Trials / Conditions / GM2 Gangliosidosis

GM2 Gangliosidosis

11 registered clinical trials studyying GM2 Gangliosidosis — 4 currently recruiting.

Status	Trial	Sponsor	Phase
Recruiting	A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or NCT07399704	Azafaros A.G.	Phase 2
Recruiting	A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile For NCT07054515	Azafaros A.G.	Phase 3
Active Not Recruiting	Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease NCT06614569	Terence Flotte	—
Active Not Recruiting	Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Pa NCT05758922	Azafaros A.G.	Phase 2
Completed	Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses NCT04470713	Idorsia Pharmaceuticals Ltd.	—
Completed	N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) NCT03759665	IntraBio Inc	Phase 2
Terminated	Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) NCT03822013	Tehran University of Medical Sciences	Phase 3
Completed	LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies NCT02699190	Children's Hospital of Philadelphia	—
Recruiting	The Myelin Disorders Biorepository Project NCT03047369	Children's Hospital of Philadelphia	—
Completed	A Natural History of Late Onset Tay-Sachs Disease NCT02851862	Massachusetts General Hospital	—
Recruiting	A Natural History Study of the Gangliosidoses NCT00668187	University of Minnesota	—