A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa (NCT03019406)
An Open-label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of Avalglucosidase Alfa (NeoGAA, GZ402666) in Patients With Infantile-onset Pompe Disease Treated With Alglucosidase Alfa Who Demonstrate Clinical Decline or Sub-optimal Clinical Response
🩺 Plain-English Summary (8th-Grade Level)
This is a multi-stage, phase 2, open-label, multicenter, multinational, ascending dose cohort, repeated intravenous (IV) infusion study of avalglucosidase alfa in pediatric patients with Infantile-Onset Pompe Disease (IOPD) who have been previously treated with alglucosidase alfa for a minimum of 6 months immediately prior to study entry and have demonstrated clinical decline or unsatisfactory clinical response.
Who can join: Review the eligibility criteria below.
Location: Regional Medical Genetics Center of New York Site Number : 8400002 — Valhalla, New York
Age range: 6 Months to 17 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (12 Sites)
- Regional Medical Genetics Center of New York Site Number : 8400002 — Valhalla, New York
- Duke University Medical Center Site Number : 8400001 — Durham, North Carolina
- Seattle Childrens Hospital and Regional Medical Center- Site Number : 8400005 — Seattle, Washington
- Investigational Site Number : 2500004 — Nantes,
- Investigational Site Number : 2500003 — Paris,
- Investigational Site Number : 2500002 — Paris,
- Investigational Site Number : 2500001 — Tours,
- Investigational Site Number : 3920001 — Fuchu-shi, Tokyo
- Investigational Site Number : 3920002 — Fuchu-shi, Tokyo
- Investigational Site Number : 1580001 — Hsinchu,
- Investigational Site Number : 8260001 — London, London, City of
- Investigational Site Number : 8260002 — Manchester,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
Explore by condition: All Glycogen Storage Disease Type II-Pompe's Disease clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.