Sanfilippo Syndrome clinical trials at UCSF

5 in progress, 1 open to eligible people

Showing trials for

  • JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)

    open to eligible people ages up to 5 years

    This is a global, open-label, Phase I/II, interventional trial in participants younger than 6 years of age with Mucopolysaccharidosis Type IIIB (MPS IIIB), designed to assess the safety and tolerability of JR-446, determine its pharmacodynamic effects, and explore its potential to demonstrate early clinical effects on disease-relevant outcomes.

    Oakland, California and other locations

  • Early Access Treatment With Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B)

    Sorry, not accepting new patients

    This is an intermediate-size patient population Early Access Program (EAP) providing access to intracerebroventricular (ICV) tralesinidase alfa (TA) for participants with mucopolysaccharidosis type IIIB (MPS IIIB, Sanfilippo Syndrome Type B). The primary objectives are to allow early access to TA treatment and to evaluate the safety and tolerability of TA ICV infusion. Approximately 10 participants will be enrolled at up to 3 US sites and treated with TA once weekly via ICV infusion for up to approximately 52 weeks, or until TA becomes commercially available.

    Oakland, California

  • DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

    Sorry, in progress, not accepting new patients

    This is a multicenter, open-label, Phase 1/2 study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and clinical efficacy of DNL126 in participants with Sanfilippo syndrome Type A (MPS IIIA). The core study period is 25 weeks (approximately 6 months); followed by an open-label extension (OLE), which extends through Week 97 (approximately 18 months); and a long-term extension (LTE), which extends through Week 193 (Year 4). Participants with MPS IIIA will be enrolled in two planned cohorts, and additional participants with MPS IIIA may be enrolled in three optional cohorts.

    Oakland, California and other locations

  • GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)

    Sorry, in progress, not accepting new patients

    The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device in patients with Sanfilippo Syndrome Type A (MPS IIIA).

    Oakland, California and other locations

  • ICV AX 250 Treatment in MPS IIIB -OLE

    Sorry, accepting new patients by invitation only

    This is a Phase 3B/4, multicenter, multinational, open label study to further evaluate intracerebroventricular (ICV) delivered AX 250 treatment in MPS IIIB subjects that complete Study 250-202 for up to an additional 3 years (144 weeks) of treatment with AX 250 administered by ICV infusion every other week. Subjects will be evaluated for neurocognitive function, communication, adaptive behavior, quality of life, imaging characteristics and biochemical markers of disease burden. Safety will be assessed by adverse events, clinical labs, and physical exams.

    Oakland, California and other locations

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