Single Injection of rAAV9-CMV-hNAGLUop Gene for Patients With Mucopolysaccharidosis (MPS) IIIB

Brief summary

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.

Source: ClinicalTrials.gov (U.S. National Library of Medicine), data processed 2026-10-29T09:00:05; modifications: selected fields only; whitespace normalized; brief summary truncated to 2,000 characters. Not a U.S. Government endorsement.

Trial details

status

NOT_YET_RECRUITING

phase

PHASE1, PHASE2

study type

INTERVENTIONAL

enrollment

9

sponsor

NeuroGT

conditions

Sanfilippo Syndrome (MPS III)

interventions

BIOLOGICAL: rAAV9-CMV-hNAGLUop

start date

2027-01-18

start date type

ESTIMATED

primary completion date

2028-12-31

has results

false

data source

ClinicalTrials.gov

data processed

2026-10-29T09:00:05

modifications

selected fields only; whitespace normalized; brief summary truncated to 2,000 characters