Prospective Longitudinal Study of Neurological Disease Trajectory in Children Living With Late-Infantile or Juvenile Onset of GM1 or GM2 Gangliosidosis
Status: Completed
Location: See all (13) locations...
Study Type: Observational
SUMMARY
The study aims to characterize prospectively longitudinal progression of neurological domains in GM1 and GM2 Gangliosidosis patients with high-quality standards (GCP compliant).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Maximum Age: 20
Healthy Volunteers: f
View:
• 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
Locations
United States
California
UCSF Benioff Children's Hospital
Oakland
Minnesota
Mayo Clinic Rochester
Rochester
Other Locations
Brazil
Hospital Pequeno Principe
Curitiba
Hospital de Clinicas de Porto Alegre
Porto Alegre
France
Hopital d'Enfants CHU Timone
Marseille
Armand-Trousseau Children's Hospital - CHU Paris Est
Paris
Hôpital des Enfants - CHU Toulouse Purpan
Toulouse
Germany
Universtitäsklinikum Giessen und Marburg
Giessen
LMU - Klinikum der Universitaet Muenchen - Neurologische Klinik und Poliklinik
Munich
Italy
Universita' di Catania
Catania
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan
University Hospital Friuli Centrale
Udine
United Kingdom
Great Ormond Street Hospital NHSFT
London
Time Frame
Start Date: 2022-02-22
Completion Date: 2025-09-16
Participants
Target number of participants: 31
Treatments
Cohort
Late infantile or juvenile onset for GM1 or GM2 Gangliosidosis. The study anticipates to include a total of approximately 35 patients.
Related Therapeutic Areas
Sponsors
Leads: Azafaros A.G.