Indication
Metachromatic Leukodystrophy
7 clinical trials
11 products
Clinical trial
An Open Label, Non-randomized Trial to Evaluate the Safety and Efficacy of a Single Infusion of OTL-200 in Patients With Late Juvenile (LJ) Metachromatic Leukodystrophy (MLD).Status: Active (not recruiting), Estimated PCD: 2025-03-31
Product
OTL-200Product
HGT-1110Clinical trial
An Open-Label Extension of Study HGT-MLD-070 Evaluating Long Term Safety and Efficacy of Intrathecal Administration of HGT-1110 in Patients With Metachromatic LeukodystrophyStatus: Active (not recruiting), Estimated PCD: 2024-12-31
Clinical trial
A Phase I/II Clinical Trial of Hematopoietic Stem Cell Gene Therapy for the Treatment of Metachromatic LeukodystrophyStatus: Active (not recruiting), Estimated PCD: 2018-04-09
Product
SHP611Clinical trial
A Global, Multicenter, Single-arm, Matched External Control Study of Intrathecal SHP611 in Subjects With Late Infantile Metachromatic LeukodystrophyStatus: Active (not recruiting), Estimated PCD: 2023-03-08
Clinical trial
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersStatus: Terminated, Estimated PCD: 2016-04-01
Clinical trial
A Single Arm, Open Label, Clinical Study of Cryopreserved Autologous CD34+ Cells Transduced With Lentiviral Vector Containing Human ARSA cDNA (OTL-200), for the Treatment of Early Onset Metachromatic Leukodystrophy (MLD)Status: Active (not recruiting), Estimated PCD: 2022-04-06
Clinical trial
MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATGStatus: Recruiting, Estimated PCD: 2025-07-14
Product
Stem Cell TransplantationProduct
IMD Preparative RegimenProduct
OsteopetrosisProduct
CALD SR-AProduct
cALD SR-BProduct
cALD HR-D