Sandhoff Disease clinical trials
The registry lists 20 studies for Sandhoff Disease across more than 10 countries. 5 are recruiting or about to start. Per ClinicalTrials.gov, last updated 2026-09-03.
Status
- Completed
- 8
- Terminated
- 5
- Recruiting
- 4
- Active, not recruiting
- 1
- Not yet recruiting
- 1
- Withdrawn
- 1
Phase
- Phase 2
- 6
- Phase 1
- 5
- Phase 3
- 4
- Phase 4
- 1
Where
- United States
- 16
- Germany
- 5
- United Kingdom
- 4
- Spain
- 3
- Italy
- 3
- France
- 3
Who runs them
University of Minnesota (3) · Masonic Cancer Center, University of Minnesota (2) · Terence Flotte (2) · IntraBio Inc (1) · SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) (1) · Idorsia Pharmaceuticals Ltd. (1) · Children's National Research Institute (1) · Exsar Corporation (1)
Studies
Recruiting first, then most recently updated. Showing 20 of 20 — search for more.
- UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like CellsRecruitingNCT02254863Phase 11 country
- Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)Not yet recruitingNCT07740512Phase 2
- A Natural History Study of the GangliosidosesRecruitingNCT006681871 country
- Longitudinal Study of Neurodegenerative DisordersRecruitingNCT033332001 country
- Registry GangliosidosesRecruitingNCT046247891 country
- A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2TerminatedNCT04221451Phase 313 countries
- Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff DiseaseActive, not recruitingNCT066145691 country
- GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)CompletedNCT051097936 countries
- Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)TerminatedNCT03822013Phase 31 country
- A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff DiseaseTerminatedNCT04669535Phase 11 country
- N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)CompletedNCT03759665Phase 24 countries
- Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersTerminatedNCT01372228Phase 1/21 country
- Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2CompletedNCT0447071310 countries
- Synergistic Enteral Regimen for Treatment of the GangliosidosesTerminatedNCT02030015Phase 41 country
- HSCT for High Risk Inherited Inborn ErrorsCompletedNCT00383448Phase 21 country
- Stem Cell Transplant for Inborn Errors of MetabolismCompletedNCT00176904Phase 2/31 country
- Gene Therapy for Tay-Sachs DiseaseCompletedNCT018692701 country
- A Phase I Study of Pyrimethamine in Patients With GM2 GangliosidosisWithdrawnNCT00679744Phase 12 countries
- Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)CompletedNCT01102686Phase 1/21 country
- Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral DosesCompletedNCT00672022Phase 31 country