New hope for kids with pompe: enzyme combo trial opens

NCT ID NCT04808505

Recruiting now Disease control Sponsor: Amicus Therapeutics Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 32 times

Summary

This study tests a new treatment (cipaglucosidase alfa/miglustat) for children with infantile-onset Pompe disease, a rare genetic disorder that causes muscle weakness and heart problems. The trial includes children from 6 months to under 18 years old who have either never received enzyme therapy or have been on it before. The main goal is to check safety, especially infusion-related reactions, and see how well the body handles the drug.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Study contacts

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Locations

AOU Federico II
RECRUITING

Naples, 80131, Italy
Cincinnati Children's Hospital Medical Center
RECRUITING

Cincinnati, Ohio, 54229, United States
Duke University Early Phase Research Unit
RECRUITING

Durham, North Carolina, 27710, United States
Erasmus MC, Sophia Kinderziekenhuis
RECRUITING

Rotterdam, 3015 GD, Netherlands
Great Ormond Street Hospital for Children NHS Foundation Trust
RECRUITING

London, WC1N3JH, United Kingdom
National Taiwan University Hospital
WITHDRAWN

Taipei, 100, Taiwan
SphinCS GmbH
RECRUITING

Höchheim, 65239, Germany
The Emory Clinic
RECRUITING

Atlanta, Georgia, 30322, United States
UPMC Hospital of Pittsburgh
RECRUITING

Pittsburgh, Pennsylvania, 15224, United States
University of Florida Clinical Research Center
RECRUITING

Gainesville, Florida, 32610, United States
University of Utah, Clinical and Translational Sciences Institute
RECRUITING

Salt Lake City, Utah, 84108, United States
Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie
RECRUITING

Giessen, 35392, Germany
Universitätsklinikum Heidelberg - Pädiatrisches Klinisch-Pharmakologisches Studienzentrum (paedKliPS)
RECRUITING

Heidelberg, 69120, Germany
Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus 1
RECRUITING

Münster, 48149, Germany

Conditions

Explore the condition pages connected to this study.

GLYCOGEN STORAGE DISEASE TYPE II INFANTILE ONSET GLYCOGEN STORAGE DISEASE TYPE II INFANTILE ONSET