NCT00004333
- Conditions
- Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +14 more
- Locations
-
Not specified
Clinical trial condition
Brain Diseases, Metabolic, Inborn
Explore clinical trials studying Brain Diseases, Metabolic, Inborn. Study availability and eligibility vary by location and protocol.
948 public trials
197 recruiting studies
Brain Diseases, Metabolic, Inborn trial results
Active, Not Recruiting
A First-in-Human, Single- and Multiple-Ascending Dose Study of YH35995 in Healthy Adult Male Participants
NCT06517914
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
- Locations
-
- CHA Bundang Medical Center Seongnam, Bundang-gu, South Korea
Active, Not Recruiting
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
NCT07082543
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
- Locations
-
- UCSF Children's Hospital and Research Center at Oakland Oakland, California, United States
- University of Minnesota Medical School Minneapolis, Minnesota, United States
- Mayo Clinic Children's Center - PIN Rochester, Minnesota, United States
- Children's Medical Center Dallas Dallas, Texas, United States
Active, Not Recruiting
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
NCT05270837
- Conditions
- Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
- Locations
-
- Phoenix Children's Hospital Phoenix, Arizona, United States
- Arkansas Children's Hospital Little Rock, Arkansas, United States
- Children's Hospital of Colorado Aurora, Colorado, United States
- University of South Florida Tampa, Florida, United States
Active, Not Recruiting
Safety, Tolerability and Efficacy of NPI-001 in Patients With Hereditary Cystatin C Amyloid Angiopathy (HCCAA)
NCT07716865
- Conditions
- Amyloidosis, Amyloidosis, Familial, Brain Diseases, +29 more
- Locations
-
- Landspitali University Hospital Reykjavik, Iceland
Active, Not Recruiting
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
NCT06150820
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
- Locations
-
- Emory Clinic Atlanta, Georgia, United States
- University of Kansas Medical Center Kansas City, Kansas, United States
- University of Michigan Ann Arbor, Michigan, United States
- Children's Hospitals and Clinics of Minnesota Minneapolis, Minnesota, United States
Active, Not Recruiting
Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)
NCT04127578
- Conditions
- Basal Ganglia Diseases, Brain Diseases, Brain Diseases, Metabolic, +20 more
- Locations
-
- Mayo Clinic, 13400 E. Shea Boulevard Scottsdale, Arizona, United States
- Esperanza Clinical, 25220 Hancock Avenue Murrieta, California, United States
- Rocky Mountain Clinical Research - CenExel - PPDS, 701 East Hampden Avenue Suite 510 Englewood, Colorado, United States
- K2 Medical Research, 101 Southhall Lane, Suite 150 Maitland, Florida, United States
Active, Not Recruiting
Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3
NCT05222906
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
- Locations
-
- Yale University School of Medicine - Investigational Site Number: 8400003 New Haven, Connecticut, United States
- University of Iowa - Investigational Site Number: 8400002 Iowa City, Iowa, United States
- Texas Oncology - Medical City Dallas Site Number : 8400008 Dallas, Texas, United States
- Lysosomal & Rare Disorders Research & Treatment Center, Inc - Investigational Site Number: 8400001 Fairfax, Virginia, United States
Active, Not Recruiting
Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension
NCT02843035
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
- Locations
-
- Yale University School of Medicine Site Number : 840002 New Haven, Connecticut, United States
- Baylor Institute of Metabolic Diseases Site Number : 840001 Dallas, Texas, United States
- Lysosomal and Rare Disorders Research and Treatment Center, Inc Site Number : 840003 Fairfax, Virginia, United States
- Investigational Site Number : 276001 Mainz, Germany
Active, Not Recruiting
An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)
NCT06961344
- Conditions
- Acid-Base Imbalance, Acidosis, Acidosis, Lactic, +23 more
- Locations
-
- UC San Diego - Altman Clinical and Translational Research Institute La Jolla, California, United States
- Children's Hospital of Colorado Aurora, Colorado, United States
- Rare Disease Research Atlanta, Georgia, United States
- Massachusetts General Hospital Boston, Massachusetts, United States