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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
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Brain Diseases, Metabolic, Inborn trial results

Recruiting

A Long-Term Study of JNT-517 in Participants With Phenylketonuria

NCT06628128

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • University of Florida (UF) Health Shands Hospital Gainesville, Florida, United States
  • University of South Florida Tampa, Florida, United States
  • Oregon Health and Science University Portland, Oregon, United States
  • University of Pittsburgh Medical Center (UPMC) - Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
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Recruiting

A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders

NCT06904027

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • Peking University First Hospital Beijing, Beijing Municipality, China
  • Guangzhou Women and Children's Medical Center Guangzhou, Guangdong, China
  • Tongji Hosipital Wuhan, Hubei, China
  • Xinhua Hospital, Shanghai Jiao Tong University School of Medicine Shanghai, Shanghai Municipality, China
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Recruiting

Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease

NCT05659901

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Ionis Investigative Site Atlanta, Georgia, United States
  • Ionis Investigative Site Philadelphia, Pennsylvania, United States
  • Ionis Investigative Site Clermont-Ferrand, France
  • Ionis Investigative Site Le Kremlin-Bicêtre, France
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Recruiting

A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

NCT04998396

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
Locations
  • UCSF Benioff Children's Hospital Oakland Oakland, California, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • Massachusetts General Hospital (MGH); Center for Rare Neurological Diseases (CRND) Boston, Massachusetts, United States
  • Weill Cornell Medicine; Division of Pediatric Neurology New York, United States
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Recruiting

Pompe Pregnancy Sub-Registry

NCT00567073

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Barrow Neurol Group- Site Number : 840087 Phoenix, Arizona, United States
  • University of Florida Pediatrics Genetics- Site Number : 157138 Jacksonville, Florida, United States
  • Emory University School Of Medicine- Site Number : 840060 Atlanta, Georgia, United States
  • Indianapolis University School of Medicine- Site Number : 840027 Indianapolis, Indiana, United States
View all 35 locations
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Recruiting

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

NCT00358943

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +17 more
Locations
  • Phoenix Children's Hospital- Site Number : 840003 Phoenix, Arizona, United States
  • University of Arizona- Site Number : 840015 Tucson, Arizona, United States
  • Arkansas Children's Hospital- Site Number : 840109 Little Rock, Arkansas, United States
  • University of Arkansas for Medical Sciences- Site Number : 840113 Little Rock, Arkansas, United States
View all 318 locations
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