Natural History Study of Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (ACAPPELLA)
Completed · NCT02998710
Conditions: Homocystinuria Due to CBS Deficiency
At a glance
- Research payments reported, 2025
- $233,554
- U.S. investigators named
- 6
- Sites paid
- 7
- Planned or actual enrollment
- 110
About the trial
- Sponsor
- Travere Therapeutics, Inc.
- Dates
- Started 2017-01; completion 2026-07-31
- Results posted
- Not yet
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
From ClinicalTrials.gov, last updated 2026-09-09. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Travere Therapeutics, Inc. reported $233,554 in research payments for this study in 2025 (37 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Kimberly A. Chapman, M.D. Medical Genetics · Los Angeles, CA
- Janet Thomas, MD Medical Genetics · Aurora, CO
- Hong Li, M.D.,PH.D. Medical Genetics · Decatur, GA
- Melissa D. Lah, MD Clinical Molecular Genetics · Indianapolis, IN
- Harvey L. Levy, MD Medical Genetics · Boston, MA
- Can H. Ficicioglu, M.D. Medical Genetics · Philadelphia, PA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Emory University Hospital, GA $62,882
- Children's Hospital Colorado, CO $46,240
- Children's Hospital of Philadelphia, PA $43,807
- Boston Childrens Hospital $32,711
- The Trustees of Indiana University $28,988
- Children S National Research Institute $16,333
- University of Miami $2,594
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.