NeuroGT
The goal of this clinical trial is to learn if one infusion of rAAV9-CMV-hNAGLUop can treat patients with Sanfilippo syndrome (MPS III) 6 months or older. The main questions it aims to answer are: * Is the treatment safe? * Is there a change in enzyme activities,? * Is there a change in natural history trajectory of motor, language, feeding, adaptive and cognitive function? Participants will be asked to come to Washington University St. Louis and stay in the hospital overnight following the infusion. Partcipant and caregiver(s) will be asked to stay near the hospital for the first month and come back periodically for clinic visits.
This is a dose escalation study where one infusion of rAAV9-CMV-hNAGLUop will be administered to patients with Sanfilippo Disease (MPS III). Patients will go through an Informed Consent process and eligibility assessed by reviewing patient medical history and laboratory values. Eligible patients will receive the infusion of rAAV9-CMV-hNAGLUop followed by an inpatient stay in the hospital for at least one night. Over the next 30 days, participants and their caregiver(s) will stay in a home near the hospital to facilitate visits back to the hospital. Participants will have laboratory samples c…
Inclusion Criteria: * Age \>6 months * Confirmed diagnosis of MPS IIIB based upon meeting the following conditions: * No detectable or significantly reduced N-acetyl-α-glucosaminidase (NAGLU) enzyme activity by leukocyte assay from CLIA-certified laboratory * Two variants classified as pathogenic or likely pathogenic in NAGLU on clinical laboratory testing. Variants will be interpreted using the American College of Medical Genetics guidelines for the interpretation of sequence variants and testing must be performed by a CLIA-certified laboratory. * Clinical history of developmental delay…
Systemic rAAV9-CMV-hNAGLUop gene delivery given once
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