Miglustat Oral Solution 40 mg/mL

DRUG2 trials

Sponsors

Amicus Therapeutics Inc.

Conditions

Late-onset Pompe diseasePediatric Subjects with Infantile-onset Pompe Disease Aged 0 to < 18 Years

Phase 3

An Open-label Study to Evaluate the Safety, Efficacy, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Both ERT-experienced and ERT-naïve Pediatric Subjects with Infantile-onset Pompe Disease Aged 0 to <18 Years

RecruitingCTIS2022-501095-25-01

Amicus Therapeutics Inc.Pediatric Subjects with Infantile-onset Pompe Disease Aged 0 to < 18 Years

Start: 2024-11-04Target: 15Updated: 2026-01-26

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

RecruitingCTIS2022-502547-36-00

Amicus Therapeutics Inc.Late-onset Pompe disease

Start: 2024-03-21Target: 9Updated: 2025-12-29