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Gene Therapy for Sanfilippo Syndrome

AF
Overseen ByAmy Fadden
Age: Any Age
Sex: Any
Trial Phase: Phase 1 & 2
Sponsor: NeuroGT
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

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.

Are You a Good Fit for This Trial?

This trial is for patients aged 6 months or older diagnosed with Sanfilippo syndrome (MPS IIIB). Participants must be able to travel to Washington University in St. Louis and stay nearby for a month after treatment. Other specific medical exclusions may apply.

Inclusion Criteria

I am older than 6 months.
Clinical history of developmental delays defined as a score >1 standard deviation below the mean in at least one domain of neuropsychological function (language, memory, non-verbal ability), OR documented historical evidence of a decline of >1 standard deviation on sequential testing, OR a score between 0.75 and 1 standard deviation below the mean with cognitive defect affecting daily performance
I have MPS IIIB with two disease-causing NAGLU gene changes confirmed by lab testing.
See 1 more

Exclusion Criteria

I do not have a fever or take medications that increase my risk for gene therapy.
Anti-AAV9 antibody titers > 1:100 by binding ELISA assay
I have had an infection or vaccine in the past 6 weeks.
See 9 more

What Are the Treatments Tested in This Trial?

Interventions

  • rAAV9-CMV-hNAGLUop

Trial Overview

The study tests a single intravenous infusion of rAAV9-CMV-hNAGLUop, a gene therapy designed to treat Sanfilippo syndrome (MPS IIIB), by seeing if it is safe and whether it improves enzyme activity and developmental functions.

How Is the Trial Designed?

2

Treatment groups

Experimental Treatment

Group I: Cohort 2Experimental Treatment1 Intervention
Group II: Cohort 1Experimental Treatment1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

NeuroGT

Lead Sponsor

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