GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
Completed
Conditions studied: GM1 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
In brief
The study aims to characterize prospectively longitudinal progression of neurological domains in GM1 and GM2 Gangliosidosis patients with high-quality standards (GCP compliant).
Key facts
- Study ID
- NCT05109793
- Run by
- Azafaros B.V.
- People needed
- 31
- Starts
- 2022-02-22
- Expected to finish
- 2025-09-16
- Last updated by the study team
- 2025-09-29
Who can join
Age: 2 and older, up to 20. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Genetically confirmed GM1 Gangliosidosis or genetically confirmed Tay-Sachs or Sandhoff disease
- Onset of neurological symptoms on or after the patient's first birthday
- Achieved 12-month developmental milestones at normal developmental time points as per Principal Investigator's judgement
- Abnormal gait and/or speech disturbance
You may not qualify if…
- Patients who have received (within 6 months before screening), are currently receiving or are planned to receive (within the following 6 months) gene therapy, stem cell transplantation, experimental drugs, or any drug, which, in the Investigator´s opinion, may (have) interfere(d) with disease progression
Where it is running
- UCSF Benioff Children's Hospital — Oakland, California, United States
- Mayo Clinic Rochester — Rochester, Minnesota, United States
- Hospital Pequeno Principe — Curitiba, Brazil
- Hospital de Clinicas de Porto Alegre — Porto Alegre, Brazil
- Hopital d'Enfants CHU Timone — Marseille, France
- Armand-Trousseau Children's Hospital - CHU Paris Est — Paris, France
- Hôpital des Enfants - CHU Toulouse Purpan — Toulouse, France
- Universtitäsklinikum Giessen und Marburg — Giessen, Germany
- LMU - Klinikum der Universitaet Muenchen - Neurologische Klinik und Poliklinik — Munich, Germany
- Universita' di Catania — Catania, Italy
- Fondazione IRCCS Istituto Neurologico Carlo Besta — Milan, Italy
- University Hospital Friuli Centrale — Udine, Italy
- Great Ormond Street Hospital NHSFT — London, United Kingdom
Full record on ClinicalTrials.gov
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