A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18

Recruiting Phase 3 Interventional Study
Glycogen Storage Disease Type II Infantile Onset
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Pivotal Trial This treatment is in the last trial phase before FDA approval. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
Up to 17
Sex
Any
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
36 (estimated)
Sponsor
Amicus Therapeutics · Industry
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About This Trial

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.

Trial Locations
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Eligibility Criteria
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 immediate…
Contacts

For Site

609-662-2000

patientadvocacy@amicusrx.com

CONTACT

For Patient

609-662-2000

patientadvocacy@amicusrx.com

CONTACT