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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.

955 public trials 199 recruiting studies
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Brain Diseases, Metabolic, Inborn trial results

Recruiting

AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)

NCT06332807

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +9 more
Locations
  • Children's Hospital of Orange County Hospital Orange, California, United States
  • University of Minnesota Minneapolis, Minnesota, United States
  • Atlantic Health System Morristown, New Jersey, United States
  • University of Pittsburgh Medical Center Pittsburgh, Pennsylvania, United States
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Recruiting

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)

NCT07082725

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 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
View all 34 locations
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Recruiting

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease

NCT07354724

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +13 more
Locations
  • University of California-Irvine Irvine, California, United States
  • Duke University School of Medicine - Early Phase Research Unit Durham, North Carolina, United States
  • The Lysosomal & Rare Disorders Research & Treatment Center Fairfax, Virginia, United States
  • Washington University School of Medicine St Louis, Missouri, United States
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Recruiting

A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis

NCT07054515

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 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 Childrens Center - PIN Rochester, Minnesota, United States
  • Children's Medical Center Dallas Dallas, Texas, United States
View all 38 locations
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Recruiting

A Gaucher Disease Gene Therapy Trial With FLT201

NCT07223944

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Kaiser Permanente - CA Los Angeles, California, United States
  • UCLA Health Los Angeles, California, United States
  • University of California, San Francisco (UCSF) San Francisco, California, United States
  • University of Colorado Anschutz Aurora, Colorado, United States
View all 33 locations
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