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Alglucosidase alfa in Chinese infants with infantile-onset Pompe disease
ChiCTR1800018514Phase 4Not yet recruiting
- Sponsor
- Shanghai Children's Medical Center
- Start
- Nov 2018
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
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Overview
Phase 4, open-label, single-arm trial of alglucosidase alfa in Pompe disease, sponsored by Shanghai Children's Medical Center. Primary endpoints: Overall survival at 52 weeks and Change from baseline in left ventricular mass index. Planned start Nov 2018; status: not yet recruiting.
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Drugs
alglucosidase alfa
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Conditions
Pompe disease
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