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Mucopolysaccharidosis clinical trials at UCSF

10 in progress, 5 open to eligible people

Showing trials for
  • 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 Allievex in this study.

    Oakland, California and other locations

  • A Study of DNL310 in Pediatric Subjects With Hunter Syndrome

    open to eligible males ages 2-18

    This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL310, an investigational central nervous system (CNS)-penetrant enzyme replacement therapy (ERT), designed to treat both the peripheral and CNS manifestations of Mucopolysaccharidosis type II (MPS II; Hunter syndrome). The study has two staggered cohorts: the first will enroll subjects with neuronopathic MPS II aged 5 to 10 years; and the second will enroll subjects with MPS II, either neuronopathic or non-neuronopathic, aged 2 to 18 years. Subjects, whose physicians feel they are deriving benefit, will have the opportunity to be reconsented into a safety extension for continued evaluation, if implemented.

    Oakland, California and other locations

  • A Study of Potential Treatment-Responsive Biomarkers in Hunter Syndrome

    open to eligible people ages 2-30

    This is an observational, Pre-phase 1 study of biomarkers in patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.

    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 Treatment Extension Study of Mucopolysaccharidosis Type IIIB

    Sorry, in progress, not accepting new patients

    The primary objectives of this study are to evaluate the long-term safety and tolerability of AX 250 administered to subjects with MPS IIIB by an implanted ICV reservoir and catheter and to evaluate the impact of long-term AX 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-318 in Subjects With MPS I

    Sorry, in progress, not accepting new patients

    The purpose of the study is to evaluate the safety, tolerability of ascending doses of SB-318. SB-318 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the α-L-iduronidase (IDUA) gene into the Albumin locus in hepatocytes with the goal of lifelong therapeutic production of the IDUA enzyme.

    Oakland, California

  • 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

  • In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

    Sorry, not yet accepting patients

    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

  • Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

    Sorry, in progress, not accepting new patients

    This extension study will allow participants that completed Study HGT-HIT-094 to continue receiving Elaprase treatment in conjunction with idursulfase IT or to continue receiving Elaprase treatment and begin concurrent IT treatment for those that did not receive idursulfase IT treatment in Study HGT-HIT-094.

    Oakland, California and other locations

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