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Intrathecal SHP611 in late infantile metachromatic leukodystrophy
2018-003291-12Phase 2Active
- Sponsor
- Takeda
- Start
- Feb 2019
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 2, open-label trial of cebsulfase alfa in metachromatic leukodystrophy (MLD) (2 arms), sponsored by Takeda. Primary endpoint: Maintenance of gross motor function at 2 years by GMFC-MLD. Started Feb 2019; status: active.
02
Drugs
cebsulfase alfa
03
Conditions
metachromatic leukodystrophy (MLD)
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