Rare disease study tracks GM1 progression to pave way for future treatments

NCT ID NCT04041102

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

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

Summary

This study followed 31 children with infantile or juvenile GM1 gangliosidosis, a rare and severe genetic disorder, for up to 3 years. Researchers collected data from exams, blood tests, and developmental assessments to better understand how the disease progresses. The goal was to identify useful markers for future clinical trials, not to test a treatment.

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Conditions

The condition(s) this trial relates to.

GM1 gangliosidosis lysosomal storage disease

As listed by the trial registrant

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

GM1 GANGLIOSIDOSIS

Contacts and locations

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

More trials for these conditions

Other studies related to the condition(s) this trial covers.