Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
Public ClinicalTrials.gov record NCT04910776. 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, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants With Infantile-Onset Pompe Disease (IOPD)
Study identification
- NCT ID
- NCT04910776
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Lead sponsor
- Sanofi
- Industry
- Enrollment
- 17 participants
Conditions and interventions
Conditions
Interventions
- avalglucosidase alfa Drug
Drug
Eligibility (public fields only)
- Age range
- 0 Days to 12 Months
- 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
- Aug 31, 2021
- Primary completion
- Mar 1, 2026
- Completion
- Aug 9, 2027
- Last update posted
- Apr 7, 2026
2021 – 2027
United States locations
- U.S. sites
- 6
- U.S. states
- 6
- U.S. cities
- 6
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Stanford Hospital- Site Number : 8400006 | Stanford | California | 94305 | — |
| Children's Hospitals and Clinics of Minnesota- Site Number : 8400008 | Minneapolis | Minnesota | 55404 | — |
| Advanced Medical Genetics- Site Number : 8400002 | Hawthorne | New York | 10532 | — |
| Duke University Medical Center- Site Number : 8400004 | Durham | North Carolina | 27710 | — |
| Cincinnati Children's Hospital Medical Center- Site Number : 8400001 | Cincinnati | Ohio | 45229 | — |
| Seattle Children's Hospital- Site Number : 8400003 | Seattle | Washington | 98105 | — |
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 10 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04910776, 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 Apr 7, 2026 · Synced May 20, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04910776 live on ClinicalTrials.gov.