Sanfilippo Syndrome clinical trials at UCSF
4 in progress, 1 open to eligible people
Sanfilippo syndrome is a rare genetic disorder that affects children. UCSF is running several clinical trials to study the safety and effects of new treatments for this condition. These trials involve young patients to better understand the disease and potential therapies.
DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
open to eligible people ages 0-18
This is a multicenter, open-label, Phase 1/2 study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and exploratory 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, California and other locations
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
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
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
Our lead scientists for Sanfilippo Syndrome research studies include Paul Harmatz, MD.
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