Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
NCT04910776 · View on ClinicalTrials.gov ↗
Study Summary
This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD. Study details include: * Study duration: Screening - up to 4 weeks; * Primary Analysis Period (PAP) - 52 weeks; * Extended Treatment Period (ETP) - 52 weeks; * Extended Long term Treatment Period (ELTP) - 104 weeks; 4-week follow-up period for a total study duration - up to 4.08 years. * Treatment duration: Up to 4 years * Visit frequency: every other week and potentially every week
Conditions Studied
Interventions
- DRUG avalglucosidase alfa
Study Locations (16)
Other
- Investigational Site Number : 0560001 — Leuven
- Investigational Site Number : 1560002 — Qingdao
- Investigational Site Number : 1560001 — Shanghai
- Investigational Site Number : 2760001 — Bad Oeynhausen
- Investigational Site Number : 5280001 — Rotterdam
- Investigational Site Number : 1580001 — Taipei
- Investigational Site Number : 8260002 — Manchester
California
- Stanford Hospital- Site Number : 8400006 — Stanford
Minnesota
- Children's Hospitals and Clinics of Minnesota- Site Number : 8400008 — Minneapolis
New York
- Advanced Medical Genetics- Site Number : 8400002 — Hawthorne
North Carolina
- Duke University Medical Center- Site Number : 8400004 — Durham
Ohio
- Cincinnati Children's Hospital Medical Center- Site Number : 8400001 — Cincinnati
Washington
- Seattle Children's Hospital- Site Number : 8400003 — Seattle
Lombardy
- Investigational Site Number : 3800002 — Monza
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 17 participants |
| Start Date | 2021-09-01 |
| Est. Completion | 2027-08-10 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04910776
The ClinicalTrials.gov registry entry for NCT04910776 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 17 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Sanofi, which has 275 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 Glycogen Storage Disease Type II appearing as the primary indexed condition, and to 1 intervention — of which avalglucosidase alfa 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: NCT04910776 reports 16 study locations spanning 10 distinct geographic areas — top geographies include Other, California, Minnesota. 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 NCT04910776 about?
NCT04910776 is a clinical study titled "Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa". This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD. Study details include: * Study duration: Screening - up to 4 weeks; * Primary Anal...
What is the current status of trial NCT04910776?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 17 participants. The study started on 2021-09-01. Estimated completion is 2027-08-10.
What conditions does trial NCT04910776 study?
This clinical trial studies the following conditions: Glycogen Storage Disease Type II. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04910776?
The interventions under investigation include: avalglucosidase alfa (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04910776?
This trial is sponsored by Sanofi, which has 275 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04910776 being conducted?
This trial has 16 study locations across California, Minnesota, New York, North Carolina, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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