Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
Active, not recruiting · Phase 3 · NCT04910776
Conditions: Glycogen Storage Disease Type II
At a glance
- Research payments reported, 2025
- $665,445
- U.S. investigators named
- 5
- Sites paid
- 5
- Planned or actual enrollment
- 17
About the trial
- Sponsor
- Sanofi
- Tested
- avalglucosidase alfa (drug)
- Dates
- Started 2021-09-01; completion 2027-08-10
- Results posted
- Not yet
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
From ClinicalTrials.gov, last updated 2026-04-08. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Genzyme Corporation reported $665,445 in research payments for this study in 2025 (43 payments).
Products named: Nexviazyme
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Ana C. Tesi Rocha, MD Physical Medicine & Rehabilitation (PM&R) · Palo Alto, CA
- Vikas Bhambhani, M.D Medical Genetics · Minneapolis, MN
- Priya Kishnani Pediatrics · Durham, NC
- Robert J. Hopkin, M.D. Medical Genetics · Cincinnati, OH
- Sihoun Hahn, MD, PHD Medical Genetics · Seattle, WA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Cincinnati Children's Hospital Medical Center, OH $476,341
- Duke University Gsrb1 4th Fl $97,459
- Childrens Hospitals and Clinics Minnesota $49,392
- Seattle Children's Hospital, WA $34,266
- Board of Trustees of the Leland $7,987
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.