ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
Public ClinicalTrials.gov record NCT03911505. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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
Study identification
- NCT ID
- NCT03911505
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 21 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 0 Years to 17 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Feb 13, 2020 (Actual)
- Primary completion
- Mar 2027 (Estimated)
- Completion
- Sep 2027 (Estimated)
- Last update posted
- Sep 17, 2026
2020 – 2027
United States locations
- U.S. sites
- 9
- U.S. states
- 8
- U.S. cities
- 9
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Florida Clinical Research Center | Gainesville | Florida | 32610 | — |
| Wolfson Children's Hospital | Jacksonville | Florida | 32207 | — |
| Woodruff Memorial Research Building | Atlanta | Georgia | 30322 | — |
| St. Louis Children's Hospital | St Louis | Missouri | 63110 | — |
| Duke University Medical Center | Durham | North Carolina | 27710 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | 15224 | — |
| University of Utah, Clinical and Translational Sciences Institute | Salt Lake City | Utah | 84108 | — |
| Lysosomal and Rare Disorders Research and Treatment Center, Inc. | Fairfax | Virginia | 22030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 8 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03911505, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Sep 17, 2026 · Synced Oct 21, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT03911505 live on ClinicalTrials.gov.