Sirolimus

• A geroscience approach to post-acute COVID-19 syndrome (PACS) by Diet and Exercise plus rapamycin or metformin to treat Frailty and restore … Not authorised
  2023-504641-32-00 Phase II 1 country Azienda Ospedaliero Universitaria Di Modena
• ATA-200 gene therapy trial in patients with LGMDR5 Pending
  2023-506440-16-00 Phase I (FiH) 2 countries Atamyo Therapeutics
• Safety study for the use of Rapamycin in children with familial adenomatous polyposis - RAPA-4-FAP Pending
  2024-512079-11-00 Phase II 1 country Centre Hospitalier Universitaire De Toulouse
• MAV-RAPA: Prospective evaluation of the efficacy of sirolimus (Rapamune) in the treatment of superficial arteriovenous malformations. Pending
  2024-516001-23-00 Phase II 1 country Centre Hospitalier Universitaire Amiens Picardie
• Safety and efficacy of steering of immunosuppression based on BKPyV-specific T cells in case of BK Polyomavirus (BKPyV) DNAemia after kidney… Pending
  2024-517219-56-00 Phase IV 1 country Paediatrisches Forschungsnetzwerk gGmbH; PROFIL Institut fuer Stoffwechselforsch…
• RAPAMALYMPH protocol Pending
  2024-517769-17-00 Phase II 1 country Centre Hospitalier Universitaire De Lille
• A Phase 1/2 study of ALXN2350 in adult participants with BAG3-associated dilated cardiomyopathy Recruiting
  2024-519674-40-00 Phase I (FiH) 3 countries Alexion Pharmaceuticals Inc.
• Phase 3 trial of eRapa in patients with familial adenomatous polyposis (FAP) Recruiting
  2025-522946-37-00 Phase III 5 countries Biodexa Limited; Rapamycin Holdings Inc.
• Immunosuppression regimen reduction in kidney transplantation recipients admitted to the intensive care unit in the setting of septic shock … Recruiting
  2024-518493-14-00 Phase II/III 1 country Les Hopitaux Universitaires De Strasbourg
• Multi-Center, Open Label Gene Therapy Study of RP-A501 in Male Patients with Danon Disease Recruiting
  2023-506480-34-00 Phase II 2 countries Rocket Pharmaceuticals Inc.
• Use of tacrolimus and MTOR inhibitors with anticipatory therapy vs. tacrolimus and mycophenolic acid in renal recipients at high risk of pos… Recruiting
  2025-520854-12-00 Phase IV 1 country Hospital Universitario La Paz
• Preparation for bone marrow transplant through the combined use of chemo and radiotherapy (intensive or myeloablative: eliminates stem cells… Recruiting
  2024-514484-25-00 Phase II 1 country Fundacion Publica Andaluza Para La Gestion De La Investigacion En Salud De Sevil…
• Study of the effectiveness of drugs selected depending on the molecular profile of diffuse intrinsic pontine glioma (DIPG). Recruiting
  2024-515952-19-00 Phase II 1 country Instytut Pomnik Centrum Zdrowia Dziecka
• Efficacy and safety trial of GNT0003 following imlifidase pre-treatment in participants aged 16 years and older with severe Crigler-Najjar s… Recruiting
  2023-510405-18-00 Phase II 1 country Genethon
• Prospective comparison of sirolimus against corticosteroids in treatment of patients with active thyroid eye disease Recruiting
  2023-510166-27-01 Phase III 1 country Helse Bergen HF
• Dose-Ranging Efficacy Study of rapamycin cream applied topically in subjects diagnosed with port wine stains Recruiting
  2025-520821-19-00 Phase II 1 country Aft Pharmaceuticals Limited
• Long-term neuropsychologic outcome of pre-emptive mTOR inhibitor treatment in children with tuberous sclerosis complex (TSC) under 4 months … Recruiting
  2022-502332-39-00 Phase II 2 countries Heidelberg University Hospital AöR
• Placebo controlled study to assess the efficacy and safety of rapamycin in drug resistant epilepsy associated with tuberous sclerosis comple… Recruiting
  2024-515937-13-00 Phase III 1 country Instytut Pomnik Centrum Zdrowia Dziecka
• Comparison of the efficacy and safety of rapamycin versus vigabatrin in the prevention of Tuberous Sclerosis Complex symptoms in infants in … Recruiting
  2024-513068-25-00 Phase II/III 1 country Instytut Pomnik Centrum Zdrowia Dziecka
• A clinical study in 3 parts with a microdystrophin (called GNT0004), a new gene therapy in boys with Duchenne disease who can still walk. Th… Recruiting
  2023-505187-11-00 Phase I (FiH) 3 countries Genethon
• SIROLIMUS VERSUS MYCOPHENOLATE MOFETIL IN SIMULTANEOUS PANCREAS AND KIDNEY TRANSPLANTATION TO EVALUATE THE RISK OF HERNIA Recruiting
  2024-517391-38-00 Phase IV 1 country Institute For Clinical And Experimental Medicine
• Gene Therapy for Crigler-Najjar Syndrome Recruiting
  2023-507007-60-00 Phase I (FiH) 3 countries Genethon
• A Phase 1/2 Study with Ascending Dose in order to Evaluate the Safety and Effects on Progranulin Levels of investigational product under cod… Ended
  2022-502942-29-01 Phase I/II 2 countries Prevail Therapeutics Inc.
• SIRIUS Sarcoidosis with moderate to severe facial skin involvement: an open multicenter study of the efficacy and safety of oral sirolimus Ended
  2024-514609-76-00 Phase II 1 country Assistance Publique Hopitaux De Paris

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