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NCT04808505Phase 3RECRUITING

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

Study 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.

Conditions
Glycogen Storage Disease Type II Infantile Onset
Interventions / Drugs
Cipaglucosidase alfaMiglustat
View on ClinicalTrials.gov ↗
Sponsor
FOLD
Amicus Therapeutics
Rare Disease
C57/100
Study Details
Enrollment36 participants
Primary Completion—
Lead SponsorAmicus Therapeutics
Data Retrieved2026-04-11 04:14:12.640544+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Currently recruiting: the trial is enrolling patients. A data readout is not expected until after enrollment closes and the follow-up period is complete.

Enrolled: 36 participants
Sponsor Catalyst Profile

Amicus Therapeutics (FOLD) is the sponsoring company for this trial. BiotechSign currently grades this company C (57/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

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