Mucopolysaccharidosis Type I clinical trials at UCSF

6 in progress, 4 open to eligible people

Mucopolysaccharidosis Type I is a condition where the body struggles to process certain substances. UCSF is exploring a new approach with modified B cells to treat adults with certain types of this disorder. Another initiative involves collecting long-term data from patients to learn more about the disease.

Showing trials for

  • ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

    open to eligible people ages 18 years and up

    A first-in-human study using ISP-001 in adult patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.

    Oakland, California and other locations

  • 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 Storage Diseases.

    San Francisco, California

  • Mucopolysaccharidosis I (MPS I) Registry

    open to all eligible people

    The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS I Registry will provide information to better characterize the natural history and progression of MPS I as well as the clinical responses of patients receiving enzyme replacement therapy, such as Aldurazyme (Recombinant Human Alpha-L-Iduronidase), or other treatment modalities. The objectives of the Registry are: - To evaluate the long-term effectiveness and safety of Aldurazyme® (laronidase) - To characterize and describe the MPS I population as a whole, including the variability, progression, and natural history of MPS I - To help the MPS I medical community with the development of recommendations for monitoring patients and reports on patient outcomes to optimize patient care

    Oakland, California and other locations

  • 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 management of patients with LSDs.

    San Francisco, California

  • JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)

    Sorry, in progress, not accepting new patients

    Phase I/II, open label, multicenter, multinational (Japan, Brazil and the US) extension study of JR-171-101 (NCT04227600) for the treatment of MPS I

    Oakland, California and other locations

  • (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

    Sorry, in progress, not accepting new patients

    Long-term follow-up of subjects who received SB-318, SB-913, or SB-FIX in a previous trial and completed at least 52 weeks post-infusion follow-up in their primary protocol. Enrolled subjects will be followed for a total of up to 10 years following exposure to SB-318, SB-913, or SB-FIX.

    Oakland, California and other locations

Our lead scientists for Mucopolysaccharidosis Type I research studies include .

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