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Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
Sponsor: Travere Therapeutics, Inc.
Summary
The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 1 to 65 years under current clinical management practices
Official title: A Multicenter, Observational, Prospective, Natural History Study of Homocystinuria Due to Cystathionine Beta-synthase Deficiency in Pediatric and Adult Patients (ACAPPELLA)
Key Details
Gender
All
Age Range
1 Year - 65 Years
Study Type
OBSERVATIONAL
Enrollment
150
Start Date
2017-01
Completion Date
2026-08
Last Updated
2024-11-22
Healthy Volunteers
No
Conditions
Locations (11)
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
Travere Investigational Site
Indianapolis, Indiana, United States
Travere Investigational Site
Boston, Massachusetts, United States
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)
Philadelphia, Pennsylvania, United States
Travere Investigational Site
Dublin, Ireland
Travere Investigational Site
Doha, Qatar, Qatar
Travere Investigational Site
Doha, Qatar
Travere Investigational Site
Salford, Manchester, United Kingdom