Gangliosidosis, GM1 (DBCOND0003891)

Identifiers

Synonyms

GM1-Gangliosidosis / GM1 Gangliosidosis / Other gangliosidosis / GM1 gangliosidosis (disorder) / Galactosialidosis / Combined deficiency of sialidase AND beta galactosidase (disorder)

Associated Data

Indicated Drugs and Targets

Not Available

Clinical Trials

Identifier	Title	Drug(s)	Purpose	Phase	Status
NCT04395339	GM1 Prophylaxis for WBRT Related Cognitive Dysfunction	Galactose Ganglioside GM1 Sodium chloride	treatment	3	terminated
NCT05368038	ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program	No drug interventions	Not Available	Not Available	enrolling_by_invitation
NCT04470713	Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2	No drug interventions	Not Available	Not Available	completed
NCT00383448	HSCT for High Risk Inherited Inborn Errors	Alemtuzumab Clofarabine Hydroxyurea Melphalan	treatment	2	completed
NCT06539169	FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases	No drug interventions	Not Available	Not Available	recruiting
NCT04041102	Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients	No drug interventions	Not Available	Not Available	recruiting
NCT00668187	A Natural History Study of the Gangliosidoses	No drug interventions	Not Available	Not Available	recruiting
NCT05109793	GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)	No drug interventions	Not Available	Not Available	active_not_recruiting
NCT04713475	Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis	PBGM01	treatment	1 / 2	active_not_recruiting
NCT04310163	Interviews and Video Capture in Patients With GM1 Gangliosidosis	No drug interventions	Not Available	Not Available	completed
NCT04624789	Registry Gangliosidoses	No drug interventions	Not Available	Not Available	recruiting
NCT00176904	Stem Cell Transplant for Inborn Errors of Metabolism	Busulfan Cyclophosphamide	treatment	2 / 3	completed
NCT04273269	A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis	No drug interventions	treatment	1 / 2	terminated