Rapamune

(Sirolimus)

ApprovedSmall molecule

Sirolimus is used to prevent the body's immune system from rejecting transplanted kidneys. It's also used to treat a rare type of cancer called lymphangioleiomyomatosis (LAM).[2] Rapamune is currently approved and in use across 184 indications[4].[1]

Rapamune is one of 2520 small molecule programs targeting Angiofibromas. It is currently in Approved.

184

Indications

420

Total Trials

Active Trials

2,519

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[4][1]

Indication

AngiofibromasActive, not recruiting

PecomaApproved

Systemic Lupus ErythematosusActive, not recruiting

LymphangiectasisActive, not recruiting

Choroidal NeovascularizationApproved

HyperinsulinismActive, not recruiting

MetastasisApproved

Peripheral Arterial StenosisActive, not recruiting

AngiofibromaActive, not recruiting

Oral Lichen PlanusApproved

AngiofibromasApproved

Active, not recruiting

PecomaApproved

Approved

Systemic Lupus ErythematosusPhase 3

Active, not recruiting

LymphangiectasisApproved

Active, not recruiting

Choroidal NeovascularizationApproved

Approved

HyperinsulinismPhase 1

Active, not recruiting

MetastasisApproved

Approved

Peripheral Arterial StenosisPreclinical

Active, not recruiting

AngiofibromaApproved

Active, not recruiting

Oral Lichen PlanusApproved

Approved

Showing 10 of 184 indications

Competition

Competitive Landscape

2828 competitors in Breast Cancer

#DrugCompanyPhaseTrials

Rapamune

PPrism Pharmaceuticals

Approved

2 2

Halaven

Eisai

Preclinical

38 3

Gemzar

GGynecologic Oncology Group

Preclinical

22 4

Voltaren

Novartis

Preclinical

1 5

Lupron

EEastern Cooperative Oncology Group

Preclinical

5 6

MS Contin

HHikma Pharmaceuticals LLC

Preclinical

3 7

Systemic Therapy

Bristol-Myers Squibb

Preclinical

1 8

Neoadjuvant Therapy

SSun Yat-sen University

Preclinical

3 9

Fentanyl Citrate

IINSYS Therapeutics Inc

Preclinical

2 10

Vit D

PPost Graduate Institute of Medical Education and Research, Chandigarh

Preclinical

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2026

Phase 1Active, not recruiting

Advanced Cancer

NCT01087554

Phase 1/2Active, not recruiting

Sickle Cell Disease

NCT03077542

Q4 2026

Phase 2Recruiting

Aneurysm

NCT04141020

Phase 1Active, not recruiting

Sickle Cell Disease

NCT05384756

Phase 2Recruiting

Thyroid Cancer

NCT03099356

Phase 2/3Recruiting

Lymphatic Malformation

NCT06673290

Clinical

Trials by Indication[1]

Kidney Transplant20 trials

Study Evaluating the Effect of Sirolimus on Non-Melanoma Skin Cancer in Kidney Transplant Recipients

Completed· Pfizer logo

Pfizer·Phase 4·NCT00129961·86 enrolled

Sirolimus vs Mycophenolate With Tacrolimus in Simultaneous Pancreas and Kidney Transplantation

Completed·IInstitute for Clinical and Experimental Medicine·Phase 4·NCT03582878·238 enrolled

Differentiating Everolimus Versus Sirolimus in Combination With Calcineurin Inhibitors in Kidney Transplant Patients

Withdrawn·UUniversity of Colorado, Denver·Phase 4·NCT02062892

Show 17 more trials

Pediatric Kidney Transplant Study of Sirolimus, Mycophenolate Mofetil, and Corticosteroids vs Calcineurin Inhibitor Based Immunosuppression

Completed·PPediatric Nephrology of Alabama·N/A·NCT00555373·52 enrolled

Safety of Immunosuppression Minimization in Children and Adolescents After Kidney Transplantation

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1·NCT00768729·7 enrolled

Sirolimus in Kidney Transplant Patients With Squamous Cell Skin Carcinoma

Terminated·UUniversity of Florida·Phase 2·NCT01764607·1 enrolled

Safety and Effectiveness of Low Molecular Weight Sulfated Dextran in Islet Transplantation After Kidney Transplant

Withdrawn·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT00790439

Sirolimus Associated With Tacrolimus at Low Doses in Elderly Kidney Transplant Patients

Completed·AAssociação Médico Espírita de Botucatu·Phase 4·NCT02683291·48 enrolled

The Comparison of Tacrolimus and Sirolimus Immunosuppression Based Drug Regimens in Kidney Transplant Recipients

Completed·MMayo Clinic·Phase 4·NCT00275535·165 enrolled

Immunotherapy in Combination With Prednisone and Sirolimus for Kidney Transplant Recipients With Unresectable or Metastatic Skin Cancer

Recruiting·NNational Cancer Institute (NCI)·Phase 1/2·NCT05896839·16 enrolled

Treatment of Children With Kidney Transplants by Injection of CD4+CD25+FoxP3+ T Cells to Prevent Organ Rejection

Unknown status·PPirogov Russian National Research Medical University·Phase 1/2·NCT01446484·30 enrolled

Belatacept to Prevent Organ Rejection in Kidney Transplant Patients

Terminated·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT00346151·5 enrolled

Kidney Spare the Nephron (STN) Study - A Study of CellCept (Mycophenolate Mofetil) and Rapamune (Sirolimus) in Kidney Transplant Recipients

Completed· Roche logo

Roche·Phase 4·NCT00121810·305 enrolled

Study to Compare the Safety and Efficacy of Sirolimus (Rapamune) to Tacrolimus (Advagraf) Associated to Mycophenolate Mofetil (CellCept) Between 12 and 36 Months After Kidney Transplantation

Completed·UUniversity Hospital, Rouen·Phase 3·NCT00811915·65 enrolled

Study Evaluating Sirolimus in the Treatment of Kidney Transplant

Completed· Pfizer logo

Pfizer·Phase 4·NCT00282217·100 enrolled

Calcineurin Inhibitor Avoidance With Thymoglobulin and Sirolimus in Kidney Transplantation

Completed·VVanderbilt University·Phase 4·NCT00305396·80 enrolled

A Study Looking at Kidney Function in Kidney Transplant Recipients Who Are Taking Anti-rejection Medication Including Tacrolimus and With or Without Sirolimus.

Completed· Astellas logo

Astellas·Phase 4·NCT01363752·853 enrolled

Combination Drug Therapy Followed by Single Drug Steroid Free Therapy to Prevent Organ Rejection in Kidney Transplantation

Completed·NNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)·Phase 2·NCT00076570·31 enrolled

The Comparison of Three Different Immunosuppressant Regimens in Kidney Transplant Recipients.

Completed·MMayo Clinic·Phase 4·NCT00275522·16 enrolled

Improved Induction and Maintenance Immunosuppression in Kidney Transplantation

Completed·UUniversity of Nebraska·Phase 4·NCT00556933·180 enrolled

Sickle Cell Disease9 trials

Siplizumab for Sickle Cell Disease Transplant

Terminated·—·Phase 1/2·NCT06078696·1 enrolled

TMLI and Alemtuzumab for Treatment of Sickle Cell Disease

Active, not recruiting·—·Phase 1·NCT05384756·2 enrolled

Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia

Active, not recruiting·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 1/2·NCT05357482·40 enrolled

Show 6 more trials

Abatacept for GVHD Prophylaxis After Hematopoietic Stem Cell Transplantation for Pediatric Sickle Cell Disease

Completed·MMonica Bhatia·Phase 1·NCT02867800·24 enrolled

Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure

Active, not recruiting·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 2·NCT02105766·56 enrolled

Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease

Active, not recruiting·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 1/2·NCT03077542·57 enrolled

131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD)

Recruiting·—·Phase 1·NCT07015684·24 enrolled

Nonmyeloablative Conditioning for Mismatched Hematopoietic Stem Cell Transplantation for Severe Sickle Cell Disease

Terminated·WWashington University School of Medicine·Phase 1·NCT02678143·1 enrolled

Stem Cell Transplant in Patients With Severe Sickle Cell Disease

Terminated·KKathleen Dorritie·Phase 1·NCT03421756·1 enrolled

Acute Graft Versus Host Disease5 trials

Tacrolimus, Sirolimus and Ustekinumab vs. Tacrolimus and Sirolimus for the Prevention of Acute Graft-Versus-Host Disease

Completed·HH. Lee Moffitt Cancer Center and Research Institute·Phase 2·NCT01713400·54 enrolled

Panobinostat (LBH589): Acute Graft Versus Host Disease (aGVHD) Prevention

Completed·HH. Lee Moffitt Cancer Center and Research Institute·Phase 2·NCT02588339·42 enrolled

Thymoglobulin, Sirolimus and Mycophenolate Mofetil for Prevention of Acute Graft-Versus-Host Disease (GVHD)

Terminated·MM.D. Anderson Cancer Center·Phase 2·NCT00506948·13 enrolled

Show 2 more trials

Tacrolimus and Mycophenolate Mofetil With or Without Sirolimus in Preventing Acute Graft-Versus-Host Disease in Patients Who Are Undergoing Donor Stem Cell Transplant for Hematologic Cancer

Completed·FFred Hutchinson Cancer Center·Phase 2·NCT00105001·210 enrolled

Sirolimus, Tacrolimus, and Methotrexate in Preventing Acute Graft-Versus-Host Disease in Patients With Hematologic Cancer Who Are Undergoing Donor Stem Cell Transplantation

Completed·FFred Hutchinson Cancer Center·Phase 1/2·NCT00089037

Efficacy and Safety5 trials

Efficacy and Safety of Sirolimus to Vascular Anomalies

Completed·WWest China Hospital·N/A·NCT03583307·126 enrolled

Efficacy and Safety of Sirolimus in Combination With Tacrolimus

Unknown status·UUniversity Hospital Muenster·Phase 3·NCT00141804·190 enrolled

Efficacy and Safety of Sirolimus in Vascular Anomalies That Are Refractory to Standard Care

Recruiting·CCliniques universitaires Saint-Luc- Université Catholique de Louvain·Phase 3·NCT02638389·250 enrolled

Show 2 more trials

Study to Evaluate the Efficacy and Safety of Sirolimus in Subjects With Metastatic, Mismatch Repair Deficient Solid Tumors After Immunotherapy

Terminated·—·Phase 2·NCT04393454·6 enrolled

Efficacy and Safety of Sirolimus in LAM

Completed·UUniversity of Cincinnati·Phase 3·NCT00414648·89 enrolled

Non-Small Cell Lung Cancer5 trials

Sirolimus and Auranofin in Treating Patients With Advanced or Recurrent Non-Small Cell Lung Cancer or Small Cell Lung Cancer

Completed·MMayo Clinic·Phase 1/2·NCT01737502·29 enrolled

Sirolimus and Pemetrexed to Treat Non-Small Cell Lung Cancer

Terminated·NNational Cancer Institute (NCI)·Phase 1/2·NCT00923273·42 enrolled

Gefitinib and Sirolimus in Treating Patients With Recurrent or Refractory Stage IIIB or Stage IV Non-Small Cell Lung Cancer

Withdrawn·JJonsson Comprehensive Cancer Center·Phase 1/2·NCT00098462

Show 2 more trials

Auranofin and Sirolimus in Treating Patients With Advanced Solid Tumors or Recurrent Non-Small Cell Lung Cancer

Withdrawn·MMayo Clinic·Phase 1·NCT02126527

Trial of Continuous Once Daily Oral Treatment Using BIBW 2992 (Afatinib) Plus Sirolimus (Rapamune®) in Patients With Non-small Cell Lung Cancer Harbouring an EGFR Mutation and/or Disease Progression Following Prior Erlotinib or Gefitinib

Completed· Boehringer Ingelheim logo

Boehringer Ingelheim·Phase 1·NCT00993499·44 enrolled

115 more indications

Acute Lymphoblastic Leukemia3 trials

225Ac-DOTA-Anti-CD38 Daratumumab Monoclonal Antibody With Fludarabine, Melphalan and Total Marrow and Lymphoid Irradiation as Conditioning Treatment for Donor Stem Cell Transplant in Patients With High-Risk Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia and Myelodysplastic Syndrome

Recruiting·—·Phase 1·NCT06287944·15 enrolled

Combination Chemotherapy With or Without Donor Stem Cell Transplant in Treating Patients With Acute Lymphoblastic Leukemia

Active, not recruiting·NNational Cancer Institute (NCI)·Phase 2·NCT00792948·97 enrolled

Tacrolimus and Methotrexate With or Without Sirolimus in Preventing Graft-Versus-Host Disease in Young Patients Undergoing Donor Stem Cell Transplant for Acute Lymphoblastic Leukemia in Complete Remission

Completed·CChildren's Oncology Group·Phase 3·NCT00382109·146 enrolled

Aplastic Anemia3 trials

Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia

Unknown status·OOffice of Rare Diseases (ORD)·Phase 1/2·NCT00319878·52 enrolled

Efficacy and Safety of Sirolimus With or Without Cyclosporin A in Chinese Patients With Aplastic Anemia Refractory/Intolerant to Cyclosporin A

Recruiting·PPeking Union Medical College Hospital·Phase 2·NCT06802055·40 enrolled

Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia

Recruiting·FFred Hutchinson Cancer Center·Phase 2·NCT06752694·20 enrolled

Treatment3 trials

A Study to Compare Vincristine to Sirolimus for Treatment of High Risk Vascular Tumors

Terminated·—·Phase 2·NCT02110069·4 enrolled

Percutaneous Administration of Sirolimus in the Treatment of Superficial Complicated Vascular Anomalies

Recruiting·—·Phase 4·NCT04921722·75 enrolled

Safety and Durability of Sirolimus for Treatment of LAM

Recruiting·UUniversity of Cincinnati··NCT02432560·600 enrolled

Type 1 Diabetes3 trials

Peritransplant Deoxyspergualin in Islet Transplantation in Type 1 Diabetes

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT00434850·14 enrolled

Raptiva and Sirolimus in Islet Transplantation for Type 1 Diabetes

Terminated·UUniversity of Minnesota·Phase 1/2·NCT00672204·23 enrolled

Islet Transplantation in Type 1 Diabetes

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 3·NCT00434811·48 enrolled

Vascular Malformation3 trials

Efficacy and Safety of the Mammalian Target of Rapamycin (mTor Rapamycin) Inhibitor in Vascular Malformations

Completed·CCliniques universitaires Saint-Luc- Université Catholique de Louvain·Phase 3·NCT01811667·19 enrolled

Treatment of Congenital Vascular Malformations Using Sirolimus: Improving Quality of Life

Unknown status·RRadboud University Medical Center·Phase 3·NCT03987152·75 enrolled

Role of Sirolimus in Treatment of Microcystic , Mixed Lymphatic and Vascular Malformations

Unknown status·SSohag University··NCT06160739·10 enrolled

Renal Transplant Recipients3 trials

Study To Evaluate Pharmacokinetics Of Sirolimus In Stable Renal Transplant Recipients

Completed· Pfizer logo

Pfizer·Phase 1·NCT01236378·24 enrolled

Pilot Study for HLA Identical Living Donor Renal Transplant Recipients

Completed·UUniversity of Cincinnati·Phase 4·NCT00352092·20 enrolled

Study Comparing Efficacy and Safety of Mycophenolate Mofetil (Cellcept) With Delayed Introduction of Sirolimus and Discontinuation of Cyclosporine, With Those of Mycophenolate Mofetil and Long Term Continuation of Cyclosporine in Renal Transplant Recipients

Completed· Roche logo

Roche·Phase 3·NCT02686619·237 enrolled

Bone Marrow Transplantation3 trials

Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503)

Completed·MMedical College of Wisconsin·Phase 2·NCT02766465·138 enrolled

Phase I/II Study to Reduce Post-transplantation Cyclophosphamide Dosing for Older or Unfit Patients Undergoing Bone Marrow Transplantation for Hematologic Malignancies

Recruiting·NNational Cancer Institute (NCI)·Phase 1/2·NCT04959175·320 enrolled

HLA-Mismatched Unrelated Donor Bone Marrow Transplantation With Post-Transplantation Cyclophosphamide

Completed·CCenter for International Blood and Marrow Transplant Research·Phase 2·NCT02793544·80 enrolled

Kaposiform Hemangioendothelioma3 trials

Different Doses of Sirolimus for the Maintenance Treatment of Kaposiform Hemangioendothelioma

Unknown status·WWest China Hospital·Phase 2·NCT05324384·30 enrolled

Guided Discontinuation Versus Maintenance Treatment of Sirolimus in Pediatric Patients With Kaposiform Hemangioendothelioma

Completed·—·Phase 4·NCT04448873·30 enrolled

Safety and Efficacy of Low-dose Sirolimus to Kaposiform Hemangioendothelioma

Completed·—·Phase 4·NCT04077515·92 enrolled

Graft Versus Host Disease3 trials

Prevention of Graft-Versus-Host Disease in Patients With Hematologic Malignancies Who Are Receiving a Bone Marrow Transplant

Withdrawn·FFred Hutchinson Cancer Center·Phase 1/2·NCT00002790

Sirolimus, Tacrolimus, and Antithymocyte Globulin in Preventing Graft-Versus-Host Disease in Patients Undergoing a Donor Stem Cell Transplant For Hematological Cancer

Completed·—·Phase 2·NCT00589563·32 enrolled

Graft-Versus-Host Disease Prophylaxis in Treating Patients With Hematologic Malignancies Undergoing Unrelated Donor Peripheral Blood Stem Cell Transplant

Completed·FFred Hutchinson Cancer Center·Phase 3·NCT01231412·174 enrolled

Beta-Thalassemia2 trials

Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients (THALA-RAP)

Unknown status·UUniversità degli Studi di Ferrara·Phase 2·NCT04247750·45 enrolled

Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients

Completed·RRare Partners srl Impresa Sociale·Phase 2·NCT03877809·26 enrolled

Breast Cancer2 trials

A Study of Sirolimus (Albumin-Bound) in Combination With Palbociclib and Fulvestrant for the Treatment of Advanced Breast Cancer

Recruiting·FFudan University·N/A·NCT06856200·36 enrolled

A Study of Sirolimus (Albumin-Bound) in Combination With Palbociclib and Fulvestrant for the Treatment of Advanced Breast Cancer

Not yet recruiting· CSPC ZhongQi Pharmaceutical Technology Co., Ltd. logo

CSPC ZhongQi Pharmaceutical Technology Co., Ltd.·Phase 1·NCT06941142·382 enrolled

Chemotherapy2 trials

Sirolimus in Combination With Metronomic Chemotherapy in Children With High-Risk Solid Tumors

Recruiting·EEmory University·Phase 2·NCT04469530·55 enrolled

Sirolimus in Combination With Metronomic Chemotherapy in Children With Recurrent and/or Refractory Solid and CNS Tumors

Active, not recruiting·EEmory University·Phase 2·NCT02574728·46 enrolled

Stem Cell Transplant2 trials

Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders

Active, not recruiting·—·Phase 2·NCT00544115·260 enrolled

Human Lysozyme Goat Milk in Treating Patients With Blood Cancer Undergoing Donor Stem Cell Transplant

Withdrawn·—·Phase 1·NCT03531281

Age-Related Macular Degeneration2 trials

Sirolimus for Advanced Age-Related Macular Degeneration

Completed·NNational Eye Institute (NEI)·Phase 1/2·NCT01445548·6 enrolled

Phase 1/2 Study of an Ocular Sirolimus (Rapamycin) Formulation in Patients With Age-Related Macular Degeneration

Terminated·SSanten Inc.·Phase 1/2·NCT00712491·20 enrolled

Advanced Cancer2 trials

Sirolimus or Vorinostat and Hydroxychloroquine in Advanced Cancer

Completed·MM.D. Anderson Cancer Center·Phase 1·NCT01266057·143 enrolled

Sirolimus or Everolimus or Temsirolimus and Vorinostat in Advanced Cancer

Active, not recruiting·MM.D. Anderson Cancer Center·Phase 1·NCT01087554·249 enrolled

Multiple Myeloma2 trials

Antithymocyte Globulin and Sirolimus in Treating Patients With Relapsed Multiple Myeloma

Completed·UUniversity of Rochester·Phase 1·NCT00317798·18 enrolled

Bortezomib Before Donor Stem Cell Transplant in Treating Patients With Multiple Myeloma

Withdrawn·MMayo Clinic·Phase 1/2·NCT00995059

Transplantation2 trials

Intestinal & Multivisceral Transplantation for Unresectable Mucinous Carcinoma Peritonei (TRANSCAPE)

Not yet recruiting·—·Phase 2·NCT06084780·20 enrolled

Completed·MMassachusetts General Hospital·Phase 2·NCT00133367·32 enrolled

Lymphoma2 trials

A Study of CD45RA+ Depleted Haploidentical Stem Cell Transplantation in Children With Relapsed or Refractory Solid Tumors and Lymphomas

Completed·SSt. Jude Children's Research Hospital·Phase 1·NCT01625351·23 enrolled

Tacrolimus/Sirolimus/Methotrexate vs Tacrolimus/Methotrexate or Cyclosporine/Mycophenolate Mofetil for GVHD Prophylaxis After Reduced Intensity Allogeneic Stem Cell Transplantation for Patients With Lymphoma

Completed·—·Phase 3·NCT00928018·139 enrolled

Refractory2 trials

Sirolimus Treatment for Refractory PRCA

Completed·BBing Han·Phase 4·NCT03364764·64 enrolled

Sirolimus in the Treatment of Refractory/Relapsed wAIHA

Recruiting·PPeking Union Medical College Hospital·N/A·NCT05925023·22 enrolled

Stent Restenosis2 trials

Systemic Rapamycin (Sirolimus) to Prevent In-Stent Restenosis Following Pulmonary Artery Stent Placement

Withdrawn·—·Phase 2·NCT02365415

Oral Sirolimus for In-Stent Restenosis

Completed·DDeutsches Herzzentrum Muenchen·Phase 4·NCT00859183·300 enrolled

Hematologic Malignancies2 trials

Cryopreserved MMUD BM With PTCy for Hematologic Malignancies

Withdrawn·OOssium Health, Inc.·Phase 1·NCT05170828

Sirolimus and Mycophenolate Mofetil in Preventing GVHD in Patients With Hematologic Malignancies Undergoing HSCT

Terminated·SStanford University·Phase 1·NCT02728700·1 enrolled

Hematopoietic Stem Cell Transplantation2 trials

Sirolimus, Mycophenolate Mofetil and Bortezomib as Graft-Versus-Host Disease (GVHD) Prophylaxis After Reduced Intensity Conditioning (RIC) Hematopoietic Stem Cell Transplantation

Terminated·—·Phase 2·NCT00548717·15 enrolled

Intravenous Administration of RGI-2001 in Patient Undergoing Allogenic Hematopoietic Stem Cell Transplantation (AHSCT)

Completed·RRegimmune Corporation·Phase 1/2·NCT01379209·68 enrolled

Liver Transplant2 trials

Effect of Rapamycin on Tolerance-related Biomarkers on Stable Liver Transplant Recipients

Unknown status·HHospital Clinic of Barcelona·Phase 2·NCT01034345·52 enrolled

Study Evaluating of Calcineurin Inhibitor and Sirolimus (Rapamune) Treatment in Liver Transplant Recipients

Terminated· Pfizer logo

Pfizer·Phase 3·NCT00086346·607 enrolled

Allograft2 trials

Study Evaluating The Use Of Sirolimus In Recipients Of Kidney Allografts From Expanded Criteria Donors (ECD)

Completed· Pfizer logo

Pfizer··NCT00697112·53 enrolled

Kidney Allograft Dysfunction Without Reversible Causes

Withdrawn·UUniversity of Minnesota·Phase 4·NCT01492894

TED2 trials

Sirolimus Effect on Hypertrophic Syndromes Related Gene PIK3CA

Completed·—·Phase 2·NCT02443818·16 enrolled

Topical Rapamycin/Sirolimus for Complicated Vascular Anomalies and Other Susceptible Lesions

Terminated·NNemours Children's Clinic·Phase 1·NCT04172922·5 enrolled

Chronic Graft Versus Host Disease2 trials

Ixazomib in the Prophylaxis of Chronic Graft-versus-host Disease.

Active, not recruiting·FFundación Pública Andaluza para la gestión de la Investigación en Sevilla·Phase 1/2·NCT03225417·142 enrolled

Sirolimus as Treatment of Steroid-Refractory or Steroid-Dependent Chronic Graft-Versus-Host Disease

Completed·SStanford University·Phase 2·NCT00388362·36 enrolled

Advanced Malignancies2 trials

Docetaxel and Sirolimus in Patients With Advanced Malignancies

Completed·MM.D. Anderson Cancer Center·Phase 1·NCT01054313·103 enrolled

Sirolimus and Cetuximab in Advanced Malignancies

Completed·MM.D. Anderson Cancer Center·Phase 1·NCT00940381·165 enrolled

Prostate Cancer2 trials

Effectiveness of Conversion to Sirolimus Versus Calcineurin Inhibitor (CNI) Reduction in Renal Transplant Patients With Prostate Cancer

Withdrawn·SSt. Joseph's Healthcare Hamilton·Phase 2/3·NCT00922129

Sirolimus, Docetaxel, and Carboplatin in Treating Patients With Metastatic Castration-Resistant Prostate Cancer

Terminated·UUniversity of Washington·Phase 1/2·NCT02565901·28 enrolled

Pancreatic Cancer2 trials

Sirolimus and Vismodegib in Treating Patients With Solid Tumors or Pancreatic Cancer That is Metastatic or Cannot Be Removed By Surgery

Completed·MMayo Clinic·Phase 1·NCT01537107·31 enrolled

Sirolimus in Treating Patients With Advanced Pancreatic Cancer

Completed·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 2·NCT00499486·47 enrolled

Children1 trial

Systematic Approach for Children With Orbital Malformation With Sirolimus Use

Recruiting·—·Phase 3·NCT07378657·30 enrolled

Pneumonia1 trial

Rapamune Improves Outcomes of Severe H1N1 Pneumonia

Completed·—·Phase 2·NCT01620307·38 enrolled

Autoimmune Disease1 trial

Sirolimus for Autoimmune Disease of Blood Cells

Completed·—·Phase 1/2·NCT00392951·30 enrolled

Familial adenomatous polyposis1 trial

Sirolimus and Familial Adenomatous Polyposis (FAP)

Completed·—·Phase 2·NCT03095703·4 enrolled

Patients With Advanced Solid Tumors1 trial

A Pharmacodynamic Study of Sirolimus and Metformin in Patients With Advanced Solid Tumors

Completed·UUniversity of Chicago·Phase 1·NCT02145559·24 enrolled

Neurofibromatosis Type 11 trial

A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas

Completed·UUniversity of Alabama at Birmingham·Phase 2·NCT00634270·58 enrolled

Safety and Efficacy1 trial

A Dose-Ranging Clinical Study to Assess the Safety and Efficacy of Subconjunctival Injection of Sirolimus in Patients With Dry Eye in the Controlled Adverse Environmental (CAE) Model

Completed·SSanten Inc.·Phase 2·NCT00814944·143 enrolled

Bronchitis1 trial

The Efficacy and Safety of Sirolimus for Plastic Bronchitis

Unknown status·PPeking Union Medical College Hospital·Phase 2·NCT03942926·10 enrolled

Tuberous Sclerosis1 trial

Trial of Efficacy and Safety of Sirolimus in Tuberous Sclerosis and LAM

Unknown status·—·Phase 2·NCT00490789·14 enrolled

Fanconi Anemia1 trial

TBI Dose De-escalation for Fanconi Anemia

Completed·MMasonic Cancer Center, University of Minnesota·Phase 2/3·NCT00352976·83 enrolled

IgG4 Related Disease1 trial

Study of Sirolimus in IgG4-related Disease

Not yet recruiting·PPeking University International Hospital·N/A·NCT05746689·20 enrolled

Completed·VVascular Therapies, Inc.·Phase 3·NCT05425056·136 enrolled

Pilot Study1 trial

Pilot Study of the Evaluation of Subconjunctival Sirolimus in the Treatment of Bilateral GA Associated With AMD

Completed·NNational Eye Institute (NEI)·Phase 1/2·NCT00766649·11 enrolled

NPC1 trial

Bioequivalence Study of NPC-12 (Sirolimus) Granules and Tablets

Completed·NNobelpharma·Phase 1·NCT03765944·10 enrolled

Myelofibrosis1 trial

Ruxolitinib Phosphate, Tacrolimus and Sirolimus in Preventing Acute Graft-versus-Host Disease During Reduced Intensity Donor Hematopoietic Cell Transplant in Patients With Myelofibrosis

Withdrawn·—·Phase 1·NCT02528877

Cardiac Allograft Vasculopathy1 trial

Strategies To Prevent Cardiac Allograft Vasculopathy Related Events in Heart Transplant Recipients

Withdrawn·UUniversity of Minnesota·N/A·NCT01305395

Juvenile Nasopharyngeal Angiofibroma1 trial

Efficacy and Safety of Sirolimus in Children and Adolescents With Juvenile Nasopharyngeal Angiofibroma (JNA)

Recruiting·FFederal Research Institute of Pediatric Hematology, Oncology and Immunology·Phase 3·NCT05549167·117 enrolled

Port Wine Stains1 trial

Treatment of Port Wine Stains Using Pulsed Dye Laser, Erbium Yag Laser and Topical Sirolimus

Terminated·EErasmus Medical Center·N/A·NCT02214706·17 enrolled

Chronic Granulomatous Disease1 trial

Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists

Recruiting·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1/2·NCT05463133·50 enrolled

Hemoglobinopathies1 trial

Allo HSCT for High Risk Hemoglobinopathies

Recruiting·MMasonic Cancer Center, University of Minnesota·Phase 2·NCT06872333·62 enrolled

Basal Cell Nevus Syndrome1 trial

Topical Sirolimus in Patients With Basal Cell Nevus Syndrome and in Healthy Participants

Completed·YYale University·Phase 1·NCT00433485·16 enrolled

T Cell1 trial

Methods of T Cell Depletion Trial (MoTD)

Unknown status·UUniversity of Birmingham·Phase 2·NCT04888741·400 enrolled

Trial1 trial

Serial Measurements of Molecular and Architectural Responses to Therapy (SMMART) PRIME Trial

Terminated·OOHSU Knight Cancer Institute·Phase 1·NCT03878524·2 enrolled

Immunosuppression1 trial

Renal Allograft Function and Histology Following Switching From A Tacrolimus to Sirolimus (SRL)-Based Immunosuppression-

Terminated·TThe Cleveland Clinic·Phase 3·NCT01166724·12 enrolled

Thyroid Cancer1 trial

Cyclophosphamide and Sirolimus for the Treatment of Metastatic, RAI-refractory, Differentiated Thyroid Cancer

Recruiting·UUniversity of Michigan Rogel Cancer Center·Phase 2·NCT03099356·22 enrolled

Leukemia1 trial

Pilot Trial of Sirolimus/MEC in High Risk Acute Myelogenous Leukemia (AML)

Completed·SSidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University·N/A·NCT01184898·36 enrolled

Malignant Peripheral Nerve Sheath Tumor1 trial

SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors

Completed·SSarcoma Alliance for Research through Collaboration·Phase 2·NCT03433183·21 enrolled

Fed1 trial

Bioavailability Study of Sirolimus Tablets 2 mg Under Fed Condition

Completed·—·Phase 1·NCT02296762·36 enrolled

Autosomal Dominant Polycystic Kidney Disease1 trial

Sirolimus (Rapamune®) for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Completed·UUniversity of Zurich·Phase 3·NCT00346918·100 enrolled

COVID-191 trial

Efficacy and Safety of Sirolimus in COVID-19 Infection

Unknown status·—·Phase 2·NCT04461340·40 enrolled

Primary Peritoneal Cancer1 trial

Sirolimus and Vaccine Therapy in Treating Patients With Stage II-IV Ovarian, Fallopian Tube, or Primary Peritoneal Cancer

Withdrawn·RRoswell Park Cancer Institute·Phase 1·NCT02833506

Refractory Solid Tumors1 trial

The Pilot Study Evaluate the Safety and Efficacy of Sirolimus in Patients With PIK3CA Mutation and/or PIK3CA Amplification Refractory Solid Tumors

Completed·SSamsung Medical Center·N/A·NCT02449564·5 enrolled

Skin Cancer1 trial

Prevention of Skin Cancer in High Risk Patients After Conversion to a Sirolimus-based Immunosuppressive Protocol

Terminated·—·Phase 4·NCT01797315·40 enrolled

Adult1 trial

This Study Consists of Two Study Parts Conducted Under a Single IRB. Part I: Short Term ApoE-dependent Cerebral Blood Flow Response to Sirolimus in Cognitively Normal Adults Part II: Short Term ApoE-dependent Cerebral Blood Flow and Lung Perfusion Response to Sirolimus in Cognitively Normal Adults

Active, not recruiting·UUniversity of Missouri-Columbia·Phase 1·NCT05386914·205 enrolled

Bladder Cancer1 trial

A Study of Rapamycin Combined With Intravesical BCG in Patients With Non-muscle Invasive Bladder Cancer

Completed·TThe University of Texas Health Science Center at San Antonio·Phase 1·NCT02753309·33 enrolled

Withdrawal1 trial

SRL (Sirolimus) Withdrawal

Completed·NNorthwestern University·N/A·NCT02062944·21 enrolled

Peripheral Blood Stem Cell Transplantation1 trial

Tacrolimus and Sirolimus as Prophylaxis After Allogenic Non-myeloablative Peripheral Blood Stem Cell Transplantation

Completed·—·Phase 2·NCT00282282·31 enrolled

Renal Transplant1 trial

Polymorphism of the Cytochrome P450-system in Renal Transplants

Completed·UUniversity Hospital Schleswig-Holstein··NCT00223054·200 enrolled

Hamartoma1 trial

Sirolimus for Retinal Astrocytic Hamartoma

Completed·TThe Hospital for Sick Children·N/A·NCT04707209·1 enrolled

Hematopoietic Stem Cell Transplant1 trial

Standard of Care vs. Bortezomib in Graft-Versus Host Disease After Hematopoietic Stem Cell Transplant

Completed·—·Phase 2·NCT01754389·138 enrolled

Allogeneic Hematopoietic Cell Transplant1 trial

Donor Lymphocyte Infusion After Allogeneic Hematopoietic Cell Transplantation for High-Risk Hematologic Malignancies

Recruiting·NNational Cancer Institute (NCI)·Phase 1/2·NCT05327023·430 enrolled

Solid Tumors1 trial

Aflac ST0901 CHOANOME - Sirolimus in Solid Tumors

Completed·EEmory University·Phase 1·NCT01331135·18 enrolled

Renal Allograft Recipients1 trial

Study Comparing Two Immunosuppressive Regimens in De Novo Renal Allograft Recipients

Completed· Pfizer logo

Pfizer·Phase 4·NCT00261820·160 enrolled

Islet Transplantation1 trial

B-Lymphocyte Immunotherapy in Islet Transplantation

Terminated·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT00468442·2 enrolled

MPR1 trial

Sirolimus for Improving Social Abilities in People With PTEN Germline Mutations

Withdrawn·SStanford University·Phase 1/2·NCT06080165

Aneurysm1 trial

Effect of Sirolimus on Molecular Alterations in Cerebral Aneurysms

Recruiting·UUniversity of Miami·Phase 2·NCT04141020·80 enrolled

Growth1 trial

Sirolimus Versus Anti-Vascular Endothelial Growth Factor (antiVEGF) for Wet AMD

Completed·MMaturi, Raj K., M.D., P.C.·Phase 2·NCT02357342·40 enrolled

Patients With Solid Tumors1 trial

Sirolimus in Treating Patients With Solid Tumors That Are Metastatic or Cannot Be Removed By Surgery

Completed·UUniversity of Chicago·Phase 1·NCT01791088·56 enrolled

B-cell NHL1 trial

A Phase II Study of Allo-HCT for B-Cell NHL Using Zevalin, Fludarabine and Melphalan

Completed·—·Phase 2·NCT00577278·41 enrolled

Allogeneic Stem Cell Transplantation1 trial

Sirolimus, Tacrolimus and Short Course Methotrexate for Prevention of Acute GVHD in Recipients of Mismatched Unrelated Donor Allogeneic Stem Cell Transplantation

Completed·YYale University·Phase 1·NCT00612274·26 enrolled

Phase II1 trial

SCD-Haplo: Phase II Study of HLA-Haploidentical SCT for Aggressive SCD

Terminated·—·Phase 2·NCT02013375·2 enrolled

Desmoid-type fibromatosis1 trial

A Pilot Study Evaluating the Use of mTor Inhibitor Sirolimus in Children and Young Adults With Desmoid-Type Fibromatosis

Completed·MMaineHealth·Phase 1/2·NCT01265030·9 enrolled

Lupus Nephritis1 trial

Sirolimus in Treatment of Proteinuric Flares of Lupus Nephritis

Unknown status·PPeking Union Medical College Hospital·Phase 2/3·NCT04892212·20 enrolled

Tolerance1 trial

Belatacept Post Depletional Repopulation to Facilitate Tolerance

Completed·—·Phase 2·NCT00565773·40 enrolled

Total Body Irradiation1 trial

Total Body Irradiation and Astatine-211-Labeled BC8-B10 Monoclonal Antibody for the Treatment of Nonmalignant Diseases

Recruiting·FFred Hutchinson Cancer Center·Phase 1/2·NCT04083183·40 enrolled

Cholangiocarcinoma1 trial

Sirolimus, Gemcitabine Hydrochloride, and Cisplatin in Treating Patients At High Risk for Cholangiocarcinoma Recurrence After Liver Transplant or Surgery

Completed·MMayo Clinic·Phase 1·NCT01888302·1 enrolled

Multiple System Atrophy1 trial

A Futility Trial of Sirolimus in Multiple System Atrophy

Terminated·NNYU Langone Health·Phase 2·NCT03589976·47 enrolled

Squamous Cell Carcinoma1 trial

Topical Sirolimus in Chemoprevention of Facial Squamous Cell Carcinomas in Solid Organ Transplant Recipients (SiroSkin)

Active, not recruiting·MMelanoma and Skin Cancer Trials Limited·Phase 3·NCT05860881·146 enrolled

Uveitis1 trial

Sirolimus as Therapeutic Approach to Uveitis

Completed·JJohns Hopkins University·Phase 1·NCT00908466·30 enrolled

Antiretroviral Treatment1 trial

Immunotherapy With Baricitinib and Sirolimus, Alone and in Combination, for the Control of HIV-1 Replication After Antiretroviral Treatment Interruption

Not yet recruiting·AANRS, Emerging Infectious Diseases·Phase 2·NCT07081763·191 enrolled

Oral1 trial

Study of Oral Rapamycin Plus Bare Metal Stents vs Drug Eltuting Stents

Completed·CCentro de estudios en Cardiologia Intervencionista·Phase 4·NCT00552669·200 enrolled

Biomarkers1 trial

Discovery of Sirolimus Sensitive Biomarkers in Blood

Completed·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 2·NCT03304678·33 enrolled

Thyroid Eye Disease1 trial

Sirolimus vs Corticosteroids in Treatment of Thyroid Eye Disease

Recruiting·HHaukeland University Hospital·Phase 2·NCT04936854·60 enrolled

Lymphatic Malformation1 trial

Different Doses of Sirolimus for the Treatment of Cystic Lymphatic Malformations

Recruiting·WWest China Hospital·Phase 2/3·NCT06673290·150 enrolled

Efficacy1 trial

Efficacy of Topical Rapamycin to Treat Chronic Erosive Oral Lichen

Terminated·UUniversity Hospital, Tours·Phase 3·NCT01061853·76 enrolled

AIN1 trial

Study Evaluating Rapamune® Maintenance Regimen

Completed· Pfizer logo

Pfizer·Phase 3·NCT00478608·79 enrolled

Statin1 trial

Safety of Simvastatin in LAM and TSC

Completed·UUniversity of Pennsylvania·Phase 1/2·NCT02061397·10 enrolled

Prevention1 trial

Calcineurin Inhibitor-Free GVHD Prevention Regimen After Related Haplo PBSCT

Completed·HH. Lee Moffitt Cancer Center and Research Institute·Phase 1·NCT03018223·32 enrolled

Advanced Solid Tumors1 trial

A Study of Sirolimus (Albumin-Bound) in Combination With Different ADCs Treatment of Advanced Solid Tumors

Recruiting· CSPC ZhongQi Pharmaceutical Technology Co., Ltd. logo

CSPC ZhongQi Pharmaceutical Technology Co., Ltd.·Phase 1/2·NCT07241936·444 enrolled

Hemangioendothelioma1 trial

Sirolimus in the Treatment for Infantile Hepatic Hemangioendothelioma(IEEH)

Unknown status·SShanghai Children's Medical Center·Phase 4·NCT04406870·36 enrolled

Solid Organ Transplant Recipients1 trial

Study Evaluating Sirolimus (Rapamune™) in Solid Organ Transplant Recipients

Completed· Pfizer logo

Pfizer·Phase 3·NCT00037531·769 enrolled

Pemphigus1 trial

Evaluating Sirolimus to Treat Autoimmune Blistering Dermatosis Pemphigus

Terminated·UUniversity of California, Irvine·Phase 1·NCT01313923·3 enrolled

Systemic Lupus Erythematosus1 trial

Efficacy and Safety of Sirolimus in Active Systemic Lupus Erythematosus

Active, not recruiting·CChinese SLE Treatment And Research Group·Phase 3·NCT04582136·146 enrolled

Myelodysplastic Syndrome1 trial

Itacitinib, Tacrolimus, and Sirolimus for the Prevention of GVHD in Patients With Acute Leukemia, Myelodysplastic Syndrome, or Myelofibrosis Undergoing Reduced Intensity Conditioning Donor Stem Cell Transplantation

Active, not recruiting·—·Phase 2·NCT04339101·59 enrolled

Stem Cell Transplantation1 trial

Sirolimus With Tacrolimus for Graft-vs-Host Disease Prophylaxis After Related Stem Cell Transplantation

Completed·—·Phase 2·NCT00144703·55 enrolled

Myeloid Neoplasm1 trial

Early Allogeneic Hematopoietic Cell Transplantation in Treating Patients With Relapsed or Refractory High-Grade Myeloid Neoplasms

Completed·UUniversity of Washington·Phase 2·NCT02756572·30 enrolled

Phase 11 trial

Phase 1 Study of the Effects of Combining Topical FDA-approved Drugs on Age-related Pathways on the Skin of Healthy Volunteers

Completed·AAnne Chang·Phase 1·NCT03072485·10 enrolled

Hepatocellular Carcinoma1 trial

Immunosuppression in Patients Undergoing Liver Transplantation for Hepatocellular Carcinoma

Completed·UUniversity of Regensburg·Phase 3·NCT00355862·525 enrolled

Kaposi's sarcoma1 trial

Sirolimus in Treating Patients With HIV-Related Kaposi's Sarcoma

Completed·AAIDS Malignancy Consortium·Phase 1·NCT00450320·7 enrolled

Renal Cell Carcinoma1 trial

Erlotinib and Sirolimus for the Treatment of Metastatic Renal Cell Carcinoma

Completed·UUniversity of Colorado, Denver·Phase 2·NCT00353301·25 enrolled

Nephropathy1 trial

Using mTOR Inhibitors in the Prevention of BK Nephropathy

Completed·—·N/A·NCT01649609·40 enrolled

Anemia1 trial

Non-Myeloablative Bone Marrow Transplant for Patients With Sickle Cell Anemia and Other Blood Disorders

Terminated·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 2·NCT00489281·43 enrolled

Hyperinsulinism1 trial

Sirolimus for the Treatment of Hyperinsulinism

Withdrawn·—·Phase 1/2·NCT02524639

Unspecified4 trials

Study Investigating Rapamune For Post-Marketing Surveillance

Completed· Pfizer logo

Pfizer··NCT00484094·209 enrolled

Weekly Sirolimus Therapy

Recruiting·MMedical University of South Carolina·Phase 2·NCT04861064·24 enrolled

Rapalog Pharmacology (RAP PAC) Study

Recruiting·UUniversity of Wisconsin, Madison·Phase 1·NCT05949658·72 enrolled

Show 1 more trial

Sirolimus for Graves' Orbitopathy (GO)

Unknown status·UUniversity of Pisa·Phase 2·NCT04598815·54 enrolled

Regulatory

FDA Approval Timeline[5]

Sep 15, 1999

RAPAMUNE

NDA NDA021083

Jan 25, 2010

RAPAMUNE

NDA NDA021110

Nov 22, 2021

FYARRO

NDA NDA213312

Mar 22, 2022

HYFTOR

NDA NDA213478

Sources

References

[1]
ClinicalTrials.gov — 420 trial records for Sirolimus. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Rapamune drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — Sirolimus compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for Sirolimus. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA213478). U.S. Food & Drug Administration.
[6]
FDA Orange Book — Patent and exclusivity data for Rapamune. U.S. Food & Drug Administration.
[7]
SEC EDGAR — Prism Pharmaceuticals company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: FK506-Binding Protein 1a Inhibitor[3]
Modality: Small molecule[3]
Highest Phase: Approved[1]
Company: PPrism Pharmaceuticals

Patent Landscape[6]

Patent Cliff

Feb 14, 2029

Earliest patent expiration — generic entry possible after this date

Patent #Expires

8911786Feb 14, 2029

10206887Apr 15, 2030

12061183Mar 5, 2036

10705070Mar 5, 2036

10973806Jun 29, 2036

2 more patents

12133844Jun 29, 2036

11497737Oct 28, 2040

Rapamune

Status by Indication[4][1]

Competitive Landscape

Expected Milestones

Q3 2026

Q4 2026

Trials by Indication[1]

Kidney Transplant20 trials

Sickle Cell Disease9 trials

Acute Graft Versus Host Disease5 trials

Efficacy and Safety5 trials

Non-Small Cell Lung Cancer5 trials

Acute Lymphoblastic Leukemia3 trials

Aplastic Anemia3 trials

Treatment3 trials

Type 1 Diabetes3 trials

Vascular Malformation3 trials

Renal Transplant Recipients3 trials

Bone Marrow Transplantation3 trials

Kaposiform Hemangioendothelioma3 trials

Graft Versus Host Disease3 trials

Beta-Thalassemia2 trials

Breast Cancer2 trials

Chemotherapy2 trials

Stem Cell Transplant2 trials

Age-Related Macular Degeneration2 trials

Advanced Cancer2 trials

Multiple Myeloma2 trials

Transplantation2 trials

Lymphoma2 trials

Refractory2 trials

Stent Restenosis2 trials

Hematologic Malignancies2 trials

Hematopoietic Stem Cell Transplantation2 trials

Liver Transplant2 trials

Allograft2 trials

TED2 trials

Chronic Graft Versus Host Disease2 trials

Advanced Malignancies2 trials

Prostate Cancer2 trials

Pancreatic Cancer2 trials

Children1 trial

Pneumonia1 trial

Autoimmune Disease1 trial

Familial adenomatous polyposis1 trial

Patients With Advanced Solid Tumors1 trial

Neurofibromatosis Type 11 trial

Safety and Efficacy1 trial

Bronchitis1 trial

Tuberous Sclerosis1 trial

Fanconi Anemia1 trial

IgG4 Related Disease1 trial

Autism1 trial

Immune Suppression1 trial

Surgery1 trial

Pilot Study1 trial

NPC1 trial

Myelofibrosis1 trial

Cardiac Allograft Vasculopathy1 trial

Juvenile Nasopharyngeal Angiofibroma1 trial

Port Wine Stains1 trial

Chronic Granulomatous Disease1 trial

Hemoglobinopathies1 trial

Basal Cell Nevus Syndrome1 trial

T Cell1 trial

Trial1 trial

Immunosuppression1 trial

Thyroid Cancer1 trial

Leukemia1 trial

Malignant Peripheral Nerve Sheath Tumor1 trial

Fed1 trial

Autosomal Dominant Polycystic Kidney Disease1 trial

COVID-191 trial

Primary Peritoneal Cancer1 trial

Refractory Solid Tumors1 trial

Skin Cancer1 trial

Adult1 trial

Bladder Cancer1 trial

Withdrawal1 trial

Peripheral Blood Stem Cell Transplantation1 trial