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NCT03911505 · ClinicalTrials.gov registry record · Phase 3
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
A Phase 3 study of Pompe Disease (Late-onset), sponsored by Amicus Therapeutics.
- Active
- Registry status
- Phase 3
- Development phase
- 21
- Enrollment target
- 17
- Study locations
NCT03911505: Active Phase 3 study of Pompe Disease (Late-onset), sponsored by Amicus Therapeutics.
NCT03911505 is a Phase 3 study of Pompe Disease (Late-onset) that is active but no longer recruiting, run by Amicus Therapeutics. The registered enrollment target is 21 participants, below the 45-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (53% lower). The trial reports 17 study locations across 13 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03911505, a Phase 3 study of Pompe Disease (Late-onset), is active but no longer recruiting, sponsored by Amicus Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 21 participants
- Enrollment target
- 17
- Study locations
Study Summary
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to \< 18 years
Conditions Studied
Interventions
- DRUG Miglustat
- BIOLOGICAL Cipaglucosidase Alfa
Study Locations (17)
Other
- San Gerardo Hospital - Monza
- Gunma University Hospital - Gunma
- Tohoku University Hospital - Miyagi
- Tokyo Women's Medical University - Tokyo
Florida
- University of Florida Clinical Research Center - Gainesville
- Wolfson Children's Hospital - Jacksonville
Georgia
- Woodruff Memorial Research Building - Atlanta
Missouri
- St. Louis Children's Hospital - St Louis
North Carolina
- Duke University Medical Center - Durham
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
Pennsylvania
- UPMC Children's Hospital of Pittsburgh - Pittsburgh
Utah
- University of Utah, Clinical and Translational Sciences Institute - Salt Lake City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 21 participants |
| Start Date | 2020-02-13 |
| Est. Completion | 2026-06 |
| Phase | Phase 3 |
What the registry record for NCT03911505 still lists
NCT03911505 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 21 participants, a relatively small participant target, below the 45-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (53% lower).
The record links to 1 condition, with Pompe Disease (Late-onset) appearing as the primary indexed condition, and to 2 interventions - of which Miglustat is the first listed.
NCT03911505 lists 17 locations in 13 states (Other, Florida, Georgia).
Frequently Asked Questions
What is clinical trial NCT03911505 about?
NCT03911505 is a clinical study titled "ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD". This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to \< 18 years
What is the current status of trial NCT03911505?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 21 participants. The study started on 2020-02-13. Estimated completion is 2026-06.
What conditions does trial NCT03911505 study?
This clinical trial studies the following conditions: Pompe Disease (Late-onset).
What interventions are being tested in trial NCT03911505?
The interventions under investigation include: Miglustat (DRUG), Cipaglucosidase Alfa (BIOLOGICAL).
Who is sponsoring clinical trial NCT03911505?
This trial is sponsored by Amicus Therapeutics, which has 34 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03911505 being conducted?
This trial has 17 study locations across Florida, Georgia, Missouri, North Carolina, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Pompe Disease (Late-onset)
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT03911505's enrollment target sits among peer trials
21 4th of 8 higher than 5 of 8 other Pompe Disease (Late-onset) trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Pompe Disease (Late-onset) trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
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Gene Transfer Study in Patients With Late Onset Pompe Disease
ACTIVE NOT RECRUITING · Phase 1
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