NCT06669949
- Conditions
- Addison Disease, Adrenal Gland Diseases, Adrenal Insufficiency, +47 more
- Locations
-
- University of California San Francisco San Francisco, California, United States
Clinical trial condition
Sphingolipidoses
Explore clinical trials studying Sphingolipidoses. Study availability and eligibility vary by location and protocol.
16 public trials
5 recruiting studies
Sphingolipidoses trial results
Recruiting
Krabbe Disease Global Patient Registry
NCT02993796
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
- Locations
-
- State University of New York at Buffalo Buffalo, New York, United States
NCT04885179
- Conditions
- Addison Disease, Adrenal Gland Diseases, Adrenal Insufficiency, +28 more
- Locations
-
- University of California San Francisco San Francisco, California, United States
NCT05629559
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- Royal Melbourne Hospital Melbourne, Australia
- Royal Perth Hospital Perth, Australia
- Westmead Hospital Westmead, Australia
- Taipei Veterans General Hospital Taipei, Taiwan
Recruiting
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
NCT04880356
- Conditions
- 2-Hydroxyglutaricaciduria, Adrenal Gland Diseases, Adrenal Insufficiency, +58 more
- Locations
-
- Fondazione IRCCS Istituto Neurologico Carlo Besta Milan, Milano, Italy
Active, not recruiting
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
NCT02171104
- Conditions
- Abnormalities, Multiple, Acyl-CoA Oxidase Deficiency, Adrenoleukodystrophy With Cerebral Involvement, +85 more
- Locations
-
- Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States
Active, not recruiting
Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT
NCT05739643
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
- Locations
-
- Children's Hospital of Orange County (CHOC) Orange, California, United States
- University of Michigan Hospitals - Michigan Medicine Ann Arbor, Michigan, United States
- Duke University Medical Center Durham, North Carolina, United States
Active, not recruiting
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease
NCT04519749
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- University of California at San Diego La Jolla, California, United States
- Emory University Atlanta, Georgia, United States
- Children's Hospital of Pittsburgh of UPMC Pittsburgh, Pennsylvania, United States
- Lysosomal & Rare Disorders Research & Treatment Center, Inc Fairfax, Virginia, United States
NCT02416661
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
- Locations
-
- University Hospital Center Mother Teresa Tirana, Albania
- Aristotle University of Thessaloniki, Ippokration General Hospital Thessaloniki, Greece
- Centre for Human Genetics Bangalore, India
- Shaare Zedek Medical Center Jerusalem, Israel
Completed
Farber Disease Natural History Study
NCT03233841
- Conditions
- ASAH1 Mutation, Acid Ceramidase Deficiency, Brain Diseases, +27 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- Children's National Health System Washington D.C., District of Columbia, United States
- Hospital de Niños de la Santisima Trinidad Córdoba, CP, Argentina
- Montreal Children's Hospital Montreal, Quebec, Canada
Related conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities (16)
- Nutritional and Metabolic Diseases (16)
- Lysosomal Storage Diseases, Nervous System (15)
- Brain Diseases (14)
- Brain Diseases, Metabolic, Inborn (14)
- Lipid Metabolism, Inborn Errors (14)
- Brain Diseases, Metabolic (13)
- Central Nervous System Diseases (12)