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Mucopolysaccharidosis clinical trials at UCSF
6 in progress, 3 open to eligible people

  • A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

    open to eligible people ages up to 18 years

    This is a natural history study for children up to 18 years of age who have been diagnosed with Mucopolysaccharidosis Type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B). Mucopolysaccharidosis type IIIB is a severe neurodegenerative disorder. The information gathered from this trial may help inform the design and interpretation of subsequent interventional studies. No clinical intervention or study drug is provided by BioMarin in this study.

    Oakland, California and other locations

  • Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)

    open to all eligible people

    The objectives of this program are: to further characterize the natural progression of MPS VI disease; to generate and disseminate information on the care and management of MPS VI patients to clinical and medical professionals; to provide a resource to physicians and patients by providing information for optimizing patient care based on aggregate data; to characterize the clinical response to long-term Naglazyme® (galsulfase) treatment; to further characterize the long-term safety of Naglazyme® treatment.

    Oakland, California and other locations

  • RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome)

    open to eligible males ages 4 months to 5 years

    RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a safety and dose ranging study to determine whether RGX-121 is safe and tolerated by patients with MPS II.

    Oakland, California and other locations

  • A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

    Sorry, in progress, not accepting new patients

    Mucopolysaccharidosis type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B) is a severe neurodegenerative disorder. The purpose of this study is to learn more about the health problems in patients with MPS IIIB and how to measure these problems over time. It will particularly look at how the disease develops in young children. This is an observational study, so no experimental drug will be given. The results from this study will help us design future studies to measure whether these health problems get better when we give experimental drug for MPS IIIB.

    Oakland, California and other locations

  • A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

    Sorry, accepting new patients by invitation only

    The primary objectives of this study are to evaluate the long-term safety and tolerability of BMN 250 administered to subjects with MPS IIIB by an implanted ICV reservoir and catheter and to evaluate the impact of long-term BMN 250 treatment on cognitive function in patients with MPS IIIB as assessed by developmental quotient (DQ).

    Oakland, California and other locations

  • Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

    Sorry, in progress, not accepting new patients

    The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913. SB-913 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the IDS gene into the Albumin locus in hepatocytes with the goal of lifelong therapeutic production of the IDS enzyme.

    Oakland, California and other locations