A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
Active, not recruiting · Phase 3 · NCT05515536
Conditions: Friedreich Ataxia
At a glance
- Research payments reported, 2025
- $224,508
- U.S. investigators named
- 5
- Sites paid
- 5
- Planned or actual enrollment
- 130
About the trial
- Sponsor
- PTC Therapeutics
- Tested
- Vatiquinone (drug)
- Dates
- Started 2022-12-08; completion 2027-12-31
- Results posted
- Not yet
The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone.
From ClinicalTrials.gov, last updated 2025-10-30. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Ptc Therapeutics, Inc. reported $224,508 in research payments for this study in 2025 (79 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
- Susan L. Perlman, MD Neurology · Los Angeles, CA
- Sankarasubramoney H. Subramony, MD Neurology · Gainesville, FL
- Theresa Zesiewicz, MD Neurology · Tampa, FL
- Katherine D. Mathews, MD Physical Medicine & Rehabilitation (PM&R) · Iowa City, IA
- David R. Lynch Child Neurology · Philadelphia, PA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Children's Hospital of Philadelphia, PA $100,870
- University of Iowa Hospital & Clinics, IA $43,791
- Ronald Reagan UCLA Medical Center, CA $29,855
- The University of South Florida Board of Trustees $29,816
- University of Florida $20,177
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.