NCT00004802
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Genetic Diseases, Inborn, +19 more
- Locations
-
Not specified
Clinical trial condition
Metabolism, Inborn Errors
Explore clinical trials studying Metabolism, Inborn Errors. Study availability and eligibility vary by location and protocol.
2,496 public trials
457 recruiting studies
Metabolism, Inborn Errors trial results
Completed
A Study of Valganciclovir in the Treatment of Cytomegalovirus (CMV) Retinitis in Patients With AIDS
NCT00002222
- Conditions
- AIDS-Related Opportunistic Infections, Amino Acid Metabolism, Inborn Errors, Blood-Borne Infections, +30 more
- Locations
-
- Cornell AIDS Clinical Trials Unit New York, United States
- Conditions
- Acquired Immunodeficiency Syndrome, Amino Acid Metabolism, Inborn Errors, Blood-Borne Infections, +30 more
- Locations
-
- Georgetown Univ Med Ctr Washington D.C., District of Columbia, United States
NCT00000102
- Conditions
- Adrenal Gland Diseases, Adrenal Hyperplasia, Congenital, Adrenogenital Syndrome, +16 more
- Locations
-
- Medical University of South Carolina Charleston, South Carolina, United States
Active, Not Recruiting
The REPLACE Registry for Cholbam® (Cholic Acid)
NCT03115086
- Conditions
- Abnormalities, Multiple, Bile Acid Synthesis Disorders, Brain Diseases, +20 more
- Locations
-
- University of Alabama Birmingham Birmingham, Alabama, United States
- Stanford School of Medicine Palo Alto, California, United States
- UC San Francisco San Francisco, California, United States
- Kidz Pediatric Multispecialty Group Hollywood, Florida, United States
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
Treatment of Hemochromatosis
NCT00007150
- Conditions
- Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Hemochromatosis, +6 more
- Locations
-
- National Institutes of Health Clinical Center Bethesda, Maryland, United States