in Complete Remission / Chronic Myelogenous Leukemia, BCR-ABL1 Positive in Remission / Chronic Myelomonocytic Leukemia in Remission / Graft Versus Host Disease (GVHD) / Lymphoma, Hodgkins / Minimal Residual Disease / Myelodysplastic Syndrome / Myeloproliferative Neoplasms / Non-Hodgkin's Lymphoma (NHL) / Plasma Cell Myeloma / Severe Aplastic Anemia (SAA) / Waldenström's Macroglobulinemia (WM)1
2RecruitingPreventionEnd Stage Renal Disease (ESRD) / Venous Stenosis1
2RecruitingPreventionGraft Versus Host Disease (GVHD) / Haematological Malignancies1
2RecruitingTreatmentAcute Lymphoblastic Leukaemias (ALL) / Aggressive Non-Hodgkin Lymphoma / Chronic Lymphocytic Leukaemia (CLL) / Chronic, recurrent Lymphocytic Leukemia / Hematopoietic Cell Transplant Recipient / Hematopoietic Cell Transplantation Recipient / Leukemia Acute Myeloid Leukemia (AML) / Leukemia, Prolymphocytic / Loss of Chromosome 17p / Lymphoma, Large B-Cell, Diffuse (DLBCL) / Mantle Cell Lymphoma (MCL) / Myelodysplastic Syndrome / Myelodysplastic/Myeloproliferative Neoplasms / Recurrent Chronic Myelogenous Leukemia, BCR-ABL1 Positive / Recurrent Hodgkin Lymphoma / Recurrent Plasma Cell Myeloma / Recurrent Small Lymphocytic Lymphoma / Recurrent Waldenstrom Macroglobulinemia / T-cell chronic lymphocytic leukaemia1
2RecruitingTreatmentAcute Lymphoblastic Leukaemias (ALL) / Chronic Myeloid Leukemia (CML) / Granulocytic Sarcoma / Juvenile Myelomonocytic Leukemia (JMML) / Leukemia Acute Myeloid Leukemia (AML) / Myelodysplastic Syndromes (MDS) / Non-Hodgkin's Lymphoma (NHL)1
2RecruitingTreatmentAcute Lymphoblastic Leukemia/Lymphoma / Biphenotypic/Undifferentiated/Prolymphocytic Leukemias / Bone Marrow Failure Syndromes / Burkitt's Lymphoma / Chronic Chronic myelogenous leukemia / Chronic Lymphocytic Leukaemia (CLL) / Follicular Lymphoma (FL) / Leukemia Acute Myeloid Leukemia (AML) / Leukemia or MDS in Aplasia / Leukemia, Acute / Leukemia, Plasma Cell / Leukemia, Prolymphocytic / Lymphoma, Hodgkins / Lymphoma, Large-Cell / Lymphoplasmacytic Lymphoma / Mantle Cell Lymphoma (MCL) / Marginal Zone B-Cell Lymphoma / MRD Positive Leukemia / Multiple Myeloma (MM) / Myelodysplastic Syndrome / Myeloproliferative Neoplasms/Myelofibrosis / Myeloproliferative Syndromes / Natural Killer Cell Malignancies / Recurrent Cutaneous T-cell lymphoma / Relapsed Chronic Lymphocytic Leukemia / Relapsed Multiple Myeloma / Relapsed Small Lymphocytic Lymphoma / Small Lymphocytic Lymphoma1
2RecruitingTreatmentAmyotrophic Lateral Sclerosis (ALS)1
2RecruitingTreatmentAngiofibroma of Face / Tuberous Sclerosis1
2RecruitingTreatmentArteriovenous Malformations / Arteriovenous Malformations (Stage II to IV)1
2RecruitingTreatmentBannayan Syndrome / Bannayan Zonana Syndrome / Cowden's Disease / Polyposis / PTEN gene mutation / PTEN Hamartoma Syndrome / PTEN Hamartoma Tumor Syndrome1
2RecruitingTreatmentBeta-Thalassemia1
2RecruitingTreatmentAcute, recurrent Myeloid Leukemia / Blasts 10 Percent or More of Bone Marrow Nucleated Cells / Chronic Myelomonocytic Leukemia-2 / High Grade Malignant Neoplasm / Myelodysplastic Syndrome / Myelodysplastic Syndrome With Excess Blasts-2 / Myeloid Neoplasm / Previously Treated Myelodysplastic Syndromes / Recurrent Adult Acute Myeloid Leukemia / Recurrent Childhood Acute Myeloid Leukemia / Refractory Acute Myeloid Leukemia1
2RecruitingTreatmentCastleman's Disease / Castleman's Disease, Multicentric1
2RecruitingTreatmentChondrosarcoma, Mesenchymal / Conventional Chondrosarcoma / Dedifferentiated Chondrosarcoma / Myxoid Liposarcoma1
2RecruitingTreatmentChorioretinitis / Panuveitis / Uveitis / Uveitis, Intermediate1
2RecruitingTreatmentEpidermolysis Bullosa Simplex / Epidermolysis Bullosa Simplex Kobner / Weber-Cockayne Syndrome1
2RecruitingTreatmentGraft Versus Host Disease (GVHD) / Sickle Cell Disorders / Thalassaemic disorders / Transplantation, Stem Cell1
2RecruitingTreatmentHHV-8 / Human Immunodeficiency Virus (HIV) / Lymphoproliferative Disorders / Malignancies1
2RecruitingTreatmentHigh Grade Recurrent Glioma and Newly Diagnosed Glioblastoma1
2RecruitingTreatmentIn-stent Stenosis / Peripheral Pulmonary Stenosis1
2RecruitingTreatmentKaposiform Hemangioendothelioma / Kasabach Merritt Phenomenon1
2RecruitingTreatmentLymphangioleiomyomatosis1
2RecruitingTreatmentLymphatic Malformations / Pediatric1
2RecruitingTreatmentLymphatic Malformations / Vascular Malformations1
2RecruitingTreatmentMalignancies1
2RecruitingTreatmentMalignant Peripheral Nerve Sheath Tumour (MPNST) / Neurofibromatosis 11
2RecruitingTreatmentMetastatic Medullary Thyroid Cancer1
2RecruitingTreatmentMultiple System Atrophy (MSA)1
2RecruitingTreatmentNeuroendocrine Tumors1
2RecruitingTreatmentPinealoblastoma1
2RecruitingTreatmentPlastic Bronchitis1
2RecruitingTreatmentPure Red Cell Aplasia / Red Blood Cell Disorder / Sickle Cell Disorders / Stem Cell Transplant Complications1
2RecruitingTreatmentRecurrent Neuroblastoma1
2RecruitingTreatmentSevere Aplastic Anemia (SAA)1
2RecruitingTreatmentSickle Cell Disorders3
2RecruitingTreatmentType 1 Insulin-Dependent Diabetes Mellitus2
2TerminatedPreventionAcute Leukemia in Remission / Leukemia, Myeloid, Accelerated-Phase / Leukemia, Myeloid, Chronic-Phase / Myelodysplastic Syndromes1
2TerminatedPreventionGraft Versus Host Disease (GVHD)1
2TerminatedSupportive CareChronic Graft Versus Host Disease / Oral Mucosal Disease Due to Graft-versus-host Disease1
2TerminatedSupportive CareChronic Myeloproliferative Disorders / Graft Versus Host Disease (GVHD) / Leukemias / Lymphoproliferative Disorders / Malignant Lymphomas / Multiple Myeloma and Plasma Cell Neoplasm / Myelodysplastic Syndromes / Myelodysplastic/Myeloproliferative Neoplasms1
2TerminatedTreatmentAge-Related Macular Degeneration (ARMD) / Subfoveal Choroidal Neovascularization1
2TerminatedTreatmentDry Macular Degeneration1
2TerminatedTreatmentEnd-Stage Renal Disease (ESRD)1
2TerminatedTreatmentHaematological Malignancies / Leukemias / Malignant Lymphomas / Myelodysplastic Syndrome1
2TerminatedTreatmentHematologic Diseases1
2TerminatedTreatmentMalignant Neoplasm of Pancreas1
2TerminatedTreatmentRefractory Multiple Myeloma / Stage I Multiple Myeloma / Stage II Multiple Myeloma / Stage III Multiple Myeloma1
2TerminatedTreatmentRelapsed / Recurrent Germ Cell Tumors1
2TerminatedTreatmentSarcomas1
2TerminatedTreatmentSickle Cell Disorders2
2TerminatedTreatmentSquamous Cell Skin Carcinoma1
2TerminatedTreatmentTransplant, Kidney1
2TerminatedTreatmentType 1 Insulin-Dependent Diabetes Mellitus1
2Unknown StatusTreatmentLymphangioleiomyomatosis / Tuberous Sclerosis1
2Unknown StatusTreatmentNonmalignant Neoplasm1
2Unknown StatusTreatmentTransplantation, Liver1
2WithdrawnPreventionHematopoietic Stem Cell Transplant (HSCT) / Severe Combined Immunodeficiency / Transplacental Maternal Engraftment1
2WithdrawnTreatmentCutaneous T-Cell Lymphoma (CTCL)1
2WithdrawnTreatmentType 1 Insulin-Dependent Diabetes Mellitus1
2, 3Active Not RecruitingTreatmentSturge-Weber Syndrome1
2, 3Active Not RecruitingTreatmentTransplantation, Kidney1
2, 3CompletedTreatmentChronic Graft Versus Host Disease1
2, 3CompletedTreatmentHepatocellular,Carcinoma1
2, 3CompletedTreatmentInclusion Body Myositis (IBM)1
2, 3Not Yet RecruitingTreatmentTransplantation, Liver1
2, 3RecruitingTreatmentCongenital Hypoplastic Anemia1
2, 3RecruitingTreatmentFacial Angiofibroma / Tuberous Sclerosis1
2, 3RecruitingTreatmentMalignant Neoplasm of Pancreas1
2, 3TerminatedTreatmentAutosomal Dominant Polycystic Kidney Disease (ADPKD)2
2, 3TerminatedTreatmentHigh Blood Pressure (Hypertension)1
2, 3Unknown StatusTreatmentPolycystic Kidney Diseases1
2, 3WithdrawnTreatmentProstate Cancer1
3Active Not RecruitingPreventionArteriovenous Fistulas / Chronic Renal Failure (CRF) / Complication of Hemodialysis / Complication of Renal Dialysis / End Stage Kidney Disease / End Stage Renal Disease (ESRD) / ESRD / Vascular Access Complications1
3Active Not RecruitingTreatmentAlpha Fetoprotein Increased / PRETEXT Stage 1 Hepatoblastoma / PRETEXT Stage 2 Hepatoblastoma / PRETEXT Stage 3 Hepatoblastoma / PRETEXT Stage 4 Hepatoblastoma1
3Active Not RecruitingTreatmentB-cell Childhood Acute Lymphoblastic Leukemia / Childhood Acute Lymphoblastic Leukemia in Remission / Graft Versus Host Disease (GVHD) / L1 Childhood Acute Lymphoblastic Leukemia / L2 Childhood Acute Lymphoblastic Leukemia / T-cell Childhood Acute Lymphoblastic Leukemia1
3Active Not RecruitingTreatmentType 1 Insulin-Dependent Diabetes Mellitus1
3CompletedNot AvailableGraft Versus Host Disease (GVHD) / Transplantation, Kidney1
3CompletedPreventionGraft Versus Host Disease (GVHD) / Survival1
3CompletedPreventionKidney Diseases1
3CompletedPreventionRenal Allograft Recipients1
3CompletedPreventionTransplant, Kidney1
3CompletedPreventionTransplantation, Renal1
3CompletedTreatmentAcute Lymphoblastic Leukaemias (ALL) / Aggressive Non-Hodgkin Lymphoma / Chronic Lymphocytic Leukaemia (CLL) / Chronic Lymphocytic Leukemia (CLL) - Refractory / Chronic, recurrent Lymphocytic Leukemia / Hematopoietic and Lymphoid Cell Neoplasm / Indolent Non-Hodgkin's Lymphomas / Leukemia Acute Myeloid Leukemia (AML) / Leukemia, Prolymphocytic / Lymphoma, Large B-Cell, Diffuse (DLBCL) / Mantle Cell Lymphoma (MCL) / Myelodysplastic Syndrome / Myeloproliferative Neoplasms / Recurrent Plasma Cell Myeloma / Refractory Chronic Myelogenous Leukemia, BCR-ABL1 Positive / Refractory Hodgkin Lymphoma / Small Lymphocytic Lymphoma / T-cell chronic lymphocytic leukaemia / Waldenström's Macroglobulinemia (WM)1
3CompletedTreatmentAutosomal Dominant Polycystic Kidney Disease (ADPKD)1
3CompletedTreatmentBirt-Hogg-Dubé Syndrome1
3CompletedTreatmentCardiovascular Abnormalities1
3CompletedTreatmentChronic Myeloid Leukemia (CML) / Leukemia, Lymphocytic, Acute / Leukemia, Myelocytic, Acute / Myelodysplastic Syndromes1
3CompletedTreatmentEnd Stage Liver Diseases / Impaired kidney function1
3CompletedTreatmentFacial Angiofibroma1
3CompletedTreatmentGraft Versus Host Disease (GVHD) / Renal Failure1
3CompletedTreatmentGraft Versus Host Disease (GVHD) / Transplantation, Kidney1
3CompletedTreatmentHepatocellular,Carcinoma1
3CompletedTreatmentLymphoma, Hodgkins / Non-Hodgkin's Lymphoma (NHL)1
3CompletedTreatmentNon-Infectious Uveitis of the Posterior Segment of the Eye1
3CompletedTreatmentSkin Cancers / Transplantation, Kidney1
3CompletedTreatmentTransplantation, Kidney1
3CompletedTreatmentTransplantation, Renal2
3CompletedTreatmentType 1 Insulin-Dependent Diabetes Mellitus2
3CompletedTreatmentUvea; Posterior, Disorder / Uveitis; Posterior, Disorder1
3RecruitingTreatmentBotryoid-Type Embryonal Rhabdomyosarcoma / Rhabdomyosarcoma, Alveolar / Rhabdomyosarcoma, Embryonal / Rhabdomyosarcomas / Sclerosing Rhabdomyosarcoma / Spindle Cell Rhabdomyosarcoma / Untreated Childhood Rhabdomyosarcoma1
3RecruitingTreatmentDyspnea / LAM / Lymphangioleiomyomatosis1
3RecruitingTreatmentPolycystic Kidney, Type 1 Autosomal Dominant Disease / Polycystic Kidney, Type 2 Autosomal Dominant Disease1
3RecruitingTreatmentVascular Malformations2
3TerminatedTreatmentAtherosclerosis / Renal Failure1
3TerminatedTreatmentEnd-Stage Renal Disease (ESRD) / Transplantation, Kidney1
3TerminatedTreatmentOral Lichen Planus1
3TerminatedTreatmentTransplant, Kidney1
3TerminatedTreatmentTransplantation, Liver1
3Unknown StatusPreventionHepatocellular,Carcinoma1
3Unknown StatusPreventionTransplantation, Kidney1
3Unknown StatusTreatmentDiabetic Nephropathies / Type 1 Insulin-Dependent Diabetes Mellitus1
3Unknown StatusTreatmentLymphangioleiomyomatosis1
3WithdrawnTreatmentBladder Cancers1
4Active Not RecruitingTreatmentConnective Tissue Diseases / Thrombocytopenia1
4Active Not RecruitingTreatmentLiver Diseases1
4Active Not RecruitingTreatmentPancreas Transplantation / Transplantation, Kidney1
4CompletedNot AvailableChronic Renal Failure (CRF) / Graft Versus Host Disease (GVHD) / Transplantation, Kidney1
4CompletedNot AvailableFunction of Renal Transplant1
4CompletedHealth Services ResearchCoronary Artery Restenosis / Coronary Heart Disease (CHD)1
4CompletedPreventionAcute Graft Rejection / Delayed Graft Function1
4CompletedPreventionHepatitis C Viral Infection1
4CompletedPreventionKidney Transplant Rejection1
4CompletedPreventionRejection, Transplant / Renal Allograft Recipients / Transplant, Kidney1
4CompletedPreventionRejection, Transplant / Renal Failure1
4CompletedPreventionSkin Neoplasms / Transplantation, Kidney1
4CompletedPreventionTransplantation, Kidney2
4CompletedTreatmentAdult Primary Kidney Transplantation1
4CompletedTreatmentAgeing / Rejection, Transplant / Renal Failure1
4CompletedTreatmentChronic Kidney Disease (CKD) / Transplantation, Kidney1
4CompletedTreatmentComparative Study / Immunosuppressive Agents1
4CompletedTreatmentCoronary Artery Restenosis1
4CompletedTreatmentDisorder Related to Renal Transplantation / Infections, Cytomegalovirus1
4CompletedTreatmentEnd-Stage Renal Disease (ESRD)1
4CompletedTreatmentGraft Versus Host Disease (GVHD) / Renal Failure2
4CompletedTreatmentHeart Transplantation / Renal Failure1
4CompletedTreatmentInflammatory Reaction1
4CompletedTreatmentKidney Diseases1
4CompletedTreatmentKidney Diseases / Renal Failure1
4CompletedTreatmentKidney Transplant Patients1
4CompletedTreatmentPrimary Renal Allograft1
4CompletedTreatmentRejection, Transplant / Renal Failure / Transplant, Kidney1
4CompletedTreatmentRejection, Transplant / Renal Failure / Transplantation, Kidney1
4CompletedTreatmentRenal Angiomyolipomas1
4CompletedTreatmentTransplant, Kidney3
4CompletedTreatmentTransplantation, Kidney6
4CompletedTreatmentTransplantation, Liver1
4CompletedTreatmentTransplantation, Renal3
4Not Yet RecruitingTreatmentFlu caused by Influenza1
4Not Yet RecruitingTreatmentParoxysmal Nocturnal Haemoglobinuria (PNH)1
4RecruitingPreventionInfections, Cytomegalovirus1
4RecruitingPreventionKidney Transplant Infection1
4RecruitingTreatmentBlue Rubber Bleb Nevus Syndrome / Venous Malformations1
4RecruitingTreatmentCrohn's Disease (CD)1
4RecruitingTreatmentHemangiomas / Kaposiform Hemangioendothelioma1
4RecruitingTreatmentPeutz-Jeghers Syndrome (PJS)1
4RecruitingTreatmentPure Red Cell Aplasia1
4RecruitingTreatmentRefractory Solid Tumors1
4RecruitingTreatmentTransplantation, Kidney1
4RecruitingTreatmentUterine Leiomyomas1
4TerminatedPreventionRenal Transplant Patients at High-risk for Skin Cancer1
4TerminatedPreventionSkin Cancers / Transplantation, Kidney1
4TerminatedTreatmentDelayed Graft Function / Slow Graft Function-defined at Creatinine >= 3.0 by Post-op Day 5 Without Requiring Dialysis / Transplant, Kidney1
4TerminatedTreatmentKidney Transplant Failure and Rejection1
4TerminatedTreatmentTransplantation, Kidney1
4Unknown StatusPreventionImmunosuppression / Transplantation, Kidney / Transplantations2
4Unknown StatusPreventionTransplantation, Kidney1
4Unknown StatusTreatmentChronic Allograft Nephropathy (CAN) / Glomerular Filtration Rate (GFR)1
4Unknown StatusTreatmentChronic Renal Failure (CRF) / Immunosuppression / Interstitial Fibrosis (IF) / Transplantations1
4Unknown StatusTreatmentEnd Stage Renal Disease (ESRD) / Transplantation, Kidney1
4Unknown StatusTreatmentKidney Diseases / Rejection, Transplant1
4Unknown StatusTreatmentTransplant, Kidney1
4Unknown StatusTreatmentTransplantation, Kidney3
4WithdrawnNot AvailableTransplantation, Kidney1
4WithdrawnTreatmentCCR5 / HIV Reservoir / Human Immunodeficiency Virus (HIV) / Transplant, Kidney1
4WithdrawnTreatmentKidney Graft Dysfunction1
4WithdrawnTreatmentLiving Donor Kidney Transplants Patients1
Not AvailableActive Not RecruitingNot AvailableLymphangioleiomyomatosis1
Not AvailableActive Not RecruitingDiagnosticAcute Rejection of Liver Transplant / Effects of Immunosuppressant Therapy1
Not AvailableActive Not RecruitingPreventionRenal Failure1
Not AvailableActive Not RecruitingTreatmentDiffuse Parenchymal Lung Diseases / Idiopathic Pulmonary Fibrosis (IPF) / Interstitial Lung Disease (ILD)1
Not AvailableAvailableNot AvailableMTOR Pathway Abberation / PEComa, Malignant / TSC1 / TSC21
Not AvailableCompletedNot AvailableAutosomal Dominant Polycystic Kidney Disease (ADPKD)1
Not AvailableCompletedNot AvailableImmune Thrombocytopenia1
Not AvailableCompletedNot AvailableImmune Thrombocytopenic Purpura ( ITP )1
Not AvailableCompletedNot AvailableIslet Transplantation / Type 1 Insulin-Dependent Diabetes Mellitus1
Not AvailableCompletedNot AvailableTransplantation, Kidney1
Not AvailableCompletedNot AvailableTransplantation, Liver1
Not AvailableCompletedNot AvailableTransplantation, Renal1
Not AvailableCompletedNot AvailableTransplantations1
Not AvailableCompletedPreventionAbsence; Kidney1
Not AvailableCompletedPreventionBK Nephropathy / BK Viremia1
Not AvailableCompletedPreventionTransplant, Kidney2
Not AvailableCompletedScreeningTransplantation, Kidney1
Not AvailableCompletedSupportive CareChronic Myeloproliferative Disorders / Graft Versus Host Disease (GVHD) / Leukemias / Malignant Lymphomas / Multiple Myeloma and Plasma Cell Neoplasm / Myelodysplastic Syndromes / Myelodysplastic/Myeloproliferative Neoplasms1
Not AvailableCompletedTreatmentEnd-Stage Renal Disease (ESRD)1
Not AvailableCompletedTreatmentLeukemia Acute Myeloid Leukemia (AML)1
Not AvailableCompletedTreatmentPosttraumatic Stress Disorders1
Not AvailableCompletedTreatmentTumors, Solid1
Not AvailableNo Longer AvailableNot AvailableType 1 Insulin-Dependent Diabetes Mellitus2
Not AvailableNot Yet RecruitingTreatmentCoronary Artery Disease1
Not AvailableRecruitingTreatmentHaemoglobinopathies congenital / Sickle Cell Disorders1
Not AvailableRecruitingTreatmentVascular Anomalies1
Not AvailableTerminatedNot AvailableIslet Transplantation / Type 1 Insulin-Dependent Diabetes Mellitus1
Not AvailableTerminatedNot AvailableTransplantation, Renal1
Not AvailableTerminatedTreatmentLymphoblastic lymphoma (Precursor T-lymphoblastic lymphoma/leukemia) refractory / Recurrent Adult Lymphoblastic Lymphoma / Refractory Acute Lymphoblastic Leukemia / Relapsed Lymphoblastic Leukemia1
Not AvailableTerminatedTreatmentPolycystic Liver Disease (PLD)1
Not AvailableTerminatedTreatmentPort-wine Stains (PWS)1
Not AvailableUnknown StatusNot AvailableAcute Rejection of Renal Transplant1
Not AvailableWithdrawnPreventionCardiac Allograft Vasculopathy1

Pharmacoeconomics

Manufacturers
  • Wyeth pharmaceuticals inc
Packagers
  • Cardinal Health
  • Hangzhou Zhongmei Huadong Pharmaceutical Co. Ltd.
  • Lake Erie Medical and Surgical Supply
  • Patheon Inc.
  • Poli Industria Chimica SPA
  • Wyeth Pharmaceuticals
Dosage forms
FormRouteStrength
SolutionOral
TabletOral
SolutionOral1 mg/ml
SolutionOral1.0 mg
TabletOral1.0 mg
TabletOral2 mg
TabletOral5 mg
Tablet, coatedOral0.5 mg
Tablet, coatedOral1 mg
Tablet, coatedOral2 mg
Tablet, sugar coatedOral1 mg/1
Tablet, sugar coatedOral2 mg/1
SolutionOral1 mg/1mL
TabletOral1 mg/1
TabletOral2 mg/1
Tablet, film coatedOral0.5 mg/1
Tablet, film coatedOral1 mg/1
Tablet, film coatedOral2 mg/1
Tablet, sugar coatedOral0.5 mg/1
Prices
Unit descriptionCostUnit
Rapamune 2 mg tablet20.59USD tablet
Rapamune 1 mg/ml Solution12.19USD ml
Rapamune 1 mg tablet11.95USD tablet
Rapamune 0.5 mg tablet5.86USD tablet
DrugBank does not sell nor buy drugs. Pricing information is supplied for informational purposes only.
Patents
Patent NumberPediatric ExtensionApprovedExpires (estimated)
US5212155No1993-05-182010-05-18Us
CA2293793No2006-07-112018-06-11Canada
CA2103571No2003-04-292012-02-21Canada
US5989591Yes1999-11-232018-09-11Us
Additional Data Available
  • Filed On
    Filed On

    The date on which a patent was filed with the relevant government.

    Learn more

Properties

State
Solid
Experimental Properties
PropertyValueSource
logP4.3Not Available
Predicted Properties
PropertyValueSource
Water Solubility0.00173 mg/mLALOGPS
logP4.85ALOGPS
logP7.45ChemAxon
logS-5.7ALOGPS
pKa (Strongest Acidic)9.96ChemAxon
pKa (Strongest Basic)-3ChemAxon
Physiological Charge0ChemAxon
Hydrogen Acceptor Count12ChemAxon
Hydrogen Donor Count3ChemAxon
Polar Surface Area195.43 Å2ChemAxon
Rotatable Bond Count6ChemAxon
Refractivity250.66 m3·mol-1ChemAxon
Polarizability101.74 Å3ChemAxon
Number of Rings4ChemAxon
Bioavailability0ChemAxon
Rule of FiveNoChemAxon
Ghose FilterNoChemAxon
Veber's RuleNoChemAxon
MDDR-like RuleYesChemAxon
Predicted ADMET features
PropertyValueProbability
Human Intestinal Absorption-0.7841
Blood Brain Barrier-0.9599
Caco-2 permeable-0.6341
P-glycoprotein substrateSubstrate0.8052
P-glycoprotein inhibitor IInhibitor0.8564
P-glycoprotein inhibitor IIInhibitor0.8021
Renal organic cation transporterNon-inhibitor0.8116
CYP450 2C9 substrateNon-substrate0.878
CYP450 2D6 substrateNon-substrate0.9138
CYP450 3A4 substrateSubstrate0.7776
CYP450 1A2 substrateNon-inhibitor0.9007
CYP450 2C9 inhibitorNon-inhibitor0.9125
CYP450 2D6 inhibitorNon-inhibitor0.9414
CYP450 2C19 inhibitorNon-inhibitor0.9158
CYP450 3A4 inhibitorNon-inhibitor0.9333
CYP450 inhibitory promiscuityLow CYP Inhibitory Promiscuity0.9742
Ames testNon AMES toxic0.6617
CarcinogenicityNon-carcinogens0.9546
BiodegradationNot ready biodegradable0.9593
Rat acute toxicity2.8689 LD50, mol/kg Not applicable
hERG inhibition (predictor I)Weak inhibitor0.9831
hERG inhibition (predictor II)Non-inhibitor0.8443
ADMET data is predicted using admetSAR, a free tool for evaluating chemical ADMET properties. (23092397)

Spectra

Mass Spec (NIST)
Not Available
Spectra
Not Available

Taxonomy

Description
This compound belongs to the class of organic compounds known as macrolide lactams. These are cyclic polyketides containing both a cyclic amide and a cyclic ester group.
Kingdom
Organic compounds
Super Class
Phenylpropanoids and polyketides
Class
Macrolide lactams
Sub Class
Not Available
Direct Parent
Macrolide lactams
Alternative Parents
Alpha amino acid esters / Macrolides and analogues / Cyclohexanols / Piperidines / Oxanes / Tertiary carboxylic acid amides / Carboxylic acid esters / Cyclic alcohols and derivatives / Cyclic ketones / Hemiacetals
show 10 more
Substituents
Macrolide lactam / Alpha-amino acid ester / Macrolide / Alpha-amino acid or derivatives / Cyclohexanol / Oxane / Piperidine / Cyclic alcohol / Tertiary carboxylic acid amide / Cyclic ketone
show 24 more
Molecular Framework
Aliphatic heteropolycyclic compounds
External Descriptors
cyclic ketone, antibiotic antifungal drug, organic heterotricyclic compound, secondary alcohol, ether, lactam, macrolide, cyclic acetal (CHEBI:9168) / Plyenes (C07909)

Targets

Kind
Protein
Organism
Humans
Pharmacological action
Yes
Actions
Inhibitor
General Function
Tfiiic-class transcription factor binding
Specific Function
Serine/threonine protein kinase which is a central regulator of cellular metabolism, growth and survival in response to hormones, growth factors, nutrients, energy and stress signals. MTOR directly...
Gene Name
MTOR
Uniprot ID
P42345
Uniprot Name
Serine/threonine-protein kinase mTOR
Molecular Weight
288889.05 Da
References
  1. Dowling RJ, Topisirovic I, Fonseca BD, Sonenberg N: Dissecting the role of mTOR: lessons from mTOR inhibitors. Biochim Biophys Acta. 2010 Mar;1804(3):433-9. doi: 10.1016/j.bbapap.2009.12.001. Epub 2009 Dec 11. [PubMed:20005306]
  2. Shuuin T, Karashima H: [Mammalian target of rapamycin, its mode of action and clinical response in metastatic clear cell carcinoma]. Gan To Kagaku Ryoho. 2009 Jul;36(7):1076-9. [PubMed:19620795]
  3. Sehgal SN: Sirolimus: its discovery, biological properties, and mechanism of action. Transplant Proc. 2003 May;35(3 Suppl):7S-14S. [PubMed:12742462]
  4. Chen X, Ji ZL, Chen YZ: TTD: Therapeutic Target Database. Nucleic Acids Res. 2002 Jan 1;30(1):412-5. [PubMed:11752352]
Kind
Protein
Organism
Humans
Pharmacological action
Yes
Actions
Other
General Function
Type i transforming growth factor beta receptor binding
Specific Function
Keeps in an inactive conformation TGFBR1, the TGF-beta type I serine/threonine kinase receptor, preventing TGF-beta receptor activation in absence of ligand. Recruites SMAD7 to ACVR1B which prevent...
Gene Name
FKBP1A
Uniprot ID
P62942
Uniprot Name
Peptidyl-prolyl cis-trans isomerase FKBP1A
Molecular Weight
11950.665 Da
References
  1. Chen X, Ji ZL, Chen YZ: TTD: Therapeutic Target Database. Nucleic Acids Res. 2002 Jan 1;30(1):412-5. [PubMed:11752352]
  2. Sehgal SN: Sirolimus: its discovery, biological properties, and mechanism of action. Transplant Proc. 2003 May;35(3 Suppl):7S-14S. [PubMed:12742462]
Kind
Protein
Organism
Humans
Pharmacological action
Yes
Actions
Other/unknown
General Function
Ligand-dependent nuclear receptor transcription coactivator activity
Specific Function
Plays an important role in the regulation of cell survival, cell division, angiogenesis, cell differentiation and cell migration. Functions as potent mitogen in vitro.
Gene Name
FGF2
Uniprot ID
P09038
Uniprot Name
Fibroblast growth factor 2
Molecular Weight
30769.715 Da
References
  1. Sehgal SN: Sirolimus: its discovery, biological properties, and mechanism of action. Transplant Proc. 2003 May;35(3 Suppl):7S-14S. [PubMed:12742462]

Enzymes

Kind
Protein
Organism
Humans
Pharmacological action
Unknown
Actions
Substrate
General Function
Vitamin d3 25-hydroxylase activity
Specific Function
Cytochromes P450 are a group of heme-thiolate monooxygenases. In liver microsomes, this enzyme is involved in an NADPH-dependent electron transport pathway. It performs a variety of oxidation react...
Gene Name
CYP3A4
Uniprot ID
P08684
Uniprot Name
Cytochrome P450 3A4
Molecular Weight
57342.67 Da
References
  1. Ekins S, Bravi G, Wikel JH, Wrighton SA: Three-dimensional-quantitative structure activity relationship analysis of cytochrome P-450 3A4 substrates. J Pharmacol Exp Ther. 1999 Oct;291(1):424-33. [PubMed:10490933]
  2. Sadaba B, Campanero MA, Quetglas EG, Azanza JR: Clinical relevance of sirolimus drug interactions in transplant patients. Transplant Proc. 2004 Dec;36(10):3226-8. doi: 10.1016/j.transproceed.2004.10.056. [PubMed:15686733]
  3. Flockhart Table of Drug Interactions [Link]
Kind
Protein
Organism
Humans
Pharmacological action
Unknown
Actions
Substrate
General Function
Oxygen binding
Specific Function
Cytochromes P450 are a group of heme-thiolate monooxygenases. In liver microsomes, this enzyme is involved in an NADPH-dependent electron transport pathway. It oxidizes a variety of structurally un...
Gene Name
CYP3A5
Uniprot ID
P20815
Uniprot Name
Cytochrome P450 3A5
Molecular Weight
57108.065 Da
References
  1. Flockhart Table of Drug Interactions [Link]
Kind
Protein
Organism
Humans
Pharmacological action
Unknown
Actions
Substrate
General Function
Oxygen binding
Specific Function
Cytochromes P450 are a group of heme-thiolate monooxygenases. In liver microsomes, this enzyme is involved in an NADPH-dependent electron transport pathway. It oxidizes a variety of structurally un...
Gene Name
CYP3A7
Uniprot ID
P24462
Uniprot Name
Cytochrome P450 3A7
Molecular Weight
57525.03 Da
References
  1. Flockhart Table of Drug Interactions [Link]

Transporters

Kind
Protein
Organism
Humans
Pharmacological action
Unknown
Actions
Inhibitor
Inducer
General Function
Xenobiotic-transporting atpase activity
Specific Function
Energy-dependent efflux pump responsible for decreased drug accumulation in multidrug-resistant cells.
Gene Name
ABCB1
Uniprot ID
P08183
Uniprot Name
Multidrug resistance protein 1
Molecular Weight
141477.255 Da
References
  1. Schuetz EG, Beck WT, Schuetz JD: Modulators and substrates of P-glycoprotein and cytochrome P4503A coordinately up-regulate these proteins in human colon carcinoma cells. Mol Pharmacol. 1996 Feb;49(2):311-8. [PubMed:8632764]
  2. Wacher VJ, Silverman JA, Wong S, Tran-Tau P, Chan AO, Chai A, Yu XQ, O'Mahony D, Ramtoola Z: Sirolimus oral absorption in rats is increased by ketoconazole but is not affected by D-alpha-tocopheryl poly(ethylene glycol 1000) succinate. J Pharmacol Exp Ther. 2002 Oct;303(1):308-13. [PubMed:12235265]
  3. Arceci RJ, Stieglitz K, Bierer BE: Immunosuppressants FK506 and rapamycin function as reversal agents of the multidrug resistance phenotype. Blood. 1992 Sep 15;80(6):1528-36. [PubMed:1381629]
  4. Nagy H, Goda K, Fenyvesi F, Bacso Z, Szilasi M, Kappelmayer J, Lustyik G, Cianfriglia M, Szabo G Jr: Distinct groups of multidrug resistance modulating agents are distinguished by competition of P-glycoprotein-specific antibodies. Biochem Biophys Res Commun. 2004 Mar 19;315(4):942-9. [PubMed:14985103]
Kind
Protein
Organism
Humans
Pharmacological action
Unknown
Actions
Inhibitor
General Function
Sodium-independent organic anion transmembrane transporter activity
Specific Function
Mediates the Na(+)-independent uptake of organic anions such as pravastatin, taurocholate, methotrexate, dehydroepiandrosterone sulfate, 17-beta-glucuronosyl estradiol, estrone sulfate, prostagland...
Gene Name
SLCO1B1
Uniprot ID
Q9Y6L6
Uniprot Name
Solute carrier organic anion transporter family member 1B1
Molecular Weight
76447.99 Da
References
  1. Fehrenbach T, Cui Y, Faulstich H, Keppler D: Characterization of the transport of the bicyclic peptide phalloidin by human hepatic transport proteins. Naunyn Schmiedebergs Arch Pharmacol. 2003 Nov;368(5):415-20. Epub 2003 Oct 3. [PubMed:14530907]
Kind
Protein
Organism
Humans
Pharmacological action
Unknown
General Function
Monovalent cation:proton antiporter activity
Specific Function
Solute transporter for tetraethylammonium (TEA), 1-methyl-4-phenylpyridinium (MPP), cimetidine, N-methylnicotinamide (NMN), metformin, creatinine, guanidine, procainamide, topotecan, estrone sulfat...
Gene Name
SLC47A1
Uniprot ID
Q96FL8
Uniprot Name
Multidrug and toxin extrusion protein 1
Molecular Weight
61921.585 Da
References
  1. Meyer zu Schwabedissen HE, Verstuyft C, Kroemer HK, Becquemont L, Kim RB: Human multidrug and toxin extrusion 1 (MATE1/SLC47A1) transporter: functional characterization, interaction with OCT2 (SLC22A2), and single nucleotide polymorphisms. Am J Physiol Renal Physiol. 2010 Apr;298(4):F997-F1005. doi: 10.1152/ajprenal.00431.2009. Epub 2010 Jan 6. [PubMed:20053795]

Drug created on June 13, 2005 07:24 / Updated on December 06, 2019 11:51