A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis
Recruiting
Phase 1
Phase 2
Interventional Study
Lysosomal Diseases
Gangliosidosis
GM1
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 6 Months – 12
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 54 (estimated)
- Sponsor
- National Human Genome Research Institute (NHGRI) · NIH
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About This Trial
Background:
GM1 gangliosidosis is a disorder that destroys nerve cells. It is fatal. There is no treatment. People with GM1 are deficient in a certain enzyme. A gene therapy may help the body make this enzyme. This could improve GM1 symptoms.
Objective:
To test if a gene therapy helps Type I and Type II GM1 gangliosidosis symptoms.
Eligibility:
Type I subjects will be male and female \>= 6 mo…
Trial Locations
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Eligibility Criteria
* INCLUSION CRITERIA:
Type I subjects
* Male or female subjects \>= 6 months old and \<= 12 months old at time of full ICF signing
* Biallelic mutations in GLB1
* Documented deficiency of Beta-galactosidase enzyme by clinical laboratory testing
* Phenotype consistent with a diagnosis of Type I GM1…
Contacts