Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
Condition(s) studied
Investigational drug(s) / intervention(s)
avalglucosidase alfa: Sterile lyophilized powder intravenous (IV) infusion
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
Eligibility
Primary outcome measure(s)
- Proportion of participants who are alive and free of invasive ventilation at Week 52 — Week 52
Trial sites (16)
| Facility | City | Region | Status |
|---|---|---|---|
| Stanford Hospital- Site Number : 8400006 | Stanford | California | |
| Children's Hospitals and Clinics of Minnesota- Site Number : 8400008 | Minneapolis | Minnesota | |
| Advanced Medical Genetics- Site Number : 8400002 | Hawthorne | New York | |
| Duke University Medical Center- Site Number : 8400004 | Durham | North Carolina | |
| Cincinnati Children's Hospital Medical Center- Site Number : 8400001 | Cincinnati | Ohio | |
| Seattle Children's Hospital- Site Number : 8400003 | Seattle | Washington | |
| Investigational Site Number : 0560001 | Leuven | Belgium | |
| Investigational Site Number : 1560002 | Qingdao | China | |
| Investigational Site Number : 1560001 | Shanghai | China | |
| Investigational Site Number : 2760001 | Bad Oeynhausen | Germany | |
| Investigational Site Number : 3800002 | Monza | Lombardy | |
| Investigational Site Number : 5280001 | Rotterdam | Netherlands | |
| Investigational Site Number : 7240001 | Esplugues de Llobregat | Catalunya [Cataluña] | |
| Investigational Site Number : 1580001 | Taipei | Taiwan | |
| Investigational Site Number : 8260001 | London | London, City of | |
| Investigational Site Number : 8260002 | Manchester | United Kingdom |
More Sanofi trials in the UK
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT04910776 on ClinicalTrials.gov ↗ ← All trials in the UK