Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
Completed · Phase 3 · NCT03179631
Conditions: Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
At a glance
- Research payments reported, 2025
- $51,220
- U.S. investigators named
- 3
- Sites paid
- 3
- Planned or actual enrollment
- 360
About the trial
- Sponsor
- PTC Therapeutics
- Tested
- Ataluren (drug), PLACEBO (drug)
- Dates
- Started 2017-07-06; completion 2023-07-25
- Results posted
- Yes
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
From ClinicalTrials.gov, last updated 2026-03-10. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Ptc Therapeutics, Inc. reported $51,220 in research payments for this study in 2025 (5 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
- John W. Day, MD Physical Medicine & Rehabilitation (PM&R) · Stanford, CA
- Jonathan B. Strober, M.D. Pediatrics · San Francisco, CA
- Leigh Maria K. Ramos-platt, M.D. Child Neurology · Center Valley, PA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Children's Hosp of Los Angeles, CA $45,209
- The Board of Trustees of the Leland Stanford Junior University $5,000
- UCSF Langley Porter Psychiatric Hospital and Clinics, CA $1,011
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.