Cure My Disease

Don't miss your cure!

 Log in / Sign up

New hope for kids with pompe: enzyme combo trial underway

NCT ID NCT03911505

Ongoing Disease control Sponsor: Amicus Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a new treatment called cipaglucosidase alfa/miglustat in children (0 to under 18 years) with late-onset Pompe disease. The goal is to see if it is safe and how well it works. Participants either have never had enzyme therapy before or have been on it for at least 6 months. The treatment aims to control the disease, not cure it.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for POMPE DISEASE LATE-ONSET are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

glycogen storage disease due to acid maltase deficiency, late-onset glycogen storage disease II Glycogen Storage Disease Type II

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

POMPE DISEASE LATE-ONSET

Contacts and locations

Locations

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Gunma University Hospital

    Gunma, 371-8511, Japan

  • Izumi City General Hospital

    Osaka, Izumi-Shi, 594-0073, Japan

  • Lysosomal and Rare Disorders Research and Treatment Center, Inc.

    Fairfax, Virginia, 22030, United States

  • San Gerardo Hospital

    Monza, 20900, Italy

  • SphinCS GmbH Clinical Science for LSD

    Hochheim am Main, Hesse, 65239, Germany

  • St. Louis Children's Hospital

    St Louis, Missouri, 63110, United States

  • Tohoku University Hospital

    Miyagi, 980-8574, Japan

  • Tokyo Women's Medical University

    Tokyo, 162-8666, Japan

  • UPMC Children's Hospital of Pittsburgh

    Pittsburgh, Pennsylvania, 15224, United States

  • University of Calgary

    Calgary, Alberta, T3B 6A8, Canada

  • University of Florida Clinical Research Center

    Gainesville, Florida, 32610, United States

  • University of Utah, Clinical and Translational Sciences Institute

    Salt Lake City, Utah, 84108, United States

  • Wolfson Children's Hospital

    Jacksonville, Florida, 32207, United States

  • Women's and Children's Hospital

    North Adelaide, South Australia, 5006, Australia

  • Woodruff Memorial Research Building

    Atlanta, Georgia, 30322, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.