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

Conditions

• Sanfilippo Syndrome (MPS III)

Eligibility Criteria

Sex

ALL

Age

6 Months - N/A

Healthy Volunteers

Not accepted

Interventions

• rAAV9-CMV-hNAGLUop — BIOLOGICAL
  Systemic rAAV9-CMV-hNAGLUop gene delivery given once

Study Details

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.

Key Dates

First listed

Sep 14, 2026

Start date

Jan 18, 2027

Status verified

Sep 2026

Primary completion

Dec 31, 2028

Completion

Dec 31, 2029

Study Design

Enrollment

9 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Cohort 1
  rAAV9-CMV-hNAGLUop Low Dose
• Experimental: Cohort 2
  rAAV9-CMV-hNAGLUop High Dose

Primary Outcome Measure

Assessment of Safety [ Time Frame: 24 months ]

Central Contacts

• Amy Fadden
  980-297-5490
  [email protected]

Locations (1)

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