NCT02998710Recruiting
Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
Travere Therapeutics, Inc.
Start Date
1/1/2017
Completion Date
8/1/2026
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
Eligibility Criteria
Age Range: 1 years to 65 years
Inclusion Criteria:
* Patients who are clinically diagnosed with homocystinuria
* Male/female patients aged 1 to 65 years
* Patients who consented and/or assented
* Patients who are willing and able to comply with all study-related procedures.
Exclusion Criteria:
* Medically significant postnatal complications or congenital anomalies that are not associated with homocystinuria
* Received any experimental therapy for homocystinuria during the 6 months prior to enrollment or expected to receive any such therapy during duration of the study
Conditions
Homocystinuria Due to CBS Deficiency
Locations
Travere Investigational Site - Virtual Site
Culver City, California 90230
United States
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)
Aurora, Colorado 80045
United States
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)
Washington D.C., District of Columbia 20010
United States
Travere Investigational Site
Atlanta, Georgia 30322
United States
Travere Investigational Site
Indianapolis, Indiana 46202
United States
Travere Investigational Site
Boston, Massachusetts 02115
United States
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)
Philadelphia, Pennsylvania 19104
United States
Travere Investigational Site
Dublin, 1
Ireland
Travere Investigational Site
Doha, Qatar
Qatar
Travere Investigational Site
Doha,
Qatar
Travere Investigational Site
Salford, Manchester M6 8HD
United Kingdom