GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
NCT05109793 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 31
Last updated 2025-09-29
Summary
The study aims to characterize prospectively longitudinal progression of neurological domains in GM1 and GM2 Gangliosidosis patients with high-quality standards (GCP compliant).
Conditions
- GM1 Gangliosidosis
- Sandhoff Disease
- Tay-Sachs Disease
Sponsors & Collaborators
-
Azafaros A.G.
lead INDUSTRY
Eligibility
- Min Age
- 2 Years
- Max Age
- 20 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2022-02-22
- Primary Completion
- 2025-09-16
- Completion
- 2025-09-16
Countries
- United States
- Brazil
- France
- Germany
- Italy
- United Kingdom
Study Locations
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