Health Trials

GM1 Gangliosidosis, Type I

1

Review clinical trials related to GM1 Gangliosidosis, Type I. 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]

Read more