Skip to main content

Pompe Disease clinical trials at UCSF

4 in progress, 3 open to eligible people

Showing trials for
  • In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

    open to eligible females ages 18-50

    The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

    San Francisco, California

  • ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

    open to eligible people ages 0-17

    This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to < 18 years

    Oakland, 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.

    Oakland, California and other locations

  • Expanded Access for ATB200/AT2221 for the Treatment of IOPD

    Sorry, not accepting new patients

    This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.

    Oakland, California and other locations

Our lead scientists for Pompe Disease research studies include .

Last updated: