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Clinical trial condition

Metabolism, Inborn Errors

Explore clinical trials studying Metabolism, Inborn Errors. Study availability and eligibility vary by location and protocol.

2,493 public trials 456 recruiting studies
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Metabolism, Inborn Errors trial results

Active, Not Recruiting

Open-Label Study of AG10 in Patients with Cardiomyopathy

NCT03536767

Conditions
Amyloid Cardiomyopathy, Amyloid Neuropathies, Amyloid Neuropathies, Familial, +13 more
Locations
  • Stanford University Palo Alto, California, United States
  • University of California San Francisco San Francisco, California, United States
  • Yale University New Haven, Connecticut, United States
  • Northwestern University Chicago, Illinois, United States
View Trial Details
Active, Not Recruiting

Long-term Follow-up of Participants With Cerebral Adrenoleukodystrophy Who Were Treated With Lenti-D Drug Product

NCT02698579

Conditions
Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +24 more
Locations
  • Mattel Children's Hospital-UCLA Los Angeles, California, United States
  • Lucile Packard Children's Hospital - Stanford Palo Alto, California, United States
  • Boston Children's Hospital/Massachusetts General Hospital Boston, Massachusetts, United States
  • University of Minnesota Minneapolis, Minnesota, United States
View all 13 locations
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Active, Not Recruiting

Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)

NCT04806451

Conditions
Adrenal Gland Diseases, Adrenal Hyperplasia, Congenital, Adrenogenital Syndrome, +17 more
Locations
  • Neurocrine Clinical Site Birmingham, Alabama, United States
  • Neurocrine Clinical Site Los Angeles, California, United States
  • Neurocrine Clinical Site Orange, California, United States
  • Neurocrine Clinical Site San Diego, California, United States
View all 46 locations
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Active, Not Recruiting

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

NCT03566043

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +23 more
Locations
  • University of California San Francisco, Benioff Children's Hospital Oakland, California, United States
  • St. Peter's University Hospital New Brunswick, New Jersey, United States
  • Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
  • Children's Hospital of Pittsburgh - UPMC: Program for Neurodevelopment in Rare Disorders Pittsburgh, Pennsylvania, United States
View Trial Details
Active, Not Recruiting

AAV Gene Therapy Study for Subjects with PKU

NCT04480567

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +10 more
Locations
  • University of South Florida Tampa, Florida, United States
  • Morristown Medical Center Morristown, New Jersey, United States
  • University Hospital Birmingham NHS Foundation Trust Birmingham, United Kingdom
View Trial Details

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