A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
Active, not recruiting · NCT04848779
Conditions: Glycogen Storage Disease Type II
At a glance
- Research payments reported, 2025
- $108,313
- U.S. investigators named
- 5
- Sites paid
- 5
- Planned or actual enrollment
- 16
About the trial
- Sponsor
- Sanofi
- Tested
- Alglucosidase alfa GZ419829 (drug)
- Dates
- Started 2021-06-10; completion 2026-10-28
- Results posted
- Not yet
Primary Objective: To describe the effect of routine practice with alglucosidase alfa in patients with IOPD ≤6 months of age, on invasive ventilation-free survival after 52 weeks of treatment. Secondary Objectives: * To describe the effect of routine practice with alglucosidase alfa on invasive ventilation-free survival and survival at 12 and 18 months of age, as well as on change in left ventricular mass (LVM) Z score, Alberta Infant Motor Scale (AIMS) score, body weight, body length, and head circumference Z scores, and urinary glucose tetrasaccharide (Hex4), at Week 52 of treatment. * To describe the safety, tolerability, and immunogenicity of alglucosidase alfa in the routine practice…
From ClinicalTrials.gov, last updated 2026-06-24. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Genzyme Corporation reported $108,313 in research payments for this study in 2025 (22 payments).
Products named: Lumizyme
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
- Priya Kishnani Pediatrics · Durham, NC
- David F. Kronn, MD Medical Genetics · Valhalla, NY
- Robert J. Hopkin, M.D. Medical Genetics · Cincinnati, OH
- Jeffrey Towbin, MD Heart Failure & Transplant Cardiology · Memphis, TN
- 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
- Duke University Gsrb1 4th Fl $67,215
- Cincinnati Children's Hospital Medical Center, OH $18,400
- Le Bonheur Children S Hospital Foundation $15,579
- New York Medical College Office of Research Administration Skyline Rm J23 $4,650
- Seattle Children's Hospital, WA $2,469
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.