OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)
Last updated on July 2021Recruitment
- Recruitment Status
- Recruiting
- Estimated Enrollment
- Same as current
Summary
- Conditions
- Lysosomal Storage Diseases
- Metachromatic Leukodystrophy
- Type
- Interventional
- Phase
- Phase 3
- Design
- Allocation: N/AIntervention Model: Single Group AssignmentIntervention Model Description: All eligible subjects will receive intravenous (IV) infusion of OTL-200 gene therapy. Subjects will also receive conditioning regimen with busulfan.Masking: None (Open Label)Primary Purpose: Treatment
Participation Requirements
- Age
- Younger than 125 years
- Gender
- Both males and females
Description
Not Provided
Not Provided
Tracking Information
- NCT #
- NCT04283227
- Collaborators
- Ospedale San Raffaele
- Investigators
- Study Director: Orchard Clinical Trials Orchard Therapeutics
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