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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.

949 public trials 197 recruiting studies
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Brain Diseases, Metabolic, Inborn trial results

Completed

First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria

NCT05781399

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • University of Florida College of Medicine Gainesville, Florida, United States
  • University of South Florida Tampa, Florida, United States
  • Rare Disease Research Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 15 locations
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Completed

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

NCT03771898

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Los Angeles Biomedical Research Institute at Harbor-UCLA Torrance, California, United States
  • Childrens Hospital Colorado Aurora, Colorado, United States
  • Rare Disease Research, LLC Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 30 locations
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Completed

A Phase 2b Study of Zagociguat in Patients With MELAS

NCT06402123

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
View all 20 locations
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Completed

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

NCT03485677

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • Investigational Site Number : 0320001 Buenos Aires, Argentina
  • Investigational Site Number : 1240002 Calgary, Alberta, Canada
  • Investigational Site Number : 1240003 Vancouver, British Columbia, Canada
  • Investigational Site Number : 1240001 Toronto, Ontario, Canada
View all 21 locations
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