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One-Time gene therapy could change pompe disease treatment

NCT ID NCT07282847

Recruiting now Disease control Sponsor: AskBio Inc Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 21, 2026 · Updated 2 times

Summary

This study tests a single intravenous dose of a gene therapy called AB-1009 in 12 adults with late-onset Pompe disease. Participants must have been on enzyme replacement therapy for at least 6 months. The main goal is to check safety and side effects, while also seeing if the treatment helps control the disease.

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Conditions

The condition(s) this trial relates to.

disorder of glycogen metabolism glycogen storage disease due to acid maltase deficiency, late-onset Glycogen Storage Disease Type II late-onset Parkinson disease lysosomal storage disease neuromuscular disease

As listed by the trial registrant

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

LOPD POMPE DISEASE LATE-ONSET

Contacts and locations

Locations

  • Barrow Neurological Institute

    NOT_YET_RECRUITING

    Phoenix, Arizona, 85013, United States

  • Duke University

    RECRUITING

    Durham, North Carolina, 27705, United States

  • Hospital of the University of Pennsylvania

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • NYU Langone

    NOT_YET_RECRUITING

    New York, New York, 10016, United States

  • Stanford Neuroscience Health Center

    NOT_YET_RECRUITING

    Palo Alto, California, 94304, United States

  • University of California, Irvine (UCI)

    RECRUITING

    Irvine, California, 92697, United States

  • University of Pittsburgh Medical Center (UPMC)

    NOT_YET_RECRUITING

    Pittsburgh, Pennsylvania, 15213, United States

  • University of Texas Southwest Medical Center

    RECRUITING

    Dallas, Texas, 75235, United States

    Contact Email: •••••@•••••

    Contact Email: •••••@•••••

  • Virginia Commonwealth University (VCU)

    NOT_YET_RECRUITING

    Richmond, Virginia, 23298, United States

More trials for these conditions

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