Rapamycin

ApprovedSmall molecule

MMaastricht Radiation Oncology

Rapamycin is one of 720 small molecule programs targeting Angiofibromas. It is currently in Approved.

Indications

Total Trials

Active Trials

719

Competitors

Pipeline

Status by Indication[3][1]

Indication

AngiofibromasActive, not recruiting

HydrocephalusActive, not recruiting

Familial Adenomatous PolyposisActive, not recruiting

Kidney AngiomyolipomaActive, not recruiting

NeuroblastomaActive, not recruiting

StrictureActive, not recruiting

Uterine FibroidActive, not recruiting

Kidney TransplantActive, not recruiting

Liver CancerActive, not recruiting

Weight LossActive, not recruiting

AngiofibromasPhase 2

Active, not recruiting

HydrocephalusPhase 2

Active, not recruiting

Familial Adenomatous PolyposisPhase 2

Active, not recruiting

Kidney AngiomyolipomaPhase 2

Active, not recruiting

NeuroblastomaPhase 2

Active, not recruiting

StricturePreclinical

Active, not recruiting

Uterine FibroidApproved

Active, not recruiting

Kidney TransplantPhase 2

Active, not recruiting

Liver CancerPhase 1

Active, not recruiting

Weight LossPhase 2/Phase 3

Active, not recruiting

Showing 10 of 32 indications

Competition

Competitive Landscape

2828 competitors in Breast Cancer

#DrugCompanyPhaseTrials

Rapamycin

MMaastricht Radiation Oncology

Approved

3 2

Halaven

Eisai

Preclinical

38 3

Trodelvy

Gilead

Preclinical

37 4

Megace

Bristol-Myers Squibb

Preclinical

4 5

Cymbalta

Alnylam Pharmaceuticals

Preclinical

3 6

Lexiscan

Astellas

Preclinical

1 7

Adenocard

Astellas

Preclinical

1 8

Methylthioninium Chloride

PProvepharm SAS

Preclinical

3 9

Zinecard

CChildren's Oncology Group

Preclinical

2 10

Chirocaine

PPurdue Pharma LP

Preclinical

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

Timeline

Expected Milestones

Q3 2026

Phase 2Active, not recruiting

Ovarian

NCT05836025

Q1 2027

Phase 3Not yet recruiting

Breast Cancer

NCT04736589

Q4 2027

Phase 2Not yet recruiting

Port Wine Stains

NCT07436611

Phase 2Active, not recruiting

Vitiligo

NCT05342519

Q3 2028

Phase 1Recruiting

Adult

NCT06658093

Clinical

Trials by Indication[1]

Advanced Cancers4 trials

Interaction Study of Rapamycin and Sunitinib in Patients With Advanced Cancers

Completed·UUniversity of Chicago·Phase 1·NCT00583063·23 enrolled

Study of Rapamycin Plus Ketoconazole in Advanced Cancers

Completed·UUniversity of Chicago·Phase 1·NCT00708591·57 enrolled

Rapamycin in Advanced Cancers

Completed·UUniversity of Chicago·Phase 1·NCT00707135·40 enrolled

Show 1 more trial

Rapamycin Plus Bevacizumab in Advanced Cancers

Completed·UUniversity of Chicago·Phase 1·NCT00667485·28 enrolled

Tuberous Sclerosis4 trials

Topical Rapamycin and Calcitriol for Angiofibroma of Tuberous Sclerosis

Completed·NNational Taiwan University Hospital·Phase 3·NCT03140449·52 enrolled

Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants

Recruiting·KKatarzyna Kotulska·Phase 2/3·NCT04987463·60 enrolled

Dose-Ranging Efficacy and Safety Study of Topical Rapamycin Cream for Facial Angiofibroma Associated With Tuberous Sclerosis Complex

Completed·DDermatology Specialties Limited Partnership·Phase 2/3·NCT03826628·107 enrolled

Show 1 more trial

Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

Completed·—·Phase 2·NCT00457808·25 enrolled

Breast Cancer3 trials

Aging Mammary Stem Cells and Breast Cancer Prevention

Terminated·LLuZhe Sun·Phase 2·NCT02642094·58 enrolled

A Phase II Study of Rapamycin and Trastuzumab for Patients With HER-2 Receptor Positive Metastatic Breast Cancer

Completed·YYale University·Phase 2·NCT00411788·11 enrolled

Inetetamab Plus Rapamycin and Chemotherapy for HER2+ Metastatic Breast Cancer With Abnormal Activation of PAM Pathway

Not yet recruiting·PPeking Union Medical College·Phase 3·NCT04736589·270 enrolled

Pancreatic Cancer2 trials

Individualized Drug Treatment for Treating Patients With Pancreatic Cancer

Terminated·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 2·NCT00276744·249 enrolled

A Study of Metformin With or Without Rapamycin as Maintenance Therapy After Induction Chemotherapy in Subjects With Pancreatic Cancer

Completed·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 1·NCT02048384·22 enrolled

Acute Myeloid Leukemia2 trials

Rapamycin in Combination With Low-dose Aracytin in Elderly Acute Myeloid Leukemia Patients

Completed·UUniversity Hospital, Toulouse·Phase 2·NCT00235560·50 enrolled

A Study of Decitabine in Combination With Escalating Doses of Rapamycin in Patients With Relapsed or Refractory Acute Myeloid Leukemia

Completed·UUniversity of Rochester·Phase 1·NCT00861874·13 enrolled

67 more indications

Kidney Transplant2 trials

TUMORAPA 1: Efficacy of Rapamycin in Secondary Prevention of Skin Cancers in Kidney Transplant Recipients

Completed·HHospices Civils de Lyon·Phase 3·NCT00133887·77 enrolled

Rapamycin and Regulatory T Cells in Kidney Transplantation

Completed·FFondazione IRCCS Policlinico San Matteo di Pavia·Phase 2·NCT01014234·56 enrolled

Acute Lymphoblastic Leukemia2 trials

Rapamycin for Immunosuppression and B Cell Modulation Post Stem Cell Transplant for Acute Lymphoblastic Leukemia (ALL)

Completed·UUniversity of Utah·Phase 2·NCT00795886·63 enrolled

Rapamycin in Relapsed Acute Lymphoblastic Leukemia

Completed·—·Phase 1·NCT00874562·6 enrolled

Advanced Malignancies2 trials

Drug Interaction Study of Sorafenib and Rapamycin in Advanced Malignancies

Completed·UUniversity of Chicago·Phase 1·NCT00449280·34 enrolled

Rapamycin With Grapefruit Juice for Advanced Malignancies

Completed·UUniversity of Chicago·Phase 1·NCT00375245·41 enrolled

Type 1 Diabetes2 trials

Proleukin and Rapamune in Type 1 Diabetes

Completed·NNational Institute of Allergy and Infectious Diseases (NIAID)·Phase 1·NCT00525889·9 enrolled

Monotherapy With Rapamycin in Long-standing Type 1 Diabetes

Completed·PPiemonti Lorenzo·Phase 2·NCT02803892·55 enrolled

Chronic Graft Versus Host Disease2 trials

Donor Regulatory T Cells Infusion in Patients With Chronic Graft-versus-host Disease (GVHD)

Terminated·UUniversity of Liege·Phase 2·NCT01903473·19 enrolled

Rapamycin for Prevention of Chronic Graft-Versus-Host Disease

Terminated·YYale University·Phase 1/2·NCT00623012·2 enrolled

Treatment2 trials

Rapamycin Treatment for Activated Phosphoinositide 3-Kinase δ Syndrome

Completed·—·Phase 1/2·NCT03383380·30 enrolled

Rapamycin Treatment for ALS

Completed·AAzienda Ospedaliero-Universitaria di Modena·Phase 2·NCT03359538·63 enrolled

Metabolism2 trials

Effect of mTOR Inhibition & Other Metabolism Modulating Interventions on the Elderly [SubStudy Rapa & cMRI to Evaluate Cardiac Function]

Completed·TThe University of Texas Health Science Center at San Antonio·Phase 2·NCT04742777·20 enrolled

Effect of mTOR Inhibition and Other Metabolism Modulating Interventions on the Elderly

Completed·TThe University of Texas Health Science Center at San Antonio·Phase 2·NCT02874924·34 enrolled

Renal Transplant Recipients1 trial

Pilot Study to Investigate a Steroid Free Immunosuppressive Regimen for Renal Transplant Recipients

Completed·UUniversity Hospital, Basel, Switzerland·Phase 4·NCT00306397·100 enrolled

Longevity1 trial

Participatory Evaluation (of) Aging (With) Rapamycin (for) Longevity Study

Completed·AAgelessRx·Phase 2·NCT04488601·129 enrolled

Hydrocephalus1 trial

The Safety and Efficacy of Rapamycin on Communicating Hydrocephalus Secondary to Intraventricular Hemorrhage

Recruiting·—·Phase 2·NCT06563817·53 enrolled

Recurrence1 trial

Adjuvant Anti-Recurrence Treatment With Rapamycin on Early-stage HCC

Completed·—·Phase 1·NCT02724332·300 enrolled

Immune Thrombocytopenia1 trial

Efficacy of Rapamycin Therapy With Chronic Immune Thrombocytopenia

Completed·—··NCT01672151·35 enrolled

MPR1 trial

Combination Gerotherapeutic Interventions for Healthspan Improvement

Active, not recruiting·AAgelessRx·Phase 3·NCT07475546·30 enrolled

Pompe Disease1 trial

Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease

Completed·UUniversity of Florida·Phase 1·NCT02240407·2 enrolled

Kidney Angiomyolipoma1 trial

RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS

Completed·—·Phase 2·NCT00126672·36 enrolled

Completed·OOspedale San Raffaele·Phase 2·NCT02505893·6 enrolled

Inclusion Body Myositis1 trial

Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis

Completed·IInstitut National de la Santé Et de la Recherche Médicale, France·Phase 2/3·NCT02481453·44 enrolled

Molecular Targeted Therapy1 trial

Prospective Pilot Trial to Assess a Multimodal Molecular Targeted Therapy in Children, Adolescent and Young Adults With Relapsed or Refractory High-grade Pineoblastoma

Completed·UUniversity of Regensburg·Phase 2·NCT02596828·4 enrolled

B-cell Malignancies1 trial

A Phase 1 Study of UB-VV111 With and Without Rapamycin in Relapsed/Refractory CD19+ B-cell Malignancies

Recruiting·UUmoja Biopharma·Phase 1·NCT06528301·106 enrolled

Weight loss1 trial

Effectiveness of Alternative Therapies in Maintaining Weight Loss Achieved by GLP-1 Medications Post-Cessation

Recruiting·AAgelessRx·Phase 2/3·NCT07092618·150 enrolled

Methotrexate1 trial

Rapamycin vs Methotrexate in Diffuse SSc

Completed·UUniversity of California, Los Angeles·Phase 1/2·NCT00241189·17 enrolled

Neuroblastoma1 trial

Multimodal Molecular Targeted Therapy to Treat Relapsed or Refractory High-risk Neuroblastoma

Completed·UUniversity of Regensburg·Phase 2·NCT01467986·130 enrolled

Insulin Sensitivity1 trial

mTOR as Mediator of Insulin Sensitivity Study

Recruiting·UUniversity of Copenhagen·N/A·NCT05233722·10 enrolled

Pregnancy1 trial

Immunomodulatory Effects of Rapamycin on Pregnancy Rate of Patient With Recurrent Implantation Failure

Completed·MMehdi Yousefi·Phase 2·NCT03161340·121 enrolled

Sjögren's Syndrome1 trial

Low-dose Interleukin-2 and Rapamycin on sjögren's Syndrome

Completed·PPeking University People's Hospital·Phase 1/2·NCT05605665·30 enrolled

Neoadjuvant1 trial

Neoadjuvant Rapamycin in Patients Undergoing Radical Cystectomy

Completed·TThe University of Texas Health Science Center at San Antonio·Phase 1·NCT01827618·29 enrolled

Kidney1 trial

Rapamycin Versus Mycophenolate Mofetil in Kidney-Pancreas Recipients

Completed·UUniversity of Miami·Phase 2·NCT00533442·170 enrolled

Patients With Advanced Solid Tumors1 trial

Study of Combination of Sirolimus and Sutent in Patients With Advanced Solid Tumors Non-Curable With Standard Therapy

Terminated·YYale University·Phase 1·NCT00402415·18 enrolled

Liver Cancer1 trial

Sirolimus and Bevacizumab in Treating Patients With Liver Cancer That Cannot Be Removed by Surgery

Completed·NNational Cancer Centre, Singapore·Phase 1·NCT00467194·27 enrolled

Alcohol Use Disorders1 trial

Altering Memories That Increase Risk of Relapse in Alcohol Use Disorders

Completed·MMedical University of South Carolina·Phase 1·NCT03732248·21 enrolled

Peutz-Jeghers syndrome1 trial

Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome

Unknown status·PPeking Union Medical College Hospital·Phase 4·NCT03781050·10 enrolled

Rectum Cancer1 trial

Safety Study of Rapamycin Administered Before and During Radiotherapy to Treat Rectum Cancer

Completed·MMaastricht Radiation Oncology·Phase 1/2·NCT00409994·44 enrolled

COVID-191 trial

Effects of mTOR Inhibition With Sirolimus (RAPA) in Patients With COVID-19 to Moderate the Progression of ARDS

Withdrawn·TThe University of Texas Health Science Center at San Antonio·Phase 1/2·NCT04482712

Familial adenomatous polyposis1 trial

Safety Study for the Use of Rapamycin in Children With Familial Adenomatous Polyposis

Not yet recruiting·UUniversity Hospital, Toulouse·Phase 2·NCT06308445·25 enrolled

Function1 trial

Renal Function Optimization With Mycophenolate Mofetil (MMF) Immunosuppressor Regimes (ALHAMBRA)

Unknown status·SSociedad Andaluza de Trasplantes de Organos y Tejidos·Phase 4·NCT00290069·94 enrolled

Vitiligo1 trial

Daily Topical Rapamycin for Vitiligo

Active, not recruiting·MMedical University of South Carolina·Phase 2·NCT05342519·20 enrolled

Adult1 trial

RESTOR: PK/PD mTORi Inhibition in Older Adults

Recruiting·TThe University of Texas Health Science Center at San Antonio·Phase 1·NCT06658093·194 enrolled

Dexamethasone1 trial

Cyclophosphamide and Pulse Dexamethasone With Rapamycin or Hydroxychloroquine

Completed·—·Phase 1·NCT01396200·6 enrolled

Systemic Inflammation1 trial

A Study of Exercise and Pharmacologic Intervention on Systemic Inflammation

Recruiting·IIcahn School of Medicine at Mount Sinai·Phase 1·NCT07058974·22 enrolled

Rehabilitation1 trial

Exercise and Low-Dose Rapamycin in Older Adults With CAD:Cardiac Rehabilitation And Rapamycin in Elderly (CARE) Trial

Completed·MMayo Clinic·Phase 1·NCT01649960·13 enrolled

Lymphoid Malignancies1 trial

Rapamycin in With High-Dose Etoposide and Cytarabine in Relapsed/Refractory Aggressive Lymphoid Malignancies

Terminated·—·Phase 1/2·NCT00776373·4 enrolled

Bladder Cancer1 trial

Secondary Prevention Trial of Rapamycin in Patients With Resected Non-muscle Invasive Bladder Cancer

Withdrawn·TThe University of Texas Health Science Center at San Antonio·Phase 3·NCT03298958

Chronic Condition1 trial

Low Dose Rapamycin in ME/CFS, Long-COVID, and Other Infection Associated Chronic Conditions

Enrolling by invitation·SSimmaron Research Inc.··NCT06257420·150 enrolled

Ovarian1 trial

Effect of Rapamycin in Ovarian Aging

Active, not recruiting·—·Phase 2·NCT05836025·50 enrolled

Uterine Fibroid1 trial

Efficacy and Safety of Rapamycin (Sirolimus) in the Treatment of Symptomatic Uterine Fibroids and Leiomyomatosis

Unknown status·PPeking Union Medical College Hospital·Phase 4·NCT03500367·25 enrolled

Hematopoietic Cell Transplantation1 trial

Graft-Versus-Host Disease (GVHD) Prophylaxis After Allogeneic Peripheral Blood Hematopoietic Cell Transplantation

Completed·HH. Lee Moffitt Cancer Center and Research Institute·Phase 2·NCT00803010·74 enrolled

Growth1 trial

Nutritional and Contractile Regulation of Muscle Growth

Completed·TThe University of Texas Medical Branch, Galveston·Phase 1·NCT00891696·144 enrolled

Glioblastoma Multiforme1 trial

Sirolimus in Treating Patients With Glioblastoma Multiforme

Completed·JJonsson Comprehensive Cancer Center·Phase 1/2·NCT00047073·13 enrolled

Aging1 trial

The Bioavailability of Compounded and Generic Rapamycin in Normative Aging Individuals

Completed·AAgelessRx··NCT06550271·67 enrolled

Islet Transplantation1 trial

Pre Transplant Rapamycin Treatment in Islet Transplantation Alone

Completed·IIRCCS San Raffaele·Phase 3·NCT01060605·11 enrolled

Venous malformation1 trial

Efficacy of Rapamycin (Sirolimus) in the Treatment of BRBNS, Hereditary or Sporadic Venous Malformation

Unknown status·PPeking Union Medical College Hospital·Phase 4·NCT03767660·20 enrolled

ESS1 trial

An Open, Single Centre, Randomised, Parallel Group Study to Investigate Three Different Immunosuppressive Regimens

Completed·UUniversity Hospital, Basel, Switzerland·Phase 4·NCT01183247·63 enrolled

Methylprednisolone1 trial

Rapamycin Plus Methylprednisolone Versus Methylprednisolone Alone in Active, Moderate-to-severe Graves' Orbitopathy.

Unknown status·FFirst Affiliated Hospital Xi'an Jiaotong University·Phase 1·NCT05532072·140 enrolled

Autosomal Dominant Polycystic Kidney Disease1 trial

Rapamycin as Treatment for Autosomal Dominant Polycystic Kidney Disease (ADPKD): The Role of Biomarkers in Predicting a Response to Therapy

Terminated·YYale University·Phase 2/3·NCT00920309·21 enrolled

Relapsed or Refractory B-cell Malignancies1 trial

UB-VV400 in Combination With Rapamycin in Relapsed or Refractory B-cell Malignancies

Recruiting·NNanjing IASO Biotechnology Co., Ltd.·Phase 1·NCT06743503·70 enrolled

Prospective1 trial

Prospective Study of Rapamycin for the Treatment of SLE

Completed·SState University of New York - Upstate Medical University·Phase 2·NCT00779194·99 enrolled

Lymphatic Malformation1 trial

Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations

Recruiting·UUniversity Hospital, Lille·Phase 2·NCT03243019·28 enrolled

Graft Failure1 trial

Mycophenolate Mofetil and Rapamycin as Secondary Intervention vs. Continuation of Calcineurin Inhibitors in Patients at Risk for Chronic Renal Allograft Failure

Completed·VVanderbilt University Medical Center·Phase 4·NCT00223678·20 enrolled

Prostate Cancer1 trial

Sirolimus Before Surgery in Treating Patients With Advanced Localized Prostate Cancer

Completed·SSidney Kimmel Comprehensive Cancer Center at Johns Hopkins·Phase 1/2·NCT00311623·32 enrolled

Inflammation1 trial

Topical-RAPA Use in Inflammation Reversal and Re-setting the Epigenetic Clock

Completed·TThe University of Texas Health Science Center at San Antonio·Phase 1·NCT04608448·22 enrolled

Age-Related Macular Degeneration1 trial

Infliximab, Sirolimus and Daclizumab to Treat Age-Related Macular Degeneration

Completed·NNational Eye Institute (NEI)·Phase 2·NCT00304954·13 enrolled

Pilot Study1 trial

A Pilot Study on Reverse Aging (The REVERSE Study)

Active, not recruiting·TThe Christ Hospital·Phase 1·NCT07354620·52 enrolled

Port Wine Stains1 trial

Rapamycin Dose-Ranging Efficacy Study: A Phase II, Proof of Concept, Double-blind, Placebo-controlled, Randomized, Parallel-group, Dose-response Comparison of the Effects of Different Strengths of Rapamycin Cream Applied Topically in Subjects Diagnosed With Port Wine Stains

Not yet recruiting·AAFT Pharmaceuticals, Ltd.·Phase 2·NCT07436611·30 enrolled

Unspecified3 trials

Novel Compositions for Treating or Preventing Dermal Disorders

Completed·—·Phase 1/2·NCT03103893·36 enrolled

Rapamycin - Effects on Alzheimer's and Cognitive Health

Recruiting·TThe University of Texas Health Science Center at San Antonio·Phase 2·NCT04629495·40 enrolled

Topical Rapamycin for Fibrofolliculomas

Completed·MMaastricht University Medical Center·Phase 3·NCT00928798·19 enrolled

Sources

References

[1]
ClinicalTrials.gov — 91 trial records for Rapamycin. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Rapamycin compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Rapamycin. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Maastricht Radiation Oncology company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Approved[1]
Company: MMaastricht Radiation Oncology

Rapamycin

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q3 2026

Q1 2027

Q4 2027

Q3 2028

Trials by Indication[1]

Advanced Cancers4 trials

Tuberous Sclerosis4 trials

Breast Cancer3 trials

Pancreatic Cancer2 trials

Acute Myeloid Leukemia2 trials

Kidney Transplant2 trials

Acute Lymphoblastic Leukemia2 trials

Advanced Malignancies2 trials

Type 1 Diabetes2 trials

Chronic Graft Versus Host Disease2 trials

Treatment2 trials

Metabolism2 trials

Renal Transplant Recipients1 trial

Longevity1 trial

Hydrocephalus1 trial

Recurrence1 trial

Immune Thrombocytopenia1 trial

MPR1 trial

Pompe Disease1 trial

Kidney Angiomyolipoma1 trial

Memory1 trial

Stricture1 trial

Angiofibromas1 trial

Diabetes1 trial

Inclusion Body Myositis1 trial

Molecular Targeted Therapy1 trial

B-cell Malignancies1 trial

Weight loss1 trial

Methotrexate1 trial

Neuroblastoma1 trial

Insulin Sensitivity1 trial

Pregnancy1 trial

Sjögren's Syndrome1 trial

Neoadjuvant1 trial

Kidney1 trial

Patients With Advanced Solid Tumors1 trial

Liver Cancer1 trial

Alcohol Use Disorders1 trial

Peutz-Jeghers syndrome1 trial

Rectum Cancer1 trial

COVID-191 trial

Familial adenomatous polyposis1 trial

Function1 trial

Vitiligo1 trial

Adult1 trial

Dexamethasone1 trial

Systemic Inflammation1 trial

Rehabilitation1 trial

Lymphoid Malignancies1 trial

Bladder Cancer1 trial

Chronic Condition1 trial

Ovarian1 trial

Uterine Fibroid1 trial

Hematopoietic Cell Transplantation1 trial

Growth1 trial

Glioblastoma Multiforme1 trial

Aging1 trial

Islet Transplantation1 trial

Venous malformation1 trial

ESS1 trial

Methylprednisolone1 trial

Autosomal Dominant Polycystic Kidney Disease1 trial

Relapsed or Refractory B-cell Malignancies1 trial

Prospective1 trial

Lymphatic Malformation1 trial

Graft Failure1 trial

Prostate Cancer1 trial

Inflammation1 trial

Age-Related Macular Degeneration1 trial

Pilot Study1 trial

Port Wine Stains1 trial