ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD (NCT03911505)
An Open-label Study of the Safety, Pharmacokinetics, Efficacy, Pharmacodynamics, and Immunogenicity of Cipaglucosidase Alfa/Miglustat in Pediatric Subjects Aged 0 to < 18 Years With Late-onset Pompe Disease
🩺 Plain-English Summary (8th-Grade Level)
This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to \< 18 years
Who can join: Review the eligibility criteria below.
Location: University of Florida Clinical Research Center — Gainesville, Florida
Age range: 0 Years 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 (17 Sites)
- University of Florida Clinical Research Center — Gainesville, Florida
- Wolfson Children's Hospital — Jacksonville, Florida
- Woodruff Memorial Research Building — Atlanta, Georgia
- St. Louis Children's Hospital — St Louis, Missouri
- Duke University Medical Center — Durham, North Carolina
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania
- University of Utah, Clinical and Translational Sciences Institute — Salt Lake City, Utah
- Lysosomal and Rare Disorders Research and Treatment Center, Inc. — Fairfax, Virginia
- Women's and Children's Hospital — North Adelaide, South Australia
- University of Calgary — Calgary, Alberta
- SphinCS GmbH Clinical Science for LSD — Hochheim am Main, Hesse
- San Gerardo Hospital — Monza,
- Izumi City General Hospital — Osaka, Izumi-Shi
- Gunma University Hospital — Gunma,
- Tohoku University Hospital — Miyagi,
- Tokyo Women's Medical University — Tokyo,
💰 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.
More studies in Pompe Disease (Late-onset)
- A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease — Atlanta, Georgia
- Gene Transfer Study in Patients With Late Onset Pompe Disease — Orange, California
- A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late-Onset Pompe Disease (PROGRESS-GT LOPD) — Phoenix, Arizona
Explore by condition: All Pompe Disease (Late-onset) 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.