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OTL-200 in late juvenile metachromatic leukodystrophy
2024-511971-13-00Phase 3Authorised
- Sponsor
- Orchard Therapeutics
- Start
- Sep 2020
- Modality
- Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Gene Therapies
- Countries
- 1
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Overview
Phase 3, open-label, single-arm trial of atidarsagene autotemcel in metachromatic leukodystrophy (MLD), sponsored by Orchard Therapeutics. Primary endpoints: Change in ARSA activity in cerebrospinal fluid and Change in NAA:Cr ratio in brain white matter. Planned start Sep 2020; status: authorised.
02
Drugs
atidarsagene autotemcel
03
Conditions
metachromatic leukodystrophy (MLD)
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