Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study)
Recruiting · Phase 1, Phase 2 · NCT03406611
Conditions: Homocystinuria
At a glance
- Research payments reported, 2025
- $697,983
- U.S. investigators named
- 7
- Sites paid
- 7
- Planned or actual enrollment
- 39
About the trial
- Sponsor
- Travere Therapeutics, Inc.
- Tested
- Pegtibatinase (drug), Placebo (drug)
- Dates
- Started 2019-01-22; completion 2027-07
- Results posted
- Not yet
Researchers are looking for a better way to treat people who have classical homocystinuria (HCU), a rare condition that is passed down by parents (or "genetic condition"). It is caused by changes in the cystathionine beta-synthase (or "CBS") gene and prevents an enzyme from working correctly in the body. This enzyme breaks down a substance called homocysteine (from dietary methionine found in protein) and keeps both homocysteine and methionine at normal levels. When this enzyme is not working, homocysteine and methionine build up in the blood, which spreads into different tissues of the body and stops these body tissues from working normally. People with HCU can experience problems with v…
From ClinicalTrials.gov, last updated 2026-08-10. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Travere Therapeutics, Inc. reported $697,983 in research payments for this study in 2025 (47 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
- Janet Thomas, MD Medical Genetics · Aurora, CO
- Mustafa Tekin, M.D. Medical Genetics · Miami, FL
- Melissa D. Lah, MD Clinical Molecular Genetics · Indianapolis, IN
- Harvey L. Levy, MD Medical Genetics · Boston, MA
- Wendy E. Smith, MD Medical Genetics · Portland, ME
- Jaya B. Ganesh, M.D. Medical Genetics · New York, NY
- 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
- Boston Childrens Hospital $354,723
- Children's Hospital of Philadelphia, PA $296,641
- Icahn School of Medicine at Mount Sinai $15,742
- The Trustees of Indiana University $15,211
- Mainehealth Maine Medical Center, ME $15,189
- Children's Hospital Colorado, CO $334
- University of Miami $143
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.