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Pompe Disease clinical trials at UC Irvine

4 in progress, 1 open to eligible people

Showing trials for
  • Gene Transfer Study in Patients With Late Onset Pompe Disease

    open to eligible people ages 18-80

    This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).

    Orange, California and other locations

  • Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

    Sorry, in progress, not accepting new patients

    The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.

    Orange, California and other locations

  • Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

    Sorry, in progress, not accepting new patients

    This is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.

    Irvine, California and other locations

  • First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

    Sorry, in progress, not accepting new patients

    This study is an international, multi-center, study of Pompe disease patients that are currently receiving enzyme-replacement therapy (ERT). The purpose of this study is to find out if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.

    Orange, California and other locations

Our lead scientists for Pompe Disease research studies include .

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