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 Therapeutics14 sites in 6 countries36 target enrollmentJuly 18, 2023

ConditionsGlycogen Storage Disease Type II Infantile Onset

InterventionsCipaglucosidase alfa Miglustat

DrugsCipaglucosidase alfa Miglustat

Overview

Phase: Phase 3
Intervention: Cipaglucosidase alfa
Conditions: Glycogen Storage Disease Type II Infantile Onset
Sponsor: Amicus Therapeutics
Enrollment: 36
Locations: 14
Primary Endpoint: Proportion of subjects with infusion-associated reactions (IARs)
Status: Recruiting
Last Updated: 3 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

July 18, 2023

End Date

July 1, 2027

Last Updated

3 months ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Amicus Therapeutics

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Male or female subjects who are aged 6 months to \< 18 years on Day 1
•Subject must have documentation of IOPD genotype
•Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
•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
•Subjects aged ≥ 12 to \< 18 years must perform one valid 6-minute walk test (6MWT) (≥ 75 meters) at screening; Subjects aged ≥ 5 to \< 12 years must perform one valid 6MWT (≥ 40 meters) at screening; Subjects aged 18 months to \< 5 years must be ambulatory and assessed to be likely to be able to perform 6MWT (≥ 40 meters) when they turn 5 years old
•Subjects must have experienced a clinical decline on their current rhGAA dose and frequency
•Male or female subjects who are aged 0 to \<6 months at Day 1
•Subject must have documentation of IOPD genotype
•Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
•Subject is ERT-naïve

Exclusion Criteria

•Cohort 1 and Cohort 2, unless specified
•Subject requires invasive ventilation (eg, tracheostomy)
•Subject is CRIM negative and has not received prophylactic immunomodulation (Cohort 1); Subject is CRIM negative and will not be receiving prophylactic immunomodulation (Cohort 2)
•Subject has a history of life-threatening IARs/hypersensitivity (eg, anaphylaxis and severe cutaneous reactions) to ERT (eg, alglucosidase alfa, cipaglucosidase alfa, miglustat) or other iminosugars, or to any of the excipients, where rechallenge was unsuccessful
•Subject has prior history of illness or condition known to affect motor function
•Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (Cohort 1)