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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

Active, Not Recruiting

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

NCT04910776

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations
  • Stanford Hospital- Site Number : 8400006 Stanford, California, United States
  • Children's Hospitals and Clinics of Minnesota- Site Number : 8400008 Minneapolis, Minnesota, United States
  • Advanced Medical Genetics- Site Number : 8400002 Hawthorne, New York, United States
  • Duke University Medical Center- Site Number : 8400004 Durham, North Carolina, United States
View all 16 locations
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Active, Not Recruiting

A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)

NCT04849741

Conditions
Alexander Disease, Brain Diseases, Brain Diseases, Metabolic, +13 more
Locations
  • Lucile Packard Children's Hospital Stanford Palo Alto, California, United States
  • Children's Hospital of Atlanta Atlanta, Georgia, United States
  • Massachusetts General Hospital Boston, Massachusetts, United States
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
View all 13 locations
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Active, Not Recruiting

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

NCT04411654

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • UCSF Benioff Children's Hospital, 747 52nd St Oakland, California, United States
  • University of Minnesota Masonic Children's Hospital, 2450 Riverside Avenue Minneapolis, Minnesota, United States
  • Children's Hospital of Pittsburgh, 4401 Penn Avenue Pittsburgh, Pennsylvania, United States
  • Lysosomal & Rare Disorders Research and Treatment Center Fairfax, Virginia, United States
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Active, Not Recruiting

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

NCT05206773

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Nephrology Clinic at Kirklin Clinic of UAB Hospital_Investigational Site Number: 8400011 Birmingham, Alabama, United States
  • UCLA Medical Center_Investigational Site Number: 8400006 Los Angeles, California, United States
  • University of California Irvine Medical Center- Site Number : 8400019 Orange, California, United States
  • Advent Health Orlando_Investigational Site Number: 8400008 Orlando, Florida, United States
View all 58 locations
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