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Clinical trial

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

NCT04470713 is registered in ClinicalTrials.gov with Idorsia Pharmaceuticals Ltd. as lead sponsor.

NCT NCT04470713
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Registry record

As filed in ClinicalTrials.gov.

Title
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
Lead sponsor
Idorsia Pharmaceuticals Ltd.
Phase
Observational
Overall status
Completed
Start date
July 31, 2019
Completion date
October 30, 2021
Enrollment
226 (actual)
Sites
17

Conditions

As the registry lists them; a condition with its own page is linked.

ab variant gangliosidosis gm2, gaucher disease, type 2, gm1 gangliosidosis, gm2 gangliosidosis, sandhoff disease, tay-sachs disease