At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 119 enrolled
Drug / intervention
AT2221 +1 moredrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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 (LOPD)
In Brief
A Phase 3 clinical trial evaluating AT2221 and ATB200 for Pompe Disease (Late-onset). Completed, enrolled 119 participants across 60 sites in 23 countries.
Detailed 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.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsPompe Disease (Late-onset)
CountriesArgentina, Australia, Austria, Belgium, Bosnia and Herzegovina, Canada, Denmark, France, Germany, Greece, Hungary, Italy, Japan, Netherlands, New Zealand, Poland, Slovenia, South Korea, Spain, Sweden, Taiwan, United Kingdom, United States
Collaborators--
Timeline
Phase 3CompletedFinished
2020202120222023202420252026
First PostedOct 2019
Enrollment StartDec 2019
Primary CompletionDec 2024
TodayJul 2026
First PostedOct 24, 2019
Enrollment StartDec 18, 2019
Primary CompletionDec 31, 2024
TodayJul 2, 2026
Enrollment to primary: 5.0 yearsPosted 6.7 years ago
Interventions
AT2221drug
Participants received ATB200 co-administered with AT2221 (miglustat)
ATB200biological
Enzyme Replacement Therapy via intravenous infusion