This study is in progress, not accepting new patients

A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

a study on Pompe Disease

Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at Irvine, California and other locations
Dates
study started
completion around

Description

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.

Official Title

A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease

Details

This is an open label extension study for subjects who completed the ATB200-03 study. The subjects will stay in this study until regulatory approval or marketing authorization and/or commercialization in the participating subject's country.

Keywords

Pompe Disease (Late-onset), Pompe, rhGAA, Glycogen Storage Disease Type II, Miglustat, ATB200, ATB200/AT2221

Eligibility

You can join if…

Open to people ages 18 years and up

  1. Subject must have completed Study ATB200-03.

You CAN'T join if...

  1. Subject plans to receive gene therapy or participate in another interventional study for Pompe disease.
  2. Subject, if female, is pregnant or breastfeeding.
  3. Subject, whether male or female, is planning to conceive a child during the study.

Locations

  • University of California, Irvine
    Irvine California 92868 United States
  • University of Utah
    Salt Lake City Utah 84108 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Amicus Therapeutics
ID
NCT04138277
Phase
Phase 3 Pompe Disease Research Study
Study Type
Interventional
Participants
Expecting 110 study participants
Last Updated