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

Amicus Therapeutics

Status and phase

Completed

Phase 3

Conditions

Pompe Disease (Late-onset)

Treatments

Drug: AT2221

Biological: ATB200

Study type

Interventional

Funder types

Industry

Identifiers

NCT04138277

ATB200-07

Details and patient eligibility

About

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.

Full description

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.

Enrollment

119 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

1. Subject must have completed Study ATB200-03.

Note: Subjects who were forced to withdraw from Study ATB200-03 for a logistical reason not related to the efficacy or safety of cipaglucosidase alfa/miglustat (eg, hospitalization for a car accident, COVID-19 pandemic, or emergency surgery) that resulted in several consecutive missed doses may have been eligible to participate in this study upon approval by the Amicus medical monitor.

Exclusion Criteria

Subject plans to receive gene therapy or participate in another interventional study for Pompe disease.
Subject, if female, is pregnant or breastfeeding.
Subject, whether male or female, is planning to conceive a child during the study.
Subject had a hypersensitivity to any of the excipients in cipaglucosidase alfa or miglustat, or had a medical condition or any other extenuating circumstance that may have, in the opinion of the investigator or medical monitor, posed an undue safety risk to the subject or may have compromised his/her ability to comply with or adversely impacted protocol requirements.