New study maps how rare brain diseases worsen over time in kids

NCT ID NCT05109793

Completed Knowledge-focused Sponsor: Azafaros B.V. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study followed 31 children with GM1 or GM2 gangliosidosis (including Tay-Sachs and Sandhoff disease) to carefully measure how their neurological symptoms, like walking and speech, change over time. The goal was to create a clear picture of disease progression to help design future treatments. No new drug or therapy was tested; this was purely an observational study.

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Conditions

The condition(s) this trial relates to.

GM1 gangliosidosis GM2 gangliosidosis hereditary disease lysosomal storage disease nervous system disorder Sandhoff disease Tay-Sachs disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

GM1 GANGLIOSIDOSIS SANDHOFF DISEASE TAY-SACHS DISEASE

Contacts and locations

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Locations

Armand-Trousseau Children's Hospital - CHU Paris Est

Paris, France
Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, Italy
Great Ormond Street Hospital NHSFT

London, United Kingdom
Hopital d'Enfants CHU Timone

Marseille, France
Hospital Pequeno Principe

Curitiba, Brazil
Hospital de Clinicas de Porto Alegre

Porto Alegre, Brazil
Hôpital des Enfants - CHU Toulouse Purpan

Toulouse, France
LMU - Klinikum der Universitaet Muenchen - Neurologische Klinik und Poliklinik

Munich, Germany
Mayo Clinic Rochester

Rochester, Minnesota, 55905, United States
UCSF Benioff Children's Hospital

Oakland, California, 94609, United States
Universita' di Catania

Catania, Italy
University Hospital Friuli Centrale

Udine, Italy
Universtitäsklinikum Giessen und Marburg

Giessen, Germany

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