NCT03161171
- Conditions
- Behavior Disorders, Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +24 more
- Locations
-
- University of Heidelberg Heidelberg, Germany
Clinical trial condition
Mucopolysaccharidosis III
Explore clinical trials studying Mucopolysaccharidosis III. Study availability and eligibility vary by location and protocol.
41 public trials
8 recruiting studies
Mucopolysaccharidosis III trial results
NCT01626092
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +50 more
- Locations
-
- Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States
Completed
Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
NCT02053064
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +13 more
- Locations
-
- Hôpitaux Universitaires Paris Sud (Bicêtre) Le Kremlin-Bicêtre, France
NCT02293382
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- Pittsburgh, Pennsylvania, United States
- Porte Alegre, Brazil
- Amsterdam, Netherlands
- Barcelona, Spain
NCT02090179
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- Birmingham, United Kingdom
NCT02037880
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +11 more
- Locations
-
- Nationwide Children's Hospital Columbus, Ohio, United States
NCT01474343
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +13 more
- Locations
-
- Hôpital Bicêtre - Assistance Publique des Hôpitaux de Paris Le Kremlin-Bicêtre, France
- Hôpital Necker, Assistance Publique des Hôpitaux de Paris Paris, France
Active, Not Recruiting
Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
NCT06181136
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- UCSF Benioff Children's Hospital Oakland Oakland, California, United States
- University of Iowa Stead Family Children's Hospital Iowa City, Iowa, United States
- University of North Carolina at Chapel Hill Chapel Hill, North Carolina, United States
- Baylor College of Medicine and Texas Children's Hospital Houston, Texas, United States
Active, Not Recruiting
Early Check: Expanded Screening in Newborns
NCT03655223
- Conditions
- 17 Alpha-Hydroxylase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, +456 more
- Locations
-
- RTI International Research Triangle Park, North Carolina, United States
Active, Not Recruiting
Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
NCT06095388
- Conditions
- Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
- Locations
-
- Universitätsklinikum Hamburg-Eppendorf Hamburg, Germany
Related conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities (41)
- Nutritional and Metabolic Diseases (41)
- Skin and Connective Tissue Diseases (41)
- Carbohydrate Metabolism, Inborn Errors (40)
- Connective Tissue Diseases (40)
- Mucinoses (40)
- Metabolism, Inborn Errors (38)
- Genetic Diseases, Inborn (37)