Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

CompletedOBSERVATIONAL
Enrollment

226

Participants

Timeline

Start Date

July 31, 2019

Primary Completion Date

October 30, 2021

Study Completion Date

October 30, 2021

Conditions
GM1 GangliosidosisGM2 GangliosidosisGaucher Disease, Type 2Tay-Sachs DiseaseAB Variant Gangliosidosis GM2Sandhoff Disease
Trial Locations (17)

1020

UCL Cliniques Universitaires Saint-Luc, Brussels

20133

Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta, Milan

22030

Lysosomal and Rare Disorders Research and Treatment Center, Fairfax

50006

Quirónsalud, Zaragoza

50139

Azienda Ospedaliero Universitaria Meyer, Florence

55905

Mayo Clinic - Rochester, Rochester

60611

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

65239

SphinCS GmbH, Höchheim

75012

AP-HP - Hôpitaux Universitaires Est Parisien, Paris

90035-903

Hospital de Clínicas de Porto Alegre - HCPA, Porto Alegre

1649-035

Centro Hospitalar Universitario Lisboa Norte, EPE, Lisbon

4200-319

Centro Universitario Hospitalar de São João, EPE, Porto

08950

Hospital Sant Joan de Deu, Barcelona

CH-3010

Universitätsspital Bern Inselspital, Bern

CH-8032

Universitäts-Kinderspital Zürich, Zurich

B4 6NH

University Hospitals Birmingham NHS Foundation Trust, Birmingham

WC1N 3JH

Great Ormond Street Hospital for Children NHS Found. Trust, London

Sponsors
All Listed Sponsors
lead

Idorsia Pharmaceuticals Ltd.

INDUSTRY