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New hope for kids with rare pompe disease: enzyme combo under trial

NCT ID NCT04808505

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

First seen Nov 01, 2025 · Last updated May 24, 2026 · Updated 34 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 birth to under 18 years old, both those who have and haven't had prior enzyme therapy. The main goal is to check safety and see how 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

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

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    WITHDRAWN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