Rapamune 1 mg coated tablets
Sponsors
Prevail Therapeutics Inc., Fir Huvh Fundacio Institut De Recerca Hospital Universitari Vall De Hebron, Instytut Pomnik Centrum Zdrowia Dziecka, Centre Hospitalier Universitaire Amiens Picardie, Institute For Clinical And Experimental Medicine
Conditions
BAG3 Mutation Associated Dilated CardiomyopathyEnd-stage kidney disease in Type 1 diabetesFronto-Temporal Dementia with Progranulin Mutations (FTD-GRN)Prevention viral infection (BKV) poliamavirus after kidney trasplantationSIROLIMUS VERSUS MYCOPHENOLATE MOFETIL IN SIMULTANEOUS PANCREAS AND KIDNEY TRANSPLANTATION TO EVALUATE THE RISK OF HERNIAType 1 Gaucher Disease (Peripheral/Non-neuronopathic Manifestations)diffuse intrinsic pontine gliomasuperficial arteriovenous malformations
Phase 1
An Open-label, Dose-Finding, Phase 1/2 Study to Evaluate the Safety and Tolerability of a Single Intravenous Dose of LY3884961 in Subjects with Peripheral Manifestations of Gaucher Disease
SuspendedCTIS2022-500281-10-02
Start: 2023-10-18Target: 6Updated: 2025-09-11
A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on Progranulin Levels of LY3884963 in Patients with Fronto-Temporal Dementia with Progranulin Mutations (FTD-GRN) (PROCLAIM)
SuspendedCTIS2022-502942-29-01
Start: 2024-08-05Target: 15Updated: 2025-11-26
A Phase 1/2, Open-Label, Dose Finding Study to Investigate the Safety, Tolerability, and
Efficacy of ALXN2350 Gene Therapy in Adult Participants with Symptomatic BAG3
Mutation-Associated Dilated Cardiomyopathy
Not yet recruitingCTIS2024-519674-40-00
Target: 8Updated: 2025-12-12
Phase 2
Multicenter, open label study using molecularly determined targeted therapies in children 3-18 years of age with DIPG (diffuse intrinsic pontine glioma- DIPG)- DIPGen
RecruitingCTIS2024-515952-19-00
Start: 2024-12-18Target: 100Updated: 2025-04-25
MAV-RAPA: Prospective evaluation of the efficacy of sirolimus (Rapamune) in the treatment of superficial arteriovenous malformations.
Not yet recruitingCTIS2024-516001-23-00
Target: 50Updated: 2025-01-17
Phase 4
Evaluation and promotion of specific adaptative immunity against polyomavirus (BKV) to prevent viral infection after kidney transplantation (BK-VAX project)
Not yet recruitingCTIS2023-504309-35-00
Target: 70Updated: 2023-06-15
SIROLIMUS VERSUS MYCOPHENOLATE MOFETIL IN SIMULTANEOUS PANCREAS AND KIDNEY TRANSPLANTATION TO EVALUATE THE RISK OF HERNIA
RecruitingCTIS2024-517391-38-00
Start: 2020-09-09Target: 120Updated: 2024-10-08