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Clinical trial condition

Homocystinuria

Explore clinical trials studying Homocystinuria. Study availability and eligibility vary by location and protocol.

14 public trials 3 recruiting studies
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Homocystinuria trial results

Recruiting

Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)

NCT03406611

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • Travere Investigational Site Aurora, Colorado, United States
  • Travere Investigational Site Miami, Florida, United States
  • Travere Investigational Site Indianapolis, Indiana, United States
  • Travere Investigational Site Portland, Maine, United States
View Trial Details
Recruiting

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment

NCT06247085

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • Yale University School of Medicine New Haven, Connecticut, United States
  • Emory University Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 52 locations
View Trial Details
Enrolling by invitation

A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE)

NCT06431893

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
Locations
  • Phoenix Children's Hospital Phoenix, Arizona, United States
  • Yale University School of Medicine New Haven, Connecticut, United States
  • Emory University Atlanta, Georgia, United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States
View all 52 locations
View Trial Details
Active, not recruiting

Early Check: Expanded Screening in Newborns

NCT03655223

Conditions
17 Alpha-Hydroxylase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, +456 more
Locations
  • RTI International Research Triangle Park, North Carolina, United States
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Completed

Evaluation of the Express Plus Range

NCT05051657

Conditions
Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +18 more
Locations
  • Royal Belfast Hospital for Sick Children Belfast, United Kingdom
  • Birmingham Women's and Children's Hospital Birmingham, United Kingdom
  • Bristol Royal Hospital for Children Bristol, United Kingdom
  • Greater Glasgow and Clyde NHS Foundation Trust Glasgow, United Kingdom
View Trial Details
Completed

Baby Detect : Genomic Newborn Screening

NCT05687474

Conditions
3-Hydroxy 3-Methyl Glutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency, +368 more
Locations
  • CRMN, Hôpital La Citadelle Liège, Wallonia, Belgium
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