Sanfilippo Syndrome clinical trials at UCSF

4 in progress, 1 open to eligible people

Showing trials for

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

    open to eligible people ages 12 months to 18 years

    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 5378538, California 5332921 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 5378538, California 5332921 and other locations

  • 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) and is followed by a 72-week (approximately 18 month) open-label extension (OLE). 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 5378538, California 5332921 and other locations

  • 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 5378538, California 5332921 and other locations

Our lead scientists for Sanfilippo Syndrome research studies include .

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