NCT02254863
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
- Locations
-
- Duke University Medical Center Durham, North Carolina, United States
Clinical trial condition
Sandhoff Disease
Explore clinical trials studying Sandhoff Disease. Study availability and eligibility vary by location and protocol.
12 public trials
3 recruiting studies
Sandhoff Disease trial results
NCT00668187
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +22 more
- Locations
-
- University of Minnesota - Pediatric Genetics and Metabolism Minneapolis, Minnesota, United States
NCT03333200
- Conditions
- ALD, Alpha-Mannosidosis, Batten Disease, +78 more
- Locations
-
- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
Not yet recruiting
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)
NCT07740512
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +24 more
- Locations
-
Not specified
Active, not recruiting
Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease
NCT06614569
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- UMass Chan Medical School Worcester, Massachusetts, United States
NCT05109793
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland, California, United States
- Mayo Clinic Rochester Rochester, Minnesota, United States
- Hospital Pequeno Principe Curitiba, Brazil
- Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil
NCT03759665
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
- University of California - Los Angeles Los Angeles, California, United States
- Mayo Clinic Rochester, Minnesota, United States
- NYU Langone School of Medicine New York, United States
- University of Giessen Giessen, Germany
NCT04470713
- Conditions
- AB Variant Gangliosidosis GM2, Brain Diseases, Brain Diseases, Metabolic, +24 more
- Locations
-
- Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
- Mayo Clinic - Rochester Rochester, Minnesota, United States
- Lysosomal and Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
- UCL Cliniques Universitaires Saint-Luc Brussels, Belgium
NCT00176904
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
- Locations
-
- Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States
Completed
Gene Therapy for Tay-Sachs Disease
NCT01869270
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- Data Management and Coordinating Center (DMCC), Univ. of South Florida Tampa, Florida, United States
- University of Minnesota Minneapolis, Minnesota, United States