Pompe Disease clinical trials at UC Irvine

5 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

  • Pompe Disease Registry Protocol

    open to all eligible people

    The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, and for other research and non-research related purposes. The objectives of the Registry are: - To enhance understanding of the variability, progression, identification, and natural history of Pompe disease, with the ultimate goal of better guiding and assessing therapeutic intervention. - To assist the Pompe medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care. - To characterize the Pompe disease population. - To evaluate the long-term effectiveness of alglucosidase alfa.

    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

  • 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 .

Last updated: