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A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With Late-Onset Pompe Disease (LOPD)

NCT04138277 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 119

Last updated 2026-03-05

Study results available
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Summary

This is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.

Conditions

  • Pompe Disease (Late-onset)

Interventions

DRUG

AT2221

Participants received ATB200 co-administered with AT2221 (miglustat)

BIOLOGICAL

ATB200

Enzyme Replacement Therapy via intravenous infusion

Sponsors & Collaborators

  • Amicus Therapeutics

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-12-18
Primary Completion
2024-12-31
Completion
2024-12-31
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Australia
  • Austria
  • Belgium
  • Bosnia and Herzegovina
  • Canada
  • Denmark
  • France
  • Germany
  • Greece
  • Hungary
  • Italy
  • Japan
  • Netherlands
  • New Zealand
  • Poland
  • Slovenia
  • South Korea
  • Spain
  • Sweden
  • Taiwan
  • United Kingdom

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 NCT04138277 on ClinicalTrials.gov