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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

A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

NCT04093349

Conditions
Acid Maltase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +17 more
Locations
  • Barrow Neurological Institute Phoenix, Arizona, United States
  • University of California Irvine Health Orange, California, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • University of Kansas Medical Center Research Institute Kansas City, Kansas, United States
View all 29 locations
View Trial Details
Active, Not Recruiting

Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE)

NCT03406611

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • Travere Investigational Site - Virtual Site Culver City, California, United States
  • Travere Investigational Site Aurora, Colorado, United States
  • Travere Investigational Site Miami, Florida, United States
  • Travere Investigational Site Indianapolis, Indiana, United States
View Trial Details
Active, Not Recruiting

Dose Finding Study to Evaluate Safety and Efficacy of 3 Dosages of SAP 001.

NCT05690204

Conditions
Arthritis, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Crystal Arthropathies, +12 more
Locations
  • California Site Sacramento, California, United States
  • California Site San Diego, California, United States
  • Denver Site Denver, Colorado, United States
  • Florida Site DeBary, Florida, United States
View all 16 locations
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Active, Not Recruiting

Gene Therapy Study for Children With CLN5 Batten Disease

NCT05228145

Conditions
Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +10 more
Locations
  • University of Rochester Rochester, New York, United States
  • Great Ormond Street Hospital for Children London, United Kingdom
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Active, Not Recruiting

Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

NCT04628871

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Carbohydrate Metabolism, Inborn Errors, +24 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
  • New York University Grossman School of Medicine New York, United States
  • University of North Carolina Chapel Hill, North Carolina, United States
View Trial Details
Active, Not Recruiting

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease

NCT04519749

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of California at San Diego La Jolla, California, United States
  • Emory University Atlanta, Georgia, United States
  • Children's Hospital of Pittsburgh of UPMC Pittsburgh, Pennsylvania, United States
  • Lysosomal & Rare Disorders Research & Treatment Center, Inc Fairfax, Virginia, United States
View Trial Details

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