Health Trials

GM1 Gangliosidoses

2

Review clinical trials related to GM1 Gangliosidoses. Use filters to narrow results by trial status, phase, treatment, biological sex and sponsor.

Condition / disease
Location
Status: Not yet recruiting

Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial

This is a study for the administration of in utero AAV9 transfer in prenatally diagnosed Type I or Type II GM1.

Participants needed: 5
Trial details
Phase: Phase 1Biological sex: AllType: InterventionalSponsor: Tippi MackenzieUpdated: Apr 13, 2026
Eligibility criteria

In the event that parents are identified as genetic carriers of Type I or Type I... [+3]

Esophageal or bowel atresia [+18]

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Status: Recruiting

Longitudinal Study of Neurodegenerative Disorders

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.

Participants needed: 1,500
Trial details
Biological sex: AllType: ObservationalSponsor: University of PittsburghUpdated: Feb 9, 2026Locations: 1
Eligibility criteria

Any patient with a genetic neurodegenerative disorder

none

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