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Pompe disease study aims to clear path for gene therapy

NCT ID NCT06150820

Ongoing Knowledge-focused Sponsor: Astellas Gene Therapies Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 17, 2026 · Updated 1 time

Summary

This study looks at people with late-onset Pompe disease to measure antibodies against a virus used in gene therapy and against the standard enzyme replacement therapy. It also checks for biomarkers in blood and urine. About 119 teenagers and adults will give samples over 2 years, but no new treatment is given. The goal is to gather information that could help design better gene therapies in the future.

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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

  • AU61001

    Herston, Australia

  • AU61003

    Adelaide, Australia

  • BR55001

    São Paulo, Brazil

  • BR55002

    Porto Alegre, Brazil

  • BR55003

    Flamengo, Brazil

  • CA15001

    Montreal, Canada

  • CN15003

    Edmonton, Canada

  • Children's Hospitals and Clinics of Minnesota

    Minneapolis, Minnesota, 55404, United States

  • DT49003

    Höchheim, Germany

  • DT49004

    Essen, Germany

  • DT49005

    Bonn, Germany

  • DT49006

    Münster, Germany

  • ES34001

    Madrid, Spain

  • ES34002

    Valencia, Spain

  • ES34003

    Albacete, Spain

  • ES34004

    Barcelona, Spain

  • ES34005

    Madrid, Spain

  • ES34007

    L'Hospitalet de Llobregat, Spain

  • ES34009

    Donostia / San Sebastian, Spain

  • Emory Clinic

    Atlanta, Georgia, 30322, United States

  • FR33001

    Strasbourg, France

  • FR33002

    Marseille, France

  • FR33003

    Nantes, France

  • FR33004

    Nice, France

  • FR33005

    Lille, France

  • FR33006

    Angers, France

  • FR33007

    Limoges, France

  • FR33009

    Garches, France

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601, United States

  • IT39002

    Florence, Italy

  • IT39003

    Udine, Italy

  • IT39004

    Roma, Italy

  • IT39005

    Gussago, Italy

  • IT39006

    Pisa, Italy

  • IT39008

    Pavia, Italy

  • IT39009

    Milan, Italy

  • IT39011

    Milan, Italy

  • IT39012

    Messina, Italy

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

    Fairfax, Virginia, 22030, United States

  • National Center of Neurology and Psychiatry

    Kodaira-Shi, Japan

  • TW88601

    Taipei, Taiwan

  • TW88602

    Taipei, Taiwan

  • TW88603

    Taoyuan City, Taiwan

  • Tokyo Women's Medical University Hospital

    Shinjuku-Ku, Japan

  • UK44001

    Newcastle upon Tyne, United Kingdom

  • UK44003

    Cambridge, United Kingdom

  • UK44004

    Salford, United Kingdom

  • University of Cincinnati

    Cincinnati, Ohio, 45221, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109, United States

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Pittsburgh Medical Center

    Pittsburgh, Pennsylvania, 15213, United States

  • University of UTAH - PPDS

    Salt Lake City, Utah, 84132, United States

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