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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
110
Start Date
2017-01
Completion Date
2026-07-31
Last Updated
2026-09-09
Healthy Volunteers
No
Conditions
Locations (11)
The Children's Hospital of Colorado
Aurora, Colorado, United States
Children's National Hospital
Washington D.C., District of Columbia, United States
Emory University Hospital
Atlanta, Georgia, United States
Indiana University School of Medicine
Indianapolis, Indiana, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Science 37 - Virtual Site for the US and Canada
Morrisville, North Carolina, United States
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Temple Street Children's University Hospital
Dublin, Ireland
Sidra Medicine
Doha, Qatar, Qatar
Hamad Medical Corporation
Doha, Qatar
Salford Royal NHS Foundation Trust
Salford, Manchester, United Kingdom