GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

CompletedOBSERVATIONAL
Enrollment

31

Participants

Timeline

Start Date

February 22, 2022

Primary Completion Date

September 16, 2025

Study Completion Date

September 16, 2025

Conditions
GM1 GangliosidosisSandhoff DiseaseTay-Sachs Disease
Trial Locations (13)

55905

Mayo Clinic Rochester, Rochester

94609

UCSF Benioff Children's Hospital, Oakland

Unknown

Hospital Pequeno Principe, Curitiba

Hospital de Clinicas de Porto Alegre, Porto Alegre

Hopital d'Enfants CHU Timone, Marseille

Armand-Trousseau Children's Hospital - CHU Paris Est, Paris

Hôpital des Enfants - CHU Toulouse Purpan, Toulouse

Universtitäsklinikum Giessen und Marburg, Giessen

LMU - Klinikum der Universitaet Muenchen - Neurologische Klinik und Poliklinik, Munich

Universita' di Catania, Catania

Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan

University Hospital Friuli Centrale, Udine

Great Ormond Street Hospital NHSFT, London

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Azafaros A.G.

INDUSTRY

NCT05109793 - GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO) | Biotech Hunter | Biotech Hunter