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Mucopolysaccharidosis clinical trials at UCSF

13 in progress, 7 open to eligible people

Showing trials for
  • A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II

    open to eligible males

    A Global Phase III multicenter, randomized, assessor-blinded, active-controlled designed to evaluate safety and efficacy of study drug for the treatment of the MPS II.

    Oakland, California and other locations

  • A Study of DNL310 in Pediatric Participants With Hunter Syndrome

    open to eligible males ages up to 18 years

    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). Participants, whose physicians feel they are deriving benefit, will have the opportunity to be reconsented into a safety extension and then an open-label extension for continued evaluation.

    Oakland, California and other locations

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

    open to eligible people ages up to 30 years

    This is a four-part prospective, multicenter, multiregional observational study of patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, to assess biomarkers potentially related to disease severity and/or treatment response and prospectively assess the progression of disease in participants with MPS II who are aged 2 through 10 years (Part 1), 2 through 30 years (Part 2), < 8 years (Part 3), and 6 to < 17 years (Part 4) at the time of enrollment.

    Oakland, California and other locations

  • An Extension Study of JR-171-101 Study in Patients With MPS I

    open to all eligible people

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

    Oakland, California and other locations

  • 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

  • RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

    open to eligible males ages 5-17

    RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a phase I/II study to determine whether RGX-121 is safe, well tolerated, and potentially effective in children five years of age and over who have severe MPS II.

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

    Sorry, in progress, not accepting new patients

    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 JR-171 in Patients With Mucopolysaccharidosis I

    Sorry, in progress, not accepting new patients

    Phase I/II, open-label, multicenter, multinational (Japan, Brazil and US),designed to evaluate the safety, pharmacokinetics and explore the efficacy for the treatment of mucopolysaccharidosis type I (MPS I).

    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

  • Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

    Sorry, accepting new patients by invitation only

    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

  • Mucopolysaccharidosis Type II Observational

    Sorry, not currently recruiting here

    This is an observational study planned to document prospectively disease manifestation and neurocognitive course in pediatric patients with a clinical presentation consistent with neuronopathic ("severe") MPS II undergoing current standard of care and/or intrathecal Elaprase® for their condition. Some patients may be offered the opportunity to screen for a gene therapy study conducted by the same sponsor.

    Oakland, California and other locations

  • 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

Our lead scientists for Mucopolysaccharidosis research studies include .

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