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Results for “Late Onset Tay-Sachs Disease”

9 trials

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Showing 9 of 9 results

Large-scale testing (Phase 3)Active Not RecruitingNCT07082543
What this trial is testing

Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

Who this might be right for
Gangliosidoses, GM2Gangliosidosis, GM1
Azafaros B.V. 75
Large-scale testing (Phase 3)Ended earlyNCT04221451
What this trial is testing

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

Who this might be right for
Tay-Sachs DiseaseSandhoff Disease
Genzyme, a Sanofi Company 75
Large-scale testing (Phase 3)Looking for participantsNCT07054515
What this trial is testing

Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

Who this might be right for
Niemann-Pick Type C DiseaseGM1 GangliosidosisGM2 Gangliosidosis
Azafaros B.V. 147
Large-scale testing (Phase 3)Looking for participantsNCT07082725
What this trial is testing

Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)

Who this might be right for
Niemann-Pick Type C Disease
Azafaros B.V. 72
Not applicableStudy completedNCT02851862
What this trial is testing

A Natural History of Late Onset Tay-Sachs Disease

Who this might be right for
GM2 Gangliosidosis
Massachusetts General Hospital 10
Testing effectiveness (Phase 2)Study completedNCT01102686
What this trial is testing

Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)

Who this might be right for
Gangliosidoses, GM2Sandhoff DiseaseTay-Sachs Disease
The Hospital for Sick Children 20
Not applicableLooking for participantsNCT00668187
What this trial is testing

A Natural History Study of the Gangliosidoses

Who this might be right for
Tay-Sachs DiseaseSandhoff DiseaseLate Onset Tay-Sachs Disease+2 more
University of Minnesota 52
Not applicableStudy completedNCT01869270
What this trial is testing

Gene Therapy for Tay-Sachs Disease

Who this might be right for
Tay Sachs DiseaseSandhoff DiseaseLate Onset Tay Sachs Disease
University of Minnesota 4
Not applicableStudy completedNCT05109793
What this trial is testing

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

Who this might be right for
GM1 GangliosidosisSandhoff DiseaseTay-Sachs Disease
Azafaros B.V. 31