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EMBOLDEN: Intrathecal SHP611 in Late Infantile Metachromatic Leukodystrophy
NCT03771898Phase 2Completed
- Sponsor
- Takeda
- Start
- May 2019
- Primary completion
- Mar 2023
- Modality
- Replacement Enzymes
- Countries
- 14
01
Overview
Phase 2, open-label, single-arm trial of cebsulfase alfa in metachromatic leukodystrophy (MLD), sponsored by Takeda. Primary endpoint: Percent Probability of Free of Loss of Locomotion Based on GMFC-MLD at 106 weeks. Started May 2019; status: completed.
02
Drugs
cebsulfase alfa
03
Conditions
metachromatic leukodystrophy (MLD)
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