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OTL-200 in Japanese patients with early-onset metachromatic leukodystrophy
jRCT2033260220Recruiting
- Sponsor
- Kyowa Kirin
- Start
- Jul 2026
- Modality
- Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Gene Therapies
- Countries
- 1
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Overview
Open-label, single-arm trial of atidarsagene autotemcel in metachromatic leukodystrophy (MLD), sponsored by Kyowa Kirin. Primary endpoint: ARSA activity in total PBMCs following administration of OTL-200. Started Jul 2026; status: recruiting.
02
Drugs
atidarsagene autotemcel
03
Conditions
metachromatic leukodystrophy (MLD)
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