Clinical trial
An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease
NCT03911505 is registered in ClinicalTrials.gov with Amicus Therapeutics as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease
- Lead sponsor
- Amicus Therapeutics
- Phase
- Phase 3
- Overall status
- Active, not recruiting
- Start date
- February 13, 2020
- Completion date
- September 30, 2027
- Enrollment
- 21 (actual)
- Sites
- 17
Conditions
As the registry lists them; a condition with its own page is linked.
pompe disease (late-onset)