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Intrathecal SHP611 in Late Infantile Metachromatic Leukodystrophy (Matched External Control)
jRCT2041200086Completed
- Sponsor
- Takeda
- Start
- Apr 2019
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
01
Overview
Open-label trial of cebsulfase alfa in metachromatic leukodystrophy (MLD) (6 arms), sponsored by Takeda. Primary endpoint: Percent probability of being free of loss of locomotion up to 2 years based on GMFC-MLD (Group A vs GLIA-MLD external control). Started Apr 2019; status: completed.
02
Drugs
cebsulfase alfa
03
Conditions
metachromatic leukodystrophy (MLD)
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