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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

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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