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 147 indications[4].[1]

Rapamune is one of 1700 small molecule programs targeting Sickle Cell Disease. It is currently in Approved.

147

Indications

427

Total Trials

Active Trials

1,699

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[4][1]

Indication

Sickle Cell DiseaseActive, not recruiting

Lung TransplantationActive, not recruiting

Chronic Graft Versus Host DiseaseCompleted

ChemotherapyRecruiting

Efficacy and SafetyCompleted

ProphylaxisCompleted

Familial Platelet DisorderRecruiting

LeukemiaApproved

ThrombocytopeniaActive, not recruiting

Idiopathic Pulmonary FibrosisApproved

Sickle Cell DiseasePhase 2

Active, not recruiting

Lung TransplantationApproved

Active, not recruiting

Chronic Graft Versus Host DiseasePhase 2

Completed

ChemotherapyPhase 2

Recruiting

Efficacy and SafetyPhase 3

Completed

ProphylaxisPhase 2

Completed

Familial Platelet DisorderPhase 2

Recruiting

LeukemiaApproved

Approved

ThrombocytopeniaApproved

Active, not recruiting

Idiopathic Pulmonary FibrosisApproved

Approved

Showing 10 of 147 indications

Competition

Competitive Landscape

744 competitors in Breast Cancer

#DrugCompanyPhaseTrials

Rapamune

PPrism Pharmaceuticals

Approved

2 2

Blenoxane

Bristol-Myers Squibb

Preclinical

1 3

Voltaren

Novartis

Preclinical

1 4

Nubain

AAultman Health Foundation

Preclinical

1 5

Duragesic

CCephalon

Preclinical

1 6

Tricor

JJagotec

Preclinical

1 7

Beta Blocker

WWeill Medical College of Cornell University

Preclinical

1 8

Neoadjuvant Treatment

SShanghai Zhongshan Hospital

Preclinical

1 9

Tianeptine

Pfizer

Preclinical

1 10

Gadolinium-Chelate

MM.D. Anderson Cancer Center

Preclinical

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

Timeline

Expected Milestones

Q4 2026

Phase 1/2Active, not recruiting

Study completion

NCT03983850

Post-ApprovalActive, not recruiting

Kidney Transplant

NCT05193565

Phase 1/2Recruiting

Thalassemia

NCT05426252

Phase 2Recruiting

Randomized

NCT06091332

Q2 2027

Phase 2Not yet recruiting

Sarcoidosis

NCT05458492

Phase 1/2Active, not recruiting

Graft Versus Host Disease

NCT03192397

Clinical

Trials by Indication[1]

Kidney Transplant21 trials

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

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

Study Evaluating Two Different Sirolimus-based Immunosuppressive Regimens in Elderly Kidney Transplant Recipients

Completed· Pfizer logo

Pfizer·Phase 4·NCT00254709·66 enrolled

Comparison of the Efficacy and Safety of Sirolimus Versus Everolimus Versus Mycophenolate in Kidney Transplantation

Completed·HHospital do Rim e Hipertensão·Phase 4·NCT03468478·1,209 enrolled

Show 18 more trials

Study Evaluating the Efficacy and Safety of Cyclosporine Reduction in Kidney Transplant Recipients Receiving Sirolimus

Completed· Pfizer logo

Pfizer·Phase 4·NCT00507793·385 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

A Study Comparing the Withdrawal of Steroids or Tacrolimus in Kidney Transplant Recipients

Completed· Pfizer logo

Pfizer·Phase 4·NCT00195429·47 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

Study Comparing Conversion to Sirolimus vs. Continued Use of Calcineurin Inhibitors in Kidney Transplant Recipients

Completed· Pfizer logo

Pfizer·Phase 3·NCT00038948·830 enrolled

Study Comparing Sirolimus With Cyclosporine in a Calcineurin Inhibitor (CNI)-Free Regimen in Kidney Transplant Recipients

Terminated· Pfizer logo

Pfizer·Phase 3·NCT00137345·500 enrolled

Effects of the Quadruple Immunosuppression on Peripheral Blood Lymphocytes and Development of Anti-HLA Antibodies in Kidney Transplant

Unknown status·UUniversity of Sao Paulo General Hospital·Phase 4·NCT02208791·45 enrolled

Study Comparing Two Sirolimus Regimens vs. Tacrolimus and Mycophenolate Mofetil Regimen in Kidney Transplant Recipients

Completed· Pfizer logo

Pfizer·Phase 4·NCT00266123·420 enrolled

Study Evaluating A Planned Transition From Tacrolimus To Sirolimus In Kidney Transplant Recipients

Completed· Pfizer logo

Pfizer·Phase 4·NCT00895583·254 enrolled

Safety in Immunomodulatory Functions of Alemtuzumab (Campath) in Pediatric Kidney Transplantation Recipients

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT00240994·35 enrolled

Study to Compare the Efficacy and Safety After Conversion to RaparoBell® or My-Rept® in Kidney Transplant Patients

Active, not recruiting· Chong Kun Dang Pharmaceutical logo

Chong Kun Dang Pharmaceutical·Phase 4·NCT05193565·206 enrolled

Pilot Study on the Use of Sirolimus to Treat Chronic Allograft Nephropathy in Children After Kidney Transplant

Completed·UUniversity of Manitoba·Phase 4·NCT00188955·40 enrolled

Study Evaluating Rapamune in Patients After Kidney Transplantation

Terminated· Pfizer logo

Pfizer··NCT00240214·500 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

Using Donor Stem Cells and Alemtuzumab to Prevent Organ Rejection in Kidney Transplant Patients

Completed·UUniversity of Miami·Phase 1/2·NCT00183248·9 enrolled

Improved Induction and Maintenance Immunosuppression in Kidney Transplantation

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

Graft Versus Host Disease7 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

Tacrolimus, Sirolimus and Methotrexate as Graft Versus Host Disease Prophylaxis After Blood Stem Cell Transplantation

Completed·—·Phase 2·NCT00146614·105 enrolled

Show 4 more trials

In Vivo Treg Expansion and Graft-Versus-Host Disease Prophylaxis

Completed·HH. Lee Moffitt Cancer Center and Research Institute·Phase 2·NCT01927120·20 enrolled

Rasburicase in Preventing Graft-Versus-Host Disease in Patients With Hematologic Cancer or Other Disease Undergoing Donor Stem Cell Transplant

Completed·MMassachusetts General Hospital·Phase 1·NCT00513474·46 enrolled

Sirolimus/Tacrolimus Versus Tacrolimus/Methotrexate for Preventing Graft-Versus-Host Disease (GVHD) (BMT CTN 0402)

Completed·MMedical College of Wisconsin·Phase 3·NCT00406393·304 enrolled

Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant

Active, not recruiting·RRoswell Park Cancer Institute·Phase 1/2·NCT03192397·35 enrolled

Treatment5 trials

Sirolimus Treatment Of Patients With SLE

Not yet recruiting·SState University of New York - Upstate Medical University·Phase 2·NCT04736953·220 enrolled

Subconjunctival Sirolimus for the Treatment of Autoimmune Active Anterior Uveiti

Completed·NNational Eye Institute (NEI)·Phase 1·NCT00876434·5 enrolled

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

Terminated·—·Phase 2·NCT02110069·4 enrolled

Show 2 more trials

A Study to Evaluate Steroid-free Treatment for Standard-Risk aGVHD (BMT CTN 1501)

Completed·MMedical College of Wisconsin·Phase 2·NCT02806947·127 enrolled

Safety and Durability of Sirolimus for Treatment of LAM

Recruiting·UUniversity of Cincinnati··NCT02432560·600 enrolled

Hematologic Malignancies4 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

Safety and Efficacy of ProHema Modulated Umbilical Cord Blood Units in Subjects With Hematologic Malignancies.

Completed·FFate Therapeutics·Phase 1·NCT00890500·12 enrolled

Show 1 more trial

Reduced Intensity, Partially HLA Mismatched BMT to Treat Hematologic Malignancies

Completed·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 1/2·NCT01203722·87 enrolled

Non-Small Cell Lung Cancer4 trials

Sirolimus and Pemetrexed to Treat Non-Small Cell Lung Cancer

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

Sirolimus and Gold Sodium Thiomalate in Treating Patients With Advanced Squamous Non-Small Cell Lung Cancer

Withdrawn·MMayo Clinic·Phase 1·NCT01383668

Sirolimus and Durvalumab for the Treatment of Stage I-IIIA Non-small Cell Lung Cancer

Terminated·EEmory University·Phase 1·NCT04348292·3 enrolled

Show 1 more trial

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

115 more indications

Stem Cell Transplantation4 trials

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

Completed·—·Phase 2·NCT00144677·30 enrolled

Anti-Third Party T Lymphocytes With Nonmyeloablative Stem Cell Transplantation for Indolent Lymphoid Malignancies

Terminated·MM.D. Anderson Cancer Center·Phase 1·NCT00473551·4 enrolled

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

Completed·—·Phase 2·NCT00144703·55 enrolled

Show 1 more trial

Stem Cell Transplantation in Patients With High-Risk and Recurrent Pediatric Sarcomas

Completed·NNational Cancer Institute (NCI)·Phase 2·NCT00043979·60 enrolled

Liver Transplant3 trials

Sirolimus-based Immunosuppression Treatment Regimen for Liver Transplantation

Unknown status·SSouthern Medical University, China·Phase 2/3·NCT03500848·130 enrolled

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

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

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

Completed· Roche logo

Roche·Phase 4·NCT00118742·293 enrolled

Renal Transplant Recipients3 trials

Pilot Study for HLA Identical Living Donor Renal Transplant Recipients

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

Impact of Conversion From Tacrolimus to Sirolimus in African American Renal Transplant Recipients

Unknown status·TTemple University·Phase 4·NCT00999258·50 enrolled

Study To Evaluate Pharmacokinetics Of Sirolimus In Stable Renal Transplant Recipients

Completed· Pfizer logo

Pfizer·Phase 1·NCT01236378·24 enrolled

Sickle Cell Disease3 trials

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

Recruiting·—·Phase 1·NCT07015684·24 enrolled

Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell Antibody

Recruiting·—·Phase 2·NCT06358638·12 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

Stem Cell Transplant3 trials

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

Withdrawn·—·Phase 1·NCT03531281

Total-Body Irradiation and Cyclophosphamide in Treating Patients Who Are Undergoing Donor Stem Cell Transplant for Hematologic Cancer and Other Diseases

Completed·FFred Hutchinson Cancer Center·Phase 2·NCT00317785·50 enrolled

Sirolimus/Tacrolimus Combination After HLA Matched Related Peripheral Blood Stem Cell Transplants

Terminated·TThe Korean Society of Blood and Marrow Transplantation·Phase 2·NCT01488253·3 enrolled

Hepatocellular Carcinoma3 trials

Immunosuppression in Patients Undergoing Liver Transplantation for Hepatocellular Carcinoma

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

De Novo Sirolimus-based Immunosuppression After Liver Transplantation for Hepatocellular Carcinoma

Completed·UUniversity of Alberta·Phase 2/3·NCT00328770·70 enrolled

Efficacy of Sirolimus In Liver Transplantation for Hepatocellular Carcinoma (HCC)

Completed·SSeoul National University Hospital·Phase 2·NCT01374750·45 enrolled

Prophylaxis3 trials

Sirolimus & Mycophenolate Mofetil as GvHD Prophylaxis in Myeloablative, Matched Related Donor HCT

Terminated·SStanford University·Phase 2·NCT01220297·3 enrolled

Sirolimus & Mycophenolate Mofetil as GVHD Prophylaxis in Myeloablative, Matched Related Donor HCT

Completed·SStanford University·Phase 2·NCT00350181·11 enrolled

Sirolimus+Abatacept+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to Calcineurin Inhibitors

Not yet recruiting·PPeking University People's Hospital·Phase 1/2·NCT06279494·6 enrolled

Immunosuppression3 trials

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

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

Efficacy of Sirolimus-Based, Steroid Avoidance Immunosuppression African Americans

Completed·UUniversity of Michigan·Phase 2/3·NCT00189202·49 enrolled

Calcineurin Inhibitor Based Immunosuppression Withdrawal

Completed·PPediatric Nephrology of Alabama·N/A·NCT02117596·52 enrolled

Malignant Peripheral Nerve Sheath Tumor3 trials

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

PLX3397 Plus Sirolimus in Unresectable Sarcoma and Malignant Peripheral Nerve Sheath Tumors

Terminated·GGulam Manji·Phase 1/2·NCT02584647·39 enrolled

SARC023: Ganetespib and Sirolimus in Patients With MPNST (Malignant Peripheral Nerve Sheath Tumors)

Completed·SSarcoma Alliance for Research through Collaboration·Phase 1/2·NCT02008877·20 enrolled

Efficacy and Safety3 trials

Efficacy and Safety of Sirolimus in Combination With Tacrolimus

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

Efficacy and Safety of Sirolimus to Vascular Anomalies

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

Safety and Efficacy3 trials

Safety and Efficacy of Sirolimus for HIV Reservoir Reduction in Individuals on Suppressive Antiretroviral Therapy (ART)

Completed·AAdvancing Clinical Therapeutics Globally for HIV/AIDS and Other Infections·Phase 1/2·NCT02440789·32 enrolled

Study to Evaluate Safety and Efficacy of Early Calcineurin Inhibitor Withdrawal in Primary Renal Allografts

Completed·UUniversity of Cincinnati·Phase 4·NCT00374647·17 enrolled

Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies

Completed·DDenise Martin Adams·Phase 2·NCT00975819·61 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

Lymphangioleiomyomatosis2 trials

Resveratrol and Sirolimus in Lymphangioleiomyomatosis Trial

Completed·UUniversity of Cincinnati·Phase 2·NCT03253913·25 enrolled

Multicenter Interventional Lymphangioleiomyomatosis (LAM) Early Disease Trial

Active, not recruiting·UUniversity of Cincinnati·Phase 3·NCT03150914·60 enrolled

Multiple Myeloma2 trials

High Dose Busulfan and Bortezomib in Treating Patients With High Risk Multiple Myeloma Undergoing Stem Cell Transplant

Terminated·BBarbara Ann Karmanos Cancer Institute·Phase 2·NCT01534143·1 enrolled

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

Withdrawn·MMayo Clinic·Phase 1/2·NCT00995059

Transplantation2 trials

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

Comparison of Triple GVHD Prophylaxis Regimens for Nonmyeloablative or Reduced Intensity Conditioning Unrelated Mobilized Blood Cell Transplantation

Terminated·FFred Hutchinson Cancer Center·Phase 2·NCT03246906·150 enrolled

Acute Lymphoblastic Leukemia2 trials

Study of Sirolimus With PEG-Asparaginase in Acute Lymphoblastic Leukemia (ALL)

Withdrawn·EEmory University·Phase 1·NCT00957320

Pilot Study Of Sirolimus Plus Multiagent Chemotherapy For Relapsed/Refractory Acute Lymphoblastic Leukemia/Lymphoma

Terminated·—·Phase 1·NCT01658007·3 enrolled

Renal Allograft Recipients2 trials

Randomized Conversion of Calcineurin-Inhibitors in Renal Allograft Recipients

Completed·NNorthwestern University·Phase 4·NCT00866879·275 enrolled

Study Comparing Two Immunosuppressive Regimens in De Novo Renal Allograft Recipients

Completed· Pfizer logo

Pfizer·Phase 4·NCT00261820·160 enrolled

Skin Cancer2 trials

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

Terminated·—·Phase 4·NCT01797315·40 enrolled

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

Terminated·—·Phase 4·NCT00866684·44 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

Bladder Cancer2 trials

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

Sirolimus, Cisplatin, and Gemcitabine Hydrochloride in Treating Patients With Bladder Cancer

Completed·UUniversity of Washington·Phase 1/2·NCT01938573·21 enrolled

Type 1 Diabetes2 trials

Raptiva and Sirolimus in Islet Transplantation for Type 1 Diabetes

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

Islet Transplantation for Type 1 Diabetes

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT00014911·36 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

Chronic Graft Versus Host Disease2 trials

An Open Label Phase II Trial of Topical Sirolimus for the Treatment of Refractory Oral Chronic Graft-versus-Host-Disease

Terminated·—·Phase 2·NCT02123966·10 enrolled

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

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

Epilepsy2 trials

Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study

Recruiting·DDarcy Krueger·Phase 2·NCT05104983·64 enrolled

Sirolomus Adjuvant Treatment of Focal Refractory Epilepsy (SATFRE)

Recruiting·XXuanwu Hospital, Beijing·Phase 1·NCT06719791·15 enrolled

Chronic Granulomatous Disease2 trials

Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide

Terminated·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1/2·NCT02282904·7 enrolled

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

Aplastic Anemia2 trials

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

Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia

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

Acute Graft Versus Host Disease2 trials

A Study Using Tacrolimus, Sirolimus and Bortezomib as Acute Graft Versus Host Disease (GVHD) Prophylaxis in Allogeneic Peripheral Blood Stem Cell (PBSC) Transplantation

Completed·—·Phase 1·NCT00670423·27 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

Anemia2 trials

Haploidentical PBMC Transplant for Severe Congenital Anemias

Active, not recruiting·NNational Heart, Lung, and Blood Institute (NHLBI)·Phase 1/2·NCT00977691·23 enrolled

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

Transplant Recipient1 trial

Study Evaluating Rapamune in Transplant Recipients in the Nordic Countries

Completed· Pfizer logo

Pfizer··NCT00195195·66 enrolled

Idiopathic Pulmonary Fibrosis1 trial

Double-blind Placebo-controlled Pilot Study of Sirolimus in Idiopathic Pulmonary Fibrosis (IPF)

Completed·UUniversity of Virginia·N/A·NCT01462006·32 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

Leigh syndrome1 trial

Sirolimus for Leigh Syndrome

Enrolling by invitation·MMatthew Demczko·Phase 2·NCT06843811·15 enrolled

Ketamine1 trial

Examining the Impact of Sirolimus on Ketamine's Antidepressant Effects

Completed·YYale University·Phase 2·NCT02487485·23 enrolled

SLE1 trial

Omental Islet Transplant

Completed·UUniversity of Alberta·Phase 1/2·NCT02821026·4 enrolled

Venous malformation1 trial

Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations

Unknown status·—·Phase 2·NCT02042326·50 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

Ovarian Cancer1 trial

Auranofin and Sirolimus in Treating Participants With Ovarian Cancer

Terminated·MMayo Clinic·Phase 2·NCT03456700·22 enrolled

Memory1 trial

Provision of TCRγδ T Cells and Memory T Cells Plus Selected Use of Blinatumomab in Naïve T-cell Depleted Haploidentical Donor Hematopoietic Cell Transplantation for Hematologic Malignancies Relapsed or Refractory Despite Prior Transplantation

Active, not recruiting·SSt. Jude Children's Research Hospital·Phase 2·NCT02790515·170 enrolled

Age-Related Macular Degeneration1 trial

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

Kaposi's sarcoma1 trial

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

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

Cardiac Transplant1 trial

The Effect and Safety Profile of Thymoglobulin® in Primary Cardiac Transplant Recipients

Enrolling by invitation·—·Phase 2·NCT03292861·60 enrolled

Newly Diagnosed1 trial

Sirolimus Treatment for Newly Diagnosed Primary Acquired PRCA

Completed·BBing Han·Phase 4·NCT04470804·56 enrolled

Nosebleed1 trial

Sirolimus for Nosebleeds in HHT

Completed·UUnity Health Toronto·Phase 2·NCT05269849·10 enrolled

Unresectable Solid Tumor1 trial

Sirolimus in Treating Patients With Metastatic or Unresectable Solid Tumors

Completed·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 1·NCT00368914·30 enrolled

Refractory1 trial

Sirolimus in the Treatment of Refractory/Relapsed wAIHA

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

Fed1 trial

Bioavailability Study of Sirolimus Tablets 2 mg Under Fed Condition

Completed·—·Phase 1·NCT02296762·36 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

COVID-191 trial

Efficacy and Safety of Sirolimus in COVID-19 Infection

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

Vascular Malformation1 trial

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

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

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

MPR1 trial

Sirolimus for Improving Social Abilities in People With PTEN Germline Mutations

Withdrawn·SStanford University·Phase 1/2·NCT06080165

Terminated·UUniversity of Maryland, Baltimore·Phase 4·NCT00931255·32 enrolled

Diabetic Macular Edema1 trial

Sirolimus to Treat Diabetic Macular Edema

Completed·NNational Eye Institute (NEI)·Phase 1/2·NCT00711490·5 enrolled

Sturge-Weber syndrome1 trial

Trial of Sirolimus for Cognitive Impairment in Sturge-Weber Syndrome

Completed·—·Phase 2/3·NCT03047980·10 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

TED1 trial

Sirolimus Effect on Hypertrophic Syndromes Related Gene PIK3CA

Completed·—·Phase 2·NCT02443818·16 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

Thalassemia1 trial

Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia

Recruiting·TThe Hospital for Sick Children·Phase 1/2·NCT05426252·20 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

Neurodevelopment1 trial

Gene Replacement Therapy for Treatment of Paediatric Patients With CTNNB1 Neurodevelopmental Syndrome

Recruiting·CCTNNB1 Foundation·Phase 1/2·NCT07270549·12 enrolled

Pneumonia1 trial

Sirolimus Treatment in Hospitalized Patients With COVID-19 Pneumonia

Completed·UUniversity of Cincinnati·Phase 2·NCT04341675·32 enrolled

Sarcoidosis1 trial

Sirolimus in Cutaneous Sarcoidosis

Not yet recruiting·AAssistance Publique - Hôpitaux de Paris·Phase 2·NCT05458492·10 enrolled

Scleritis1 trial

Sirolimus Injections for Autoimmune Scleritis

Completed·NNational Eye Institute (NEI)·Phase 1/2·NCT01517074·5 enrolled

Chronic Lymphocytic Leukemia1 trial

Donor Stem Cell Transplant in Treating Patients With High-Risk Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma

Completed·AAlliance for Clinical Trials in Oncology·Phase 2·NCT01027000·68 enrolled

Immunodeficiency1 trial

Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders

Terminated·FFred Hutchinson Cancer Center·Phase 2·NCT00358657·14 enrolled

Homeostasis1 trial

Tolerance by Engaging Antigen During Cellular Homeostasis

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1·NCT03504241·8 enrolled

Single Dose1 trial

A Study to Evaluate the Effect of Multiple Doses of Isavuconazole on the Pharmacokinetics of a Single Dose of Sirolimus in Healthy Subjects

Completed· Astellas logo

Astellas·Phase 1·NCT01809860·22 enrolled

Lung Transplantation1 trial

Comparison of Sirolimus and Azathioprine in Lung Transplantation

Completed·UUniversity of Chicago·Phase 4·NCT00321906·181 enrolled

Antiphospholipid Syndrome1 trial

The Safety and Efficiency of Sirolimus in Primary Antiphospholipid Syndrome: a Randomized Control Study

Not yet recruiting·PPeking University People's Hospital·Phase 2·NCT06504420·70 enrolled

Randomized1 trial

Hemorrhagic Brainstem Cavernous Malformations Treatment With Sirolimus: aSingle Centre, Randomized, Placebo-controlled Pilot Trial

Recruiting·HHuashan Hospital·Phase 2·NCT06091332·75 enrolled

AMD1 trial

Sirolimus in Conjunction With Eylea vs Eylea Alone for Exudative AMD

Completed·MMaturi, Raj K., M.D., P.C.·Phase 2·NCT02732899·20 enrolled

Kidney Cancer1 trial

Low-Intensity Stem Cell Transplantation With Multiple Lymphocyte Infusions to Treat Advanced Kidney Cancer

Completed·NNational Cancer Institute (NCI)·Phase 2·NCT00923845·25 enrolled

Acute Myeloid Leukemia1 trial

Sirolimus, Idarubicin, and Cytarabine in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia

Completed·SSidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University·Phase 1·NCT01822015·55 enrolled

Survival1 trial

Strategies to Improve Islet Survival

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT00464555·5 enrolled

Smokers1 trial

Enhancing Self Regulation Among Smokers

Completed·MMedical University of South Carolina·Phase 2·NCT04161144·22 enrolled

Renal Transplantation1 trial

Research Study of ATG and Rituximab in Renal Transplantation

Terminated·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1·NCT01318915·10 enrolled

Fibrosis1 trial

Fibrosis in Renal Allografts

Unknown status·UUniversity Hospital, Antwerp·Phase 4·NCT00493194·100 enrolled

Geographic Atrophy1 trial

Intravitreal Injections of Sirolimus in the Treatment of Geographic Atrophy

Terminated·NNational Eye Institute (NEI)·Phase 2·NCT01675947·52 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

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

Hemoglobinopathies1 trial

Allo HSCT for High Risk Hemoglobinopathies

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

Dilated Cardiomyopathy1 trial

The Effects of Sirolimus in Patients With Dilated Cardiomyopathy Infected With Kaposi Sarcoma-associated Virus

Recruiting·TTongji Hospital·Phase 4·NCT06236022·276 enrolled

Bone Marrow Transplantation1 trial

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

Chemotherapy1 trial

A Pilot Study to Determine the Safety and Tolerability of Sirolimus Given With Hyper-CVAD Chemotherapy

Completed·SSidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University·Phase 1·NCT01184885·7 enrolled

Nephropathy1 trial

Using mTOR Inhibitors in the Prevention of BK Nephropathy

Completed·—·N/A·NCT01649609·40 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

Systemic Sclerosis1 trial

A Pilot-Study of Sirolimus for the Treatment of Systemic Sclerosis

Unknown status·PPeking University People's Hospital·Phase 2·NCT03365869·72 enrolled

Multiple Sclerosis1 trial

Treating Multiple Sclerosis With Sirolimus, an Immune System Suppressor

Terminated·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1/2·NCT00095329·14 enrolled

Alzheimer's Disease1 trial

Evaluating Rapamycin Treatment in Alzheimer's Disease Using Positron Emission Tomography

Completed·KKarolinska Institutet·Phase 1/2·NCT06022068·14 enrolled

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

Tolerance1 trial

Belatacept Post Depletional Repopulation to Facilitate Tolerance

Completed·—·Phase 2·NCT00565773·40 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

Myelofibrosis1 trial

Ruxolitinib Phosphate and Chemotherapy Given Before and After Reduced Intensity Donor Stem Cell Transplant in Treating Patients With Myelofibrosis

Completed·—·Phase 1·NCT02917096·18 enrolled

Induction Therapy1 trial

A Study To Compare Sirolimus Versus Tacrolimus In De Novo Simultaneous Pancreas- Kidney Allograft Recipients Receiving An Induction Therapy With Antithymocyte Globulin Plus Mycophenolate Mofetil Plus Corticosteroids

Unknown status·NNantes University Hospital·Phase 4·NCT00693446·118 enrolled

Solid Tumors1 trial

Aflac ST0901 CHOANOME - Sirolimus in Solid Tumors

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

Lymphoma1 trial

Tandem Auto-Allo Transplant for Lymphoma

Completed·MMassachusetts General Hospital·Phase 2·NCT01181271·42 enrolled

Myelodysplastic Syndrome1 trial

Sirolimus and Azacitidine in Treating Patients With High Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia That is Recurrent or Not Eligible for Intensive Chemotherapy

Completed·SSidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University·Phase 2·NCT01869114·57 enrolled

Germ Cell Tumor1 trial

A Phase II Study of Sirolimus and Erlotinib in Recurrent/Refractory Germ Cell Tumors

Terminated·TTheodore Laetsch·Phase 2·NCT01962896·4 enrolled

Focal Segmental Glomerulosclerosis1 trial

Sirolimus for Focal Segmental Glomerulosclerosis

Completed·NNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)·Phase 2·NCT00040508·30 enrolled

Advanced Malignancies1 trial

Sirolimus and Cetuximab in Advanced Malignancies

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

Prostate Cancer1 trial

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

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

Hyperinsulinism1 trial

Sirolimus for the Treatment of Hyperinsulinism

Withdrawn·—·Phase 1/2·NCT02524639

Pancreatic Cancer1 trial

Study of Sirolimus in Patients With Advanced Pancreatic Cancer

Unknown status·SSecond Affiliated Hospital, School of Medicine, Zhejiang University·Phase 1/2·NCT03662412·36 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

Unspecified5 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

Sirolimus in Graves' Orbitopathy

Completed·UUniversity of Pisa··NCT05345119·30 enrolled

Show 2 more trials

Optimizing PTCy Dose and Timing

Active, not recruiting·NNational Cancer Institute (NCI)·Phase 1/2·NCT03983850·105 enrolled

Study of Sirolimus in CTD-TP in China

Unknown status·PPeking Union Medical College Hospital·Phase 4·NCT03688191·20 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 — 427 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

Q4 2026

Q2 2027

Trials by Indication[1]

Kidney Transplant21 trials

Graft Versus Host Disease7 trials

Treatment5 trials

Hematologic Malignancies4 trials

Non-Small Cell Lung Cancer4 trials

Stem Cell Transplantation4 trials

Liver Transplant3 trials

Renal Transplant Recipients3 trials

Sickle Cell Disease3 trials

Stem Cell Transplant3 trials

Hepatocellular Carcinoma3 trials

Prophylaxis3 trials

Immunosuppression3 trials

Malignant Peripheral Nerve Sheath Tumor3 trials

Efficacy and Safety3 trials

Safety and Efficacy3 trials

Stent Restenosis2 trials

Lymphangioleiomyomatosis2 trials

Multiple Myeloma2 trials

Transplantation2 trials

Acute Lymphoblastic Leukemia2 trials

Renal Allograft Recipients2 trials

Skin Cancer2 trials

Allograft2 trials

Bladder Cancer2 trials

Type 1 Diabetes2 trials

Hematopoietic Stem Cell Transplantation2 trials

Chronic Graft Versus Host Disease2 trials

Epilepsy2 trials

Chronic Granulomatous Disease2 trials

Aplastic Anemia2 trials

Acute Graft Versus Host Disease2 trials

Anemia2 trials

Transplant Recipient1 trial

Idiopathic Pulmonary Fibrosis1 trial

Basal Cell Nevus Syndrome1 trial

Leigh syndrome1 trial

Ketamine1 trial

SLE1 trial

Venous malformation1 trial

Renal Cell Carcinoma1 trial

Ovarian Cancer1 trial

Memory1 trial

Age-Related Macular Degeneration1 trial

Kaposi's sarcoma1 trial

Cardiac Transplant1 trial

Newly Diagnosed1 trial

Nosebleed1 trial

Unresectable Solid Tumor1 trial

Refractory1 trial

Leukemia1 trial

Fed1 trial

Withdrawal1 trial

Peripheral Blood Stem Cell Transplantation1 trial

Renal Transplant1 trial

COVID-191 trial

Vascular Malformation1 trial

Primary Peritoneal Cancer1 trial

Refractory Solid Tumors1 trial

Adult1 trial

MPR1 trial

Islet Transplantation1 trial

Head and Neck Squamous Cell Carcinoma1 trial

Delayed Graft Function1 trial

Diabetic Macular Edema1 trial

Sturge-Weber syndrome1 trial

Growth1 trial

TED1 trial

Patients With Solid Tumors1 trial

Thalassemia1 trial

B-cell NHL1 trial

Allogeneic Stem Cell Transplantation1 trial

Neurodevelopment1 trial