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A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease

NCT03729362 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 125

Last updated 2025-09-11

Study results available
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Summary

This is a phase 3 double-blind randomized study to study the efficacy and safety of intravenous ATB200 Co-administered with oral AT2221 in adult subjects with Late Onset Pompe Disease compared with Alglucosidase Alfa/placebo.

Conditions

  • Pompe Disease (Late-onset)

Interventions

BIOLOGICAL

Cipaglucosidase Alfa

Participants received an intravenous (IV) infusion dose over a 4-hour duration every 2 weeks (Q2W).

DRUG

Miglustat

Participants received weight-based doses 1 hour prior to cipaglucosidase alfa infusion Q2W.

BIOLOGICAL

Alglucosidase Alfa

Participants received an IV infusion dose over a 4-hour duration Q2W.

DRUG

Placebo

Miglustat matching placebo was administered orally 1 hour prior to alglucosidase alfa infusion Q2W.

Sponsors & Collaborators

  • Amicus Therapeutics

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-12-04
Primary Completion
2020-12-15
Completion
2021-01-15
FDA Drug
Yes

Countries

  • United States
  • Argentina
  • Australia
  • Austria
  • Belgium
  • Bosnia and Herzegovina
  • Bulgaria
  • 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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Entities

Drugs

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 NCT03729362 on ClinicalTrials.gov