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

a study on Pompe Disease (Late-onset)

Summary

for people ages 18 years and up (full criteria)
at Irvine, California
study started
estimated completion

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.

Location

  • University of California, Irvine accepting new patients
    Irvine California 92868 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Amicus Therapeutics
ID
NCT04138277
Phase
Phase 3
Study Type
Interventional
Last Updated