Indication
Lysosomal Storage Diseases
5 clinical trials
4 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
FLT190Clinical trial
A Phase 1/2, Baseline-controlled, Non-randomized, Open-label, Single-ascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry DiseaseStatus: Terminated, Estimated PCD: 2023-05-02
Clinical trial
Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe DiseaseStatus: Active (not recruiting), Estimated PCD: 2032-04-01
Product
SPK-3006Clinical trial
A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects With Fabry DiseaseStatus: Completed, Estimated PCD: 2023-09-28
Clinical trial
Longitudinal Study of Neurodegenerative DisordersStatus: Recruiting, Estimated PCD: 2030-01-01