10 completed, 6 terminated, 3 recruiting, 2 active not recruiting. 8 Phase 2, 6 Phase 1, 5 Phase 3, 1 Not applicable, 1 Phase 4.
Follow Sandhoff Disease: new trials by email Literature Review of Sandhoff Disease trials Research Report on Sandhoff Disease
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol) PLX-200 - Lysosomal Storage Disorders, Sandhoff Disease | Phase 2 | Not yet recruiting | Polaryx Therapeutics, Inc. | 24 | 2026-12-01 | 2026-07-31 |
| Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease AXO-AAV-GM2 - GM2 Gangliosidosis, Tay Sachs Disease | Active not recruiting | Terence Flotte | 7 | 2024-09-17 | 2025-10-16 | |
| GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO) GM1 Gangliosidosis, Sandhoff Disease | Completed | Azafaros B.V. | 31 | 2022-02-22 | 2025-09-29 | |
| First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis TSHA-101 - Infantile GM2 Gangliosidosis (Disorder) | Phase 1 / Phase 2 | Active not recruiting | Dr. Anupam Sehgal | 3 | 2021-03-12 | 2023-05-09 |
| A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease AXO-AAV-GM2 Starting Dose, AXO-AAV-GM2 Low Dose, AXO-AAV-GM2 Middle Dose - Tay-Sachs Disease, Sandhoff Disease | Phase 1 | Terminated | Terence Flotte | 9 | 2021-01-15 | 2025-09-02 |
| A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 venglustat GZ402671, placebo - Tay-Sachs Disease, Sandhoff Disease | Phase 3 | Terminated | Genzyme, a Sanofi Company | 75 | 2020-06-29 | 2026-01-28 |
| Registry Gangliosidoses Gangliosidoses, GM1 Gangliosidosis | Unknown | SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) | 40 | 2020-06-08 | 2020-11-12 | |
| Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2 GM1 Gangliosidosis, GM2 Gangliosidosis | Completed | Idorsia Pharmaceuticals Ltd. | 226 | 2019-07-31 | 2021-11-08 | |
| N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) IB1001 - GM2 Gangliosidosis, Tay-Sachs Disease | Phase 2 | Completed | IntraBio Inc | 30 | 2019-06-07 | 2024-03-12 |
| Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD) Miglustat - GM2 Gangliosidosis, Supportive Care | Phase 3 | Terminated | Tehran University of Medical Sciences | 30 | 2019-01-14 | 2025-09-17 |
| Synergistic Enteral Regimen for Treatment of the Gangliosidoses miglustat, Ketogenic Diet - GM1 Gangliosidoses, GM2 Gangliosidoses | Phase 4 | Terminated | University of Minnesota | 16 | 2015-12-22 | 2021-04-14 |
| UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells DUOC-01 - Adrenoleukodystrophy, Batten Disease | Phase 1 | Recruiting | Joanne Kurtzberg, MD | 40 | 2014-09-01 | 2026-09-03 |
| Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders Campath-1H, Clofarabine, Melphalan - Lysosomal Storage Disease, Peroxisomal Disorder | Not applicable | Completed | Masonic Cancer Center, University of Minnesota | 3 | 2012-07-11 | 2017-12-05 |
| Longitudinal Study of Neurodegenerative Disorders Palliative Care, Hematopoetic Stem Cell Transplantation - MLD, Krabbe Disease | Recruiting | University of Pittsburgh | 1500 | 2012-01-11 | 2026-02-09 | |
| Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders hematopoietic stem cell infusion - Hurler Syndrome (MPS I), Hurler-Scheie Syndrome | Phase 1 / Phase 2 | Terminated | Talaris Therapeutics Inc. | 3 | 2011-04-01 | 2023-04-12 |
| A Natural History Study of the Gangliosidoses Tay-Sachs Disease, Sandhoff Disease | Recruiting | University of Minnesota | 52 | 2010-12-01 | 2026-03-04 | |
| Gene Therapy for Tay-Sachs Disease Tay Sachs Disease, Sandhoff Disease | Completed | University of Minnesota | 4 | 2010-12-01 | 2014-12-04 | |
| Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease) Pyrimethamine, Leucovorin - Gangliosidoses, GM2, Sandhoff Disease | Phase 1 / Phase 2 | Completed | The Hospital for Sick Children | 20 | 2009-08-01 | 2012-02-23 |
| A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis Pyrimethamine - G(M2) Ganglioside, Tay-Sachs Disease Ganglioside | Phase 1 | Withdrawn | Exsar Corporation | 2008-05-01 | 2013-02-25 | |
| ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases ALD-101 - Inherited Metabolic Diseases, Lysosomal Storage Disorders | Phase 3 | Terminated | Aldagen | 40 | 2008-03-01 | 2014-07-08 |
Showing 20 of 24. Sign in to see all of them.
Tay-Sachs Disease (22) · Gangliosidoses, GM2 (15) · Gangliosidosis, GM1 (9) · Leukodystrophy, Globoid Cell (8) · Lysosomal Storage Diseases (7) · Adrenoleukodystrophy (6) · Leukodystrophy, Metachromatic (6) · Mucopolysaccharidosis III (5) · Gangliosidoses (4) · Mucolipidoses (4) · alpha-Mannosidosis (4) · Gaucher Disease (3) · Mucopolysaccharidosis II (3) · Neuronal Ceroid-Lipofuscinoses (3)
Brain Diseases (24) · Brain Diseases, Metabolic (24) · Central Nervous System Diseases (24) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (24) · Lipid Metabolism Disorders (24) · Lipid Metabolism, Inborn Errors (24) · Lipidoses (24) · Lysosomal Storage Diseases, Nervous System (24) · Metabolic Diseases (24) · Nutritional and Metabolic Diseases (24) · Sphingolipidoses (24) · Brain Diseases, Metabolic, Inborn (23) · Genetic Diseases, Inborn (23) · Nervous System Diseases (23) · Metabolism, Inborn Errors (22)
miglustat (4) · Alemtuzumab (2) · Clofarabine (2) · Hematopoietic Stem Cell Transplantation (2) · Melphalan (2) · Pyrimethamine (2) · Stem Cell Transplantation (2) · Whole-Body Irradiation (2) · Antilymphocyte Serum (1) · Bone Marrow Transplantation (1) · Busulfan (1) · Cyclophosphamide (1) · Cyclosporine (1) · Diet, Ketogenic (1) · Gemfibrozil (1)
Masonic Cancer Center, University of Minnesota (3) · University of Minnesota (3) · Terence Flotte (2) · The Hospital for Sick Children (2) · Aldagen (1) · Azafaros B.V. (1) · Children's National Research Institute (1) · Dr. Anupam Sehgal (1) · Exsar Corporation (1) · Genzyme, a Sanofi Company (1) · Idorsia Pharmaceuticals Ltd. (1) · IntraBio Inc (1) · Joanne Kurtzberg, MD (1) · Polaryx Therapeutics, Inc. (1) · SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) (1)
United States (18) · Germany (5) · Canada (4) · United Kingdom (4) · Brazil (3) · France (3) · Italy (3) · Spain (3) · Portugal (2) · Argentina (1) · Belgium (1) · Czechia (1) · Iran (1) · Japan (1) · Russia (1)
Jeanine R. Jarnes, PharmD (2) · Joanne Kurtzberg, MD (2) · Paul Orchard, MD (2) · Terence Flotte, MD (2) · Anupam Sehgal, MBBS (1) · Bijan Almassian, Ph.D. (1) · Chester B. Whitley, PhD, MD (1) · Cynthia J TIfft, MD, PhD (1) · James Hinson, MD (1) · Jeffrey Krischer, PhD (1) · Joe T Clarke, MD (1) · Joe TR Clarke, MD (1) · Suzanne T Ildstad, MD (1)
PMID 21586746, PMID 28476546, PMID 40817303, PMID 19595619, PMID 25557439, PMID 29352662, PMID 33482890, PMID 34349180, PMID 34539759, PMID 36194207, PMID 36456200, PMID 37624739, PMID 38205442, PMID 42467089
U54NS065768 (3), 2019 (2)