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

NCT03911505 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 21

Last updated 2025-10-27

No results posted yet for this 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

BIOLOGICAL

Cipaglucosidase Alfa

Enzyme Replacement Therapy via intravenous infusion

DRUG

Miglustat

Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat(AT2221)

Sponsors & Collaborators

  • Amicus Therapeutics

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
0 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-02-13
Primary Completion
2026-06-30
Completion
2026-06-30
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Canada
  • Germany
  • Italy
  • Japan

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03911505 on ClinicalTrials.gov