A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
Completed · Phase 3 · NCT05166161
Conditions: Phenylketonuria
At a glance
- Research payments reported, 2025
- $754,107
- U.S. investigators named
- 8
- Sites paid
- 8
- Planned or actual enrollment
- 247
About the trial
- Sponsor
- PTC Therapeutics
- Tested
- PTC923 (drug)
- Dates
- Started 2022-02-14; completion 2026-07-21
- Results posted
- Not yet
The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption.
From ClinicalTrials.gov, last updated 2026-09-09. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Ptc Therapeutics, Inc. reported $754,107 in research payments for this study in 2025 (62 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
- Ingrid Cristian, MD Medical Genetics · Orlando, FL
- Roberto T. Zori, MD Medical Genetics · Gainesville, FL
- Melissa D. Lah, MD Clinical Molecular Genetics · Indianapolis, IN
- Stephanie J. Sacharow, M.D. Medical Genetics · Boston, MA
- Margo S. Breilyn, MD Medical Genetics · Bronx, NY
- Gerard Vockley, MD, PHD Specialist · Pittsburgh, PA
- Ashley L. Andrews, MSN, CPNP Nurse Practitioners · Salt Lake City, UT
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- University of Utah Hospital and Clinics, UT $233,629
- Boston Children's Hospital, MA $187,251
- The Trustees of Indiana University $149,475
- Children's Hospital Colorado, CO $84,616
- University of Florida $36,102
- University of Pittsburgh Physicians $27,430
- Icahn School of Medicine at Mount Sinai $20,604
- Orlando Health, FL $15,000
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.