ATB200/AT2221 Phase 3 Open-label Extension Study

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

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2011200003

Enrollment

110

Registered

2020-10-05

Start date

2020-10-12

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Pompe disease

Interventions

ATB200 20 mg/kg IV infusion over a 4-hour duration 1 hour after AT2221 administration every 2 weeks AT2221 Subjects weighing >= 50 kg, 260mg (4 oral capsules of AT2221) 1hour prior to ATB200 infusion

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Subject must have completed Study ATB200-03

Exclusion criteria

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.

Design outcomes

Primary

Measure	Time frame
Proportion of participants with Treatment Emergent Adverse Events (TEAE)	—

Secondary

Measure	Time frame
Change from baseline in 6MWD, sitting FVC (% predicted), the manual muscle test score, physical function, fatigue score and et.al	—

Countries

Asia, Europe, Japan, North America, Oceania, South America

Contacts

Public ContactMasashi Sakamoto

Amicus Therapeutics K.K.

msakamoto@amicusrx.com+81-3-4563-4794

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026