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

Amicus Therapeutics

Status and phase: Active, not recruiting; Phase 3

Conditions: Pompe Disease (Late-onset)

Treatments: Biological: ATB200; Drug: AT2221

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: 110 estimated 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.

Exclusion Criteria

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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110 participants in 1 patient group

ATB200/AT2221

Experimental group

Description:

Participants received ATB200 co-administered with AT2221 capsule (Miglustat)

Treatment:

Biological: ATB200

Drug: AT2221