127 completed, 30 terminated, 25 recruiting, 18 active not recruiting. 124 Phase 2, 80 Phase 1, 30 Phase 3, 17 Phase 4, 10 Early Phase 1.
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Ventricular mTOR Inhibition to Prevent Hydrocephalus After Brain Hemorrhage Sirolimus (Rapamune®) - Post-hemorrhagic Hydrocephalus (PHH) | Phase 1 / Phase 2 | Not yet recruiting | Massachusetts General Hospital | 15 | 2027-01-01 | 2026-06-25 |
| Total Marrow and Lymphoid Irradiation in Combination With Fludarabine and Melphalan as Conditioning for Allogeneic Peripheral Blood Stem Cell Hematopoietic Cell Transplant in Older Patients With Refractory and Relapsed Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome Biospecimen Collection, Bone Marrow Biopsy, Computed Tomography - Acute Myeloid Leukemia With Complex Karyotype, Myelodysplastic Chronic Myelomonocytic Leukemia | Phase 2 | Recruiting | City of Hope Medical Center | 35 | 2026-11-05 | 2026-10-08 |
| A Nutritional and Pharmacological Intervention for Body, Brain, and Longevity Effects (NIBBLE) FMD1 (LNT22-017-1), Rapamycin, Placebo - Cerebral Blood Flow, APOE 4 | Phase 1 / Phase 2 | Not yet recruiting | Cedars-Sinai Medical Center | 60 | 2026-11-01 | 2026-09-16 |
| Low-Dose Sirolimus for the Treatment of RUNX1 Familial Platelet Disorder Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy - Familial Platelet Disorder With Associated Myeloid Malignancy | Phase 1 | Recruiting | OHSU Knight Cancer Institute | 6 | 2026-10-05 | 2026-10-13 |
| Safety Study for the Use of Rapamycin in Children With Familial Adenomatous Polyposis Rapamycin - Familial Adenomatous Polyposis | Phase 2 | Not yet recruiting | University Hospital, Toulouse | 25 | 2026-09-01 | 2026-05-08 |
| Sirolimus Pre-conditioning on T Cell Activity and T-cell Engaging Bispecific Antibody Efficacy in Multiple Myeloma Sirolimus, Teclistamab, Talquetamb - Multiple Myeloma | Phase 1 | Recruiting | Christopher Strouse | 10 | 2026-06-01 | 2026-06-04 |
| Vaccination Response Modulation With a Targeted Rapamycin Protocol Study Sirolimus (RAPAMUNE), Influenza vaccine (Sequiris Fluad Quad), RSV vaccine (Pfizer Abrysvo) - Vaccine Immune Response, Haemodialysis Patients | Phase 2 | Not yet recruiting | Central Adelaide Local Health Network Incorporated | 60 | 2026-05-01 | 2026-05-14 |
| RESTOR: PK/PD mTORi Inhibition in Older Adults Rapamycin, Everolimus, Placebo - Aging | Early Phase 1 | Recruiting | The University of Texas Health Science Center at San Antonio | 194 | 2026-03-04 | 2026-06-01 |
| Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy - Aplastic Anemia | Phase 2 | Active not recruiting | Fred Hutchinson Cancer Center | 1 | 2025-09-25 | 2026-05-22 |
| A Study of Exercise and Pharmacologic Intervention on Systemic Inflammation lamivudine, rapamycin, Exercise Regimen - Smokers, Former Smokers | Phase 1 | Active not recruiting | Icahn School of Medicine at Mount Sinai | 20 | 2025-08-12 | 2026-08-27 |
| Combination Gerotherapeutic Interventions for Healthspan Improvement Rapamycin, Naltrexone, Metformin - Healthspan Improvement, Aging | Phase 3 | Completed | AgelessRx | 26 | 2025-07-23 | 2026-10-30 |
| 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 Actinium Ac 225-DOTA-Daratumumab, Biospecimen Collection, Bone Marrow Aspiration - Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia | Phase 1 | Recruiting | City of Hope Medical Center | 15 | 2025-07-01 | 2026-10-30 |
| 131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD) 131I-apamistmab, Sirolimus, Campath - Sickling Disorder Due to Hemoglobin S | Phase 1 | Recruiting | Columbia University | 24 | 2025-04-28 | 2026-06-10 |
| Low Dose Sirolimus in People With Post-Acute Sequelae of COVID-19 (PASC) Long COVID-19 Low-dose sirolimus, Placebo - Long COVID-19 | Phase 3 | Recruiting | Icahn School of Medicine at Mount Sinai | 80 | 2025-04-18 | 2026-05-18 |
| UB-VV400 in Combination With Rapamycin in Relapsed or Refractory B-cell Malignancies UB-VV400, Rapamycin - Large B-cell Lymphoma | Phase 1 | Recruiting | Nanjing IASO Biotechnology Co., Ltd. | 70 | 2025-04-11 | 2025-09-18 |
| A Phase 1 Study of UB-VV111 With and Without Rapamycin in Relapsed/Refractory CD19+ B-cell Malignancies UB-VV111, rapamycin - Lymphoma, B-Cell, Lymphoma, Non-Hodgkin (NHL) | Phase 1 | Recruiting | Umoja Biopharma | 106 | 2025-03-10 | 2026-10-15 |
| Sirolimus for Leigh Syndrome Sirolimus - Leigh Syndrome | Phase 2 | Enrolling by invitation | Matthew Demczko | 15 | 2025-02-27 | 2026-03-31 |
| Sirolimus Monotherapy in the Treatment of Antiphospholipid Antibody Related Thrombocytopenia Sirolimus, Placebo - Antiphospholipid (aPL)-Positive, Thrombocytopaenia | Phase 4 | Recruiting | Peking University First Hospital | 84 | 2025-01-07 | 2026-06-30 |
| Sirolimus Treatment Of Patients With SLE Sirolimus, Placebo - Lupus Erythematosus, Systemic | Phase 2 | Not yet recruiting | State University of New York - Upstate Medical University | 220 | 2025-01-01 | 2023-06-05 |
| Effectiveness of Alternative Therapies in Maintaining Weight Loss Achieved by GLP-1 Medications Post-Cessation Metformin, Rapamycin, Naltrexone - Longevity, GLP-1 | Phase 2 / Phase 3 | Recruiting | AgelessRx | 150 | 2024-12-17 | 2025-07-30 |
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Leukemia, Myeloid, Acute (19) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (18) · Myelodysplastic Syndromes (15) · Lymphoma, Non-Hodgkin (11) · Multiple Myeloma (11) · Leukemia, Lymphocytic, Chronic, B-Cell (10) · Hodgkin Disease (9) · Leukemia (8) · Leukemia, Myelogenous, Chronic, BCR-ABL Positive (8) · Lymphoma (8) · Tuberous Sclerosis (8) · Diabetes Mellitus, Type 1 (7) · Graft vs Host Disease (7) · Lymphoma, Mantle-Cell (7) · Neoplasms (7)
City of Hope Medical Center (11) · Mayo Clinic (9) · Fred Hutchinson Cancer Center (8) · National Institute of Allergy and Infectious Diseases (NIAID) (8) · The University of Texas Health Science Center at San Antonio (8) · Peking Union Medical College Hospital (5) · Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University (5) · University of Chicago (5) · West China Hospital (5) · Yale University (5) · AgelessRx (4) · Children's Hospital of Fudan University (4) · Dana-Farber Cancer Institute (4) · Stanford University (4) · University of Cincinnati (4)
United States (166) · China (27) · France (7) · Italy (7) · Australia (6) · Canada (6) · Germany (4) · Denmark (3) · Spain (3) · United Kingdom (3) · Belgium (2) · Netherlands (2) · Poland (2) · Sweden (2) · Switzerland (2)
Margaret Kasner, MD (5) · Ezra Cohen, MD (3) · Haris Ali (3) · Kunle Odunsi (3) · Yi Ji, MD, PhD (3) · AeRang Kim, MD, PhD (2) · Ali Naji, PhD (2) · Andrew Posselt, MD, PhD (2) · Bernhard Hering, MD (2) · Brenda Sandmaier (2) · Brigitte Widemann, MD (2) · Camillo Ricordi, MD (2) · Emanuele Bosi, MD (2) · Kai Li, MD, PhD (2) · Laura Johnston (2)
PMID 28486787, PMID 31489702, PMID 32014025, PMID 35030255, PMID 40132910, PMID 12176626, PMID 12176628, PMID 15061852, PMID 15233820, PMID 15753371, PMID 16030064, PMID 16614412, PMID 16873412, PMID 16896165, PMID 17351147, PMID 17478528, PMID 17510057, PMID 17569770, PMID 17885021, PMID 18056791, PMID 18215105, PMID 18236467, PMID 18317375, PMID 18323467, PMID 18342407, PMID 18347658, PMID 18403916, PMID 18535123, PMID 18713956, PMID 18787087, PMID 18827171, PMID 18845784, PMID 18997171, PMID 19057193, PMID 19150856, PMID 19167681, PMID 19188252, PMID 19208097, PMID 19710204, PMID 19828686, PMID 19950289, PMID 20070283, PMID 20150565, PMID 20304764, PMID 20519362, PMID 20664591, PMID 20844186, PMID 20876843, PMID 21046356, PMID 21076294
P30CA015704 (8), P01CA018029 (3), P30CA033572 (3), P01HL122173 (2), P30CA006973 (2), P30CA015083 (2), P30CA016056 (2), P30CA069533 (2), R01CA158318 (2), 2023 (1), KL2TR000118-05 (1), P01CA030206 (1), P01CA078902 (1), P30AG044271 (1), P30CA006516 (1)