Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
Terminated · Phase 3 · NCT01247207
Conditions: Duchenne Muscular Dystrophy
At a glance
- Research payments reported, 2025
- $498,899
- U.S. investigators named
- 27
- Sites paid
- 27
- Planned or actual enrollment
- 265
About the trial
- Sponsor
- PTC Therapeutics
- Tested
- Ataluren (drug)
- Dates
- Started 2010-11-22; completion 2026-02-10
- Results posted
- Yes
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial). The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after initiating ataluren treatment, withdrawal by investigator, withdrawal due to participant unable to tolerate ataluren, participant is eligible to participate in another ataluren nmDBMD clinical trial pr…
From ClinicalTrials.gov, last updated 2026-10-08. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Ptc Therapeutics, Inc. reported $498,899 in research payments for this study in 2025 (248 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
- Saunder M. Bernes, M.D. Child Neurology · Phoenix, AZ
- John W. Day, MD Physical Medicine & Rehabilitation (PM&R) · Stanford, CA
- Craig M. Mcdonald, M.D. Physical Medicine & Rehabilitation (PM&R) · Sacramento, CA
- Perry B. Shieh, MD Neurology · Los Angeles, CA
- Jonathan B. Strober, M.D. Pediatrics · San Francisco, CA
- Julie Parsons, MD Child Neurology · Aurora, CO
- James B. Renfroe, MD Child Neurology · Gulf Breeze, FL
- Han C. Phan, MD Child Neurology · Atlanta, GA
- Katherine D. Mathews, MD Physical Medicine & Rehabilitation (PM&R) · Iowa City, IA
- Peter T. Heydemann, M.D. Child Neurology · Chicago, IL
- Jeffrey M. Statland, MD Physical Medicine & Rehabilitation (PM&R) · Kansas City, KS
- Basil T. Darras, M.D. Child Neurology · Boston, MA
- Erin E. Neil Knierbein, DO Physical Medicine & Rehabilitation (PM&R) · Ann Arbor, MI
- Peter I. Karachunski, MD Child & Adolescent Psychiatry · Minneapolis, MN
- Mai K. El Mallah, MD Pediatric Pulmonology · Durham, NC
- Michio Hirano, M.D. Neurology · New York, NY
- Cuixia Tian, M.D. Child Neurology · Cincinnati, OH
- Erika L. Finanger, M.D. Child Neurology · Portland, OR
- Hoda Z. Abdel-hamid, MD Pediatrics · Pittsburgh, PA
- Leigh Maria K. Ramos-platt, M.D. Child Neurology · Center Valley, PA
- Sabrina S. Yum, MD Child Neurology · Philadelphia, PA
- Ratna K. Bhavaraju-sanka, M.D Physical Medicine & Rehabilitation (PM&R) · San Antonio, TX
- Susan T. Iannaccone, MD Neurology · Dallas, TX
- Timothy Lotze, MD Child Neurology · Houston, TX
- Russell J. Butterfield, MD/PHD Child Neurology · Salt Lake City, UT
- Crystal M. Proud, M.D. Child Neurology · Norfolk, VA
- Seth J. Perlman, MD Child Neurology · Seattle, WA
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Kumc Research Institute Inc $59,113
- The University of Texas Southwestern Medical Center $36,361
- Ronald Reagan UCLA Medical Center, CA $31,238
- The Trustees of Columbia University in The City of New York $30,207
- Children's Hospital of Philadelphia, PA $29,710
- Cincinnati Children's Hospital Medical Center, OH $29,564
- Baylor College of Medicine $29,207
- Northwest Florida Clinical Research Group LLC $26,513
- Seattle Children's Hospital, WA $26,210
- University of Michigan Health System, MI $18,351
- University of Pittsburgh Physicians $16,214
- Regents of the University of Minnesota $15,518
- University of Texas Health Science Center at San Antonio $15,152
- Rush University Medical Center, IL $15,128
- UCSF Langley Porter Psychiatric Hospital and Clinics, CA $14,339
- The Board of Trustees of the Leland Stanford Junior University $14,115
- Children's Hosp of Los Angeles, CA $13,931
- Boston Children's Hospital, MA $11,896
- Childrens Specialty Group PLLC $10,871
- Phoenix Children's Hospital, AZ $10,094
- Duke University $9,814
- Shriners Hospitals for Children $9,776
- University of California Davis Medical Center, CA $6,590
- University of Utah Hospital and Clinics, UT $5,207
- Rare Disease Research LLC $5,165
- University of Iowa Hospital & Clinics, IA $5,130
- Children's Hospital Colorado, CO $3,485
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.