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Open-Label Extension of HGT-MLD-070 Assessing Long-Term Safety and Efficacy of Intrathecal HGT-1110 in Metachromatic Leukodystrophy
2024-514403-34-00Phase 2Completed
- Sponsor
- Takeda
- Start
- Oct 2024
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 8
01
Overview
Phase 2, open-label, single-arm trial of cebsulfase alfa in metachromatic leukodystrophy (MLD), sponsored by Takeda. Primary endpoints: Treatment-emergent adverse events, Changes in clinical laboratory tests and 2 more. Started Oct 2024; status: completed.
02
Drugs
cebsulfase alfa
03
Conditions
metachromatic leukodystrophy (MLD)
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