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GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO) — PRONTO  

Sandhoff Disease Clinical Trial

Official title:
Prospective Longitudinal Study of Neurological Disease Trajectory in Children Living With Late-Infantile or Juvenile Onset of GM1 or GM2 Gangliosidosis

Clinical Trial Summary

The study aims to characterize prospectively longitudinal progression of neurological domains in GM1 and GM2 Gangliosidosis patients with high-quality standards (GCP compliant).

Clinical Trial Description

The study is a prospective longitudinal, multicentric decentralized trial which will be performed in children diagnosed with late infantile or juvenile onset of neurological disease of either GM1 or GM2 Gangliosidoses (Tay-Sachs or Sandhoff disease). The study anticipates to include a total of approximately 35 patients. A large set of neurological functions will be evaluated by rating scales used by physicians and questionnaires answered by parents. Digital tools will be used to support the study procedures with virtual visits and also a passive monitoring approach with a medical device. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05109793
Study type Observational
Source Azafaros A.G.
Contact
Status Active, not recruiting
Phase
Start date February 22, 2022
Completion date May 30, 2026
View Details
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