Clinical trial

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

Name

ATB200-07

Description

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.

Trial arms

Trial start

2019-12-18

Estimated PCD

2024-12-01

Trial end

2024-12-01

Status

Active (not recruiting)

Phase

Early phase I

Treatment

AT2221

Participants received ATB200 co-administered with AT2221 (Miglustat)

Arms:

ATB200/AT2221

Other names:

Miglustat

ATB200

Enzyme Replacement Therapy via intravenous infusion

Arms:

ATB200/AT2221

Size

110

Primary endpoint

Proportion of participants with Treatment Emergent Adverse Events (TEAE)

baseline, up to approximately 4 years

Eligibility 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.

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 110, 'type': 'ESTIMATED'}}

Updated at

2024-03-20

1 organization

2 products

1 indication

Product

Indication

Organization

Product