Sanfilippo Syndrome clinical trials at University of California Health
5 in progress, 2 open to eligible people
GC1130A in Pediatric Patients with Sanfilippo Syndrome Type a (MPS IIIA)
open to eligible people ages 24 months to 72 months
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 pediatric patients …
at UCSF
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…
at UCSF
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 …
at UCSF
Cannabidiol in Sanfilippo Syndrome
Sorry, not yet accepting patients
The goal of this clinical trial is to test cannabidiol in Sanfilippo syndrome. The main questions it aims to answer are: 1) determine the safety of cannabidiol in Sanfilippo syndrome, and 2) explore the efficacy of cannabidiol in treating the…
at UCLA
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…
at UCSF
Our lead scientists for Sanfilippo Syndrome research studies include Paul Harmatz, MD Lynda E Polgreen.
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