Rare disease study maps GM1's devastating course in children

NCT ID NCT04041102

Completed Knowledge-focused Sponsor: University of Pennsylvania Source: ClinicalTrials.gov ↗

First seen Nov 01, 2025 · Last updated May 20, 2026 · Updated 27 times

Summary

This study followed 31 children with infantile or juvenile GM1 gangliosidosis, a rare and fatal genetic disorder, for up to 3 years. Researchers collected data from exams, blood tests, and imaging to better understand how the disease progresses. The goal was to identify reliable ways to measure disease changes, which could help design future treatment trials.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Locations

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Gazi University

Ankara, Turkey (Türkiye)
Hospital de Clínicas de Porto Alegre

Porto Alegre, Brazil
Montreal Children's Hospital Research Institute - McGill University

Montreal, Quebec, Canada
UCL Great Ormond Street Institute of Child Health

London, United Kingdom
UCSF Benioff Children's Hospital Oakland

Oakland, California, 94610, United States

Conditions

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GM1 GANGLIOSIDOSIS