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.
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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

Jean M Johnston

(240) 515-1448

johnstonjm@mail.nih.gov

CONTACT

Cynthia J Tifft, M.D.

(301) 451-8485

cynthiat@mail.nih.gov

CONTACT