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
Amicus Therapeutics
Summary
This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
Eligibility
- Age range
- Up to 17 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: Cohort 1: 1. Male or female subjects who are aged 6 months to \< 18 years on Day 1 2. Subject must have documentation of IOPD genotype 3. Subject must have had hypertrophic cardiomyopathy at the time of diagnosis 4. Subject must have received ERT for at least 6 months immediately before enrollment. For subjects whose ERT dosage has been modified, the subject must have been on the modified dosage and regimen for at least 3 months before enrollment 5. Subjects aged ≥ 12 to \< 18 years must perform one valid 6-minute walk test (6MWT) (≥ 75 meters) at screening; Subjects aged…
Interventions
- BiologicalCipaglucosidase alfa
Sterile lyophilized powder intravenous (IV) infusion
- DrugMiglustat
65 mg oral capsules
Locations (14)
- University of Florida Clinical Research CenterGainesville, Florida
- The Emory ClinicAtlanta, Georgia
- Duke University Early Phase Research UnitDurham, North Carolina
- Cincinnati Children's Hospital Medical CenterCincinnati, Ohio
- UPMC Hospital of PittsburghPittsburgh, Pennsylvania
- University of Utah, Clinical and Translational Sciences InstituteSalt Lake City, Utah