NCT04283227View on ClinicalTrials.gov

OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

6

Participants

Timeline

Start Date

January 17, 2022

Primary Completion Date

January 31, 2026

Study Completion Date

March 31, 2031

Conditions
Lysosomal Storage DiseasesMetachromatic Leukodystrophy
Interventions
GENETIC

OTL-200

All subjects will receive OTL-200 gene therapy and will be followed up for 8 years following treatment with OTL-200.

Trial Locations (1)

20132

Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET), Milan

Sponsors
All Listed Sponsors
collaborator

Ospedale San Raffaele

OTHER

lead

Orchard Therapeutics

INDUSTRY

NCT04283227 - OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) | Biotech Hunter | Biotech Hunter