Pompe's Disease (Infantile Onset)
Pompe's disease, particularly the infantile onset type, is a rare genetic disorder that leads to the buildup of glycogen in the body's cells, causing severe muscle weakness and respiratory issues in infants.
Infantile-onset Pompe Disease: Cipaglucosidase Alfa/Miglustat Study
We are evaluating a new treatment for children under 18 with infantile-onset Pompe disease to see if it is safe and effective. This study includes both children who have and have not received previous enzyme replacement therapy.
>2 yearsConfirmation phase (III)Post-Trial Drug AccessNo PlaceboCost ReimbursementPartially RemoteInvestigational MedicinesOralInjectionHematologyInternal MedicineNeurologyPediatrics
What diseases are being studied?
Health conditions and diseases that the clinical trial is designed to study and treat.
Who can participate in the clinical trial?
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
- Are you between 6 months and 18 years old?
- Are you currently pregnant or breastfeeding?
- Do you require respiratory assistance for 16 hours or more each day?
- Have you received any investigational drug for Pompe disease in the last 30 days?
- Do you have a history of serious allergic reactions to enzyme replacement therapy (ERT) medications?
What experimental substances are being tested?
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Atb200
At2221
Where is the clinical trial being conducted?
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
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Raymond Poincare Hospital
Pediatric intensive care unit and neurology
Vaucresson, France
Justus Liebig University Giessen
Abteilung für Kinderneurologie, Sozialpädiatrie und Epileptologie
Gießen, Germany
Universitaetsklinikum Heidelberg AöR
Pädiatrisches Klinisch-Pharmakologisches Studienzentrum (paedKliPS)
Heidelberg, Germany
Sponsor: Amicus Therapeutics Inc.
Last updated: Nov 27, 2025
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.