Study detail
Recruiting
A Multicenter, Observational, Prospective, Natural History Study of Homocystinuria Due to Cystathionine Beta-synthase Deficiency in Pediatric and Adult Patients (ACAPPELLA)
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
Eligibility
- Age range
- 1–65 years
- Sex
- All
- Healthy volunteers
- No
Detailed criteria
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
Locations (11)
- Travere Investigational Site - Virtual SiteCulver City, California
- Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)Aurora, Colorado
- Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)Washington D.C., District of Columbia
- Travere Investigational SiteAtlanta, Georgia
- Travere Investigational SiteIndianapolis, Indiana
- Travere Investigational SiteBoston, Massachusetts