Sandhoff Disease

16 registered clinical trials studyying Sandhoff Disease — 3 currently recruiting.

Status	Trial	Sponsor	Phase
Active Not Recruiting	Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease NCT06614569	Terence Flotte	—
Completed	GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO) NCT05109793	Azafaros A.G.	—
Terminated	A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease NCT04669535	Terence Flotte	Phase 1
Terminated	A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, NCT04221451	Genzyme, a Sanofi Company	Phase 3
Unknown	Registry Gangliosidoses NCT04624789	SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt)	—
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	Synergistic Enteral Regimen for Treatment of the Gangliosidoses NCT02030015	University of Minnesota	Phase 4
Recruiting	UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte- NCT02254863	Joanne Kurtzberg, MD	Phase 1
Recruiting	Longitudinal Study of Neurodegenerative Disorders NCT03333200	University of Pittsburgh	—
Terminated	Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders NCT01372228	Talaris Therapeutics Inc.	Phase 1 / Phase 2
Recruiting	A Natural History Study of the Gangliosidoses NCT00668187	University of Minnesota	—
Completed	Gene Therapy for Tay-Sachs Disease NCT01869270	University of Minnesota	—
Completed	Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease) NCT01102686	The Hospital for Sick Children	Phase 1 / Phase 2
Completed	Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidos NCT00672022	Children's National Research Institute	Phase 3
Completed	Stem Cell Transplant for Inborn Errors of Metabolism NCT00176904	Masonic Cancer Center, University of Minnesota	Phase 2 / Phase 3