Rapamune 0.5 mg coated tablets
Sponsors
Prevail Therapeutics Inc., Helse Bergen HF, Les Hopitaux Universitaires De Strasbourg, Alexion Pharmaceuticals Inc., Hospital Universitario La Paz
Conditions
BAG3 Mutation Associated Dilated CardiomyopathyFronto-Temporal Dementia with Progranulin Mutations (FTD-GRN)Thyroid eye diseaseType 1 Gaucher Disease (Peripheral/Non-neuronopathic Manifestations)advanced chronic renal insufficiencykidney transplant
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