Gaucher Disease clinical trials at University of California Health

4 in progress, 3 open to eligible people

Showing trials for

  • PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

    open to eligible females ages 18-50

    For detailed information, please view our study website: https://pearltrial.ucsf.edu/ The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal…

    at UCSF

  • International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

    open to all eligible people

    The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status. No experimental intervention is…

    at UC Davis UC Irvine UCSD UCSF

  • Registry of Patients Diagnosed With Lysosomal Storage Diseases

    open to eligible people ages up to 64 years

    This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal…

    at UCSF

  • PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

    Sorry, in progress, not accepting new patients

    J3Z-MC-OJAB is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose LY3884961 (formerly PR001) in infants diagnosed with Type 2 Gaucher disease (GD2). For each patient, the study will be approximately 5…

    at UCSF

Our lead scientists for Gaucher Disease research studies include .

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