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NCT03911505Phase 3ACTIVE NOT RECRUITING

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

Study Summary

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to \< 18 years

Conditions
Pompe Disease (Late-onset)
Interventions / Drugs
Cipaglucosidase AlfaMiglustat
View on ClinicalTrials.gov ↗
Sponsor
FOLD
Amicus Therapeutics
Rare Disease
C57/100
Study Details
Enrollment21 participants
Primary Completion—
Lead SponsorAmicus Therapeutics
Data Retrieved2026-04-11 04:14:12.640530+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

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 21 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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