Pompe Disease clinical trials at UC Irvine

4 in progress, 2 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

  • Global Prospective Observational Registry of Patients With Pompe Disease

    open to all eligible people

    This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both untreated patients and those being treated with an approved therapy for Pompe disease are eligible to participate. The objectives of the registry are: - To evaluate the long-term safety of Pompe disease treatments through collection of data that describe the frequency of adverse events (AEs)/serious adverse events (SAEs) occurring in Pompe disease patients - To evaluate the long-term real-world effectiveness of Pompe disease treatments - To evaluate the long-term real-world impact of Pompe disease treatments on quality of life (QOL) and patient-reported outcomes (PROs) - To describe the natural history of untreated Pompe disease

    Irvine, 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

Our lead scientists for Pompe Disease research studies include .

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