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Home › Medications › sirolimus

sirolimus

16 published articles · Updated continuously

Clinical Trial Landscape

Clinical Trials of sirolimus

96 trials tracked of sirolimus: 24 in phase 3 or 4 and 6 with published results. The most-cited published study has 1106 citations.

96Trials tracked
24Phase 3 & 4
0Recruiting
6With published results
Phase distribution
Phase 4 11 Phase 3 13 Phase 2 62 Phase 1 3 Other / NA 7
  1. Phase 3 Efficacy and Safety of Sirolimus in LAM Completed · 1,106 cited
  2. Phase 3 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 · 230 cited
  3. Phase 3 Campath, Calcineurin Inhibitor Reduction and Chronic Allograft Nephropathy Completed · 150 cited
  4. Phase 3 Graft-Versus-Host Disease Prophylaxis in Treating Patients With Hematologic Malignancies Undergoing Unrelated Donor Peripheral Blood Stem Cell Transplant Completed · 114 cited
  5. Phase 3 TBI Dose De-escalation for Fanconi Anemia Completed · 111 cited
  6. Phase 4 Comparison of the Efficacy and Safety of Sirolimus Versus Everolimus Versus Mycophenolate in Kidney Transplantation Completed · 13 cited
Show 44 more trials
  1. Phase 4 Randomized Conversion of Calcineurin-Inhibitors in Renal Allograft Recipients Completed
  2. Phase 4 Study Evaluating Conversion From Tacrolimus to Sirolimus in Stable Kidney Transplant Recipients Receiving Myfortic Completed
  3. Phase 4 Improved Induction and Maintenance Immunosuppression in Kidney Transplantation Completed
  4. Phase 4 Kidney Spare the Nephron (STN) Study - A Study of CellCept (Mycophenolate Mofetil) and Rapamune (Sirolimus) in Kidney Transplant Recipients Completed
  5. Phase 4 A Study Comparing the Withdrawal of Steroids or Tacrolimus in Kidney Transplant Recipients Completed
  6. Phase 4 Liver Spare the Nephron (STN) Study - A Study of CellCept (Mycophenolate Mofetil) and Sirolimus in Recipients of a Liver Transplant Completed
  7. Phase 4 Comparison of Sirolimus and Azathioprine in Lung Transplantation Completed
  8. Phase 4 Study Evaluating the Effect of Sirolimus on Non-Melanoma Skin Cancer in Kidney Transplant Recipients Completed
  9. Phase 4 Study Evaluating A Planned Transition From Tacrolimus To Sirolimus In Kidney Transplant Recipients Completed
  10. Phase 4 Study Of The Safety And Efficacy Of Conversion From A CNI To Sirolimus In Renally-Impaired Heart Transplant Recipients Completed
  11. Phase 3 Efficacy of Sirolimus-Based, Steroid Avoidance Immunosuppression African Americans Completed
  12. Phase 3 Trial of Sirolimus for Cognitive Impairment in Sturge-Weber Syndrome Completed
  13. Phase 3 Trial to Evaluate the Sirolimus-Eluting Collagen Implant on AV Fistula Outcomes Completed
  14. Phase 3 De Novo Sirolimus-based Immunosuppression After Liver Transplantation for Hepatocellular Carcinoma Completed
  15. Phase 3 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
  16. Phase 3 Study Comparing Conversion to Sirolimus vs. Continued Use of Calcineurin Inhibitors in Kidney Transplant Recipients Completed
  17. Phase 3 Sirolimus/Tacrolimus Versus Tacrolimus/Methotrexate for Preventing Graft-Versus-Host Disease (GVHD) (BMT CTN 0402) Completed
  18. Phase 3 Study Evaluating Sirolimus in Kidney Transplant Recipients Completed
  19. Phase 2 Sirolimus and Thymoglobulin to Prevent Kidney Transplant Rejection Completed
  20. Phase 2 In Vivo Treg Expansion and Graft-Versus-Host Disease Prophylaxis Completed
  21. Phase 2 Examining the Impact of Sirolimus on Ketamine's Antidepressant Effects Completed
  22. Phase 2 Tacrolimus, Sirolimus and Ustekinumab vs. Tacrolimus and Sirolimus for the Prevention of Acute Graft-Versus-Host Disease Completed
  23. Phase 2 Sirolimus to Treat Cowden Syndrome and Other PTEN Hamartomatous Tumor Syndromes Completed
  24. Phase 2 Sirolimus, Cisplatin, and Gemcitabine Hydrochloride in Treating Patients With Bladder Cancer Completed
  25. Phase 2 Sirolimus, Tacrolimus, and Antithymocyte Globulin in Preventing Graft-Versus-Host Disease in Patients Undergoing a Donor Stem Cell Transplant For Hematological Cancer Completed
  26. Phase 2 Islet Transplantation for Type 1 Diabetes Completed
  27. Phase 2 Reduced Intensity, Partially HLA Mismatched BMT to Treat Hematologic Malignancies Completed
  28. Phase 2 Panobinostat (LBH589): Acute Graft Versus Host Disease (aGVHD) Prevention Completed
  29. Phase 2 Sirolimus in Conjunction With Eylea vs Eylea Alone for Exudative AMD Completed
  30. Phase 2 Stem Cell Transplantation in Patients With High-Risk and Recurrent Pediatric Sarcomas Completed
  31. Phase 2 Using Donor Stem Cells and Alemtuzumab to Prevent Organ Rejection in Kidney Transplant Patients Completed
  32. Phase 2 Rapalogues for Autism Phenotype in TSC: A Feasibility Study Completed
  33. Phase 2 Pilot Study of the Evaluation of Subconjunctival Sirolimus in the Treatment of Bilateral GA Associated With AMD Completed
  34. Phase 2 SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors Completed
  35. Phase 2 Sirolimus Injections for Autoimmune Scleritis Completed
  36. Phase 2 Sirolimus, Cyclosporine, and Mycophenolate Mofetil in Preventing Graft-versus-Host Disease in Treating Patients With Blood Cancer Undergoing Donor Peripheral Blood Stem Cell Transplant Completed
  37. Phase 2 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
  38. Phase 2 Sirolimus and Familial Adenomatous Polyposis (FAP) Completed
  39. Phase 2 UCB Transplant for Hematological Diseases Using a Non Myeloablative Prep Completed
  40. Phase 2 A Phase II Study of Belatacept (BMS-224818) With a Steroid-free Regimen in Subjects Undergoing Kidney Transplantation Completed
  41. Phase 2 A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas Completed
  42. Phase 2 Combination Immunosuppressive Therapy to Prevent Kidney Transplant Rejection in Adults Completed
  43. Phase 2 A Pilot Study Evaluating the Use of mTor Inhibitor Sirolimus in Children and Young Adults With Desmoid-Type Fibromatosis Completed
  44. Phase 2 Sirolimus to Treat Diabetic Macular Edema Completed

Showing the 50 most-cited and recently-updated of 96 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Proteomic profiling in anti-VEGF-treated AMD patients reveals VEGF-independent immune pathway activation
Ophthalmology
Proteomic profiling in anti-VEGF-treated AMD patients reveals VEGF-independent immune pathway activation Why Immune Cells Keep Damaging Your Eyes
This observational study analyzed vitreous proteomic profiles in eight patients with neovascular AMD receiving anti-VEGF therapy compared to…
Standard eye drops for AMD stop one problem, but a second, hidden immune attack is still hurting your vision.
medRxiv Apr 14, 2026
Itacitinib plus standard therapy in acute leukemia or myelofibrosis patients undergoing reduced-intensity conditioning donor stem cell transplantation
Oncology Phase II
Itacitinib plus standard therapy in acute leukemia or myelofibrosis patients undergoing reduced-intensity conditioning donor stem cell transplantation Early trial explores itacitinib for stem cell transplant patients with blood disorders
This Phase IIa trial evaluated itacitinib combined with tacrolimus and sirolimus in patients with acute leukemia, myelodysplastic syndrome, …
A new trial adds itacitinib to standard care for stem cell transplant patients with blood disorders to help prevent graft-versus-host diseas…
CT.gov Apr 13, 2026
Rapamycin therapy reduced infections and lymph node size in a female child with APDS1
Rheumatology Sys. Review
Rapamycin therapy reduced infections and lymph node size in a female child with APDS1 Neck Mass Was Not Cancer, It Was This Rare Condition
This case report describes a single female patient with Activated phosphoinositide 3-kinase delta syndrome type I (APDS1) who received rapam…
A rare genetic immune disorder caused a neck mass mistaken for cancer, ending years of failed treatments for recurrent lung infections and s…
Frontiers Apr 11, 2026
Emergency sclerotherapy resolves airway obstruction in neonates with giant head and neck lymphatic malformations
Pediatrics Cohort
Emergency sclerotherapy resolves airway obstruction in neonates with giant head and neck lymphatic malformations A Simple Procedure Saves Newborns Struggling to Breathe
A retrospective cohort study of 13 neonates with giant head and neck lymphatic malformations complicated by airway obstruction found that em…
Doctors have refined a life-saving technique that can relieve a newborn’s airway obstruction in days, preventing the need for risky surgery.
Frontiers Apr 11, 2026