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ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
NCT03911505 · View on ClinicalTrials.gov ↗
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03911505
The ClinicalTrials.gov registry entry for NCT03911505 describes a study currently listed as active not recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 21 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Amicus Therapeutics, which has 14 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03911505 reports 17 study locations spanning 13 distinct geographic areas — top geographies include Other, Florida, Georgia. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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). These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT03911505?
The interventions under investigation include: Miglustat (DRUG), Cipaglucosidase Alfa (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03911505?
This trial is sponsored by Amicus Therapeutics, which has 14 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
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