Trials / Conditions / GM1 Gangliosidosis

GM1 Gangliosidosis

13 registered clinical trials studyying GM1 Gangliosidosis — 4 currently recruiting.

Status	Trial	Sponsor	Phase
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
Recruiting	FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases NCT06539169	xCures	—
Completed	GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO) NCT05109793	Azafaros A.G.	—
Terminated	A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis NCT04273269	LYSOGENE	Phase 1 / Phase 2
Enrolling By Invitation	ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program NCT05368038	Albert Einstein College of Medicine	—
Active Not Recruiting	Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis NCT04713475	Gemma Biotherapeutics	Phase 1 / Phase 2
Completed	Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients NCT04041102	University of Pennsylvania	—
Unknown	Registry Gangliosidoses NCT04624789	SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt)	—
Completed	Interviews and Video Capture in Patients With GM1 Gangliosidosis NCT04310163	LYSOGENE	—
Completed	Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses NCT04470713	Idorsia Pharmaceuticals Ltd.	—
Recruiting	A Natural History Study of the Gangliosidoses NCT00668187	University of Minnesota	—
Completed	HSCT for High Risk Inherited Inborn Errors NCT00383448	Masonic Cancer Center, University of Minnesota	Phase 2
Completed	Stem Cell Transplant for Inborn Errors of Metabolism NCT00176904	Masonic Cancer Center, University of Minnesota	Phase 2 / Phase 3