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- Conditions
- Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
- Locations
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- 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
Recruiting
Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
NCT02998710
- Conditions
- Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +13 more
- Locations
-
- Travere Investigational Site - Virtual Site Culver City, California, United States
- Travere Investigational Site (Enrolling 1 to <5 Year-olds Only) Aurora, Colorado, United States
- Travere Investigational Site (Enrolling 1 to <5 Year-olds Only) Washington D.C., District of Columbia, United States
- Travere Investigational Site Atlanta, Georgia, United States
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
NCT05462132
- Conditions
- Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
- Locations
-
- High Point Clinical Trials Center High Point, North Carolina, United States
NCT01192828
- Conditions
- Amino Acid Metabolism, Inborn Errors, Brain Diseases, Brain Diseases, Metabolic, +12 more
- Locations
-
- Childrens Hospital Colorado Aurora, Colorado, United States
- University of Colorado Aurora, Colorado, United States
- Duke University Durham, North Carolina, United States
- Childrens Hospital of Philadelphia Philadelphia, Pennsylvania, United States
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
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