A Multicenter, Observational, Prospective, Natural History Study of Homocystinuria Due to Cystathionine Beta-synthase Deficiency in Pediatric and Adult Patients (ACAPPELLA)
Status: Recruiting
Location: See all (11) locations...
Study Type: Observational
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
Participation Requirements
Sex: All
Minimum Age: 1
Maximum Age: 65
Healthy Volunteers: f
View:
• 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.
Locations
United States
California
Travere Investigational Site - Virtual Site
COMPLETED
Culver City
Colorado
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)
RECRUITING
Aurora
Washington, D.c.
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)
RECRUITING
Washington D.c.
Georgia
Travere Investigational Site
COMPLETED
Atlanta
Indiana
Travere Investigational Site
COMPLETED
Indianapolis
Massachusetts
Travere Investigational Site
COMPLETED
Boston
Pennsylvania
Travere Investigational Site (Enrolling 1 to <5 Year-olds Only)
RECRUITING
Philadelphia
Other Locations
Ireland
Travere Investigational Site
RECRUITING
Dublin
Qatar
Travere Investigational Site
RECRUITING
Doha
Travere Investigational Site
RECRUITING
Doha
United Kingdom
Travere Investigational Site
COMPLETED
Salford
Contact Information
Primary
Travere Call Center
medinfo@travere.com
1-877-659-5518
Time Frame
Start Date: 2017-01
Estimated Completion Date: 2026-08
Participants
Target number of participants: 150
Related Therapeutic Areas
Sponsors
Leads: Travere Therapeutics, Inc.