Last updated on November 2019

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy


Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions: DUCHENNE MUSCULAR DYSTROPHY | Musculoskeletal Disease | Amyotonia Congenita | nervous system disorder | Muscular Dystrophy | Genetic Diseases | Disuse muscle atrophy | Myopathy | Hereditary Disease | X-Linked
  • Age: Between 5 - 100 Years
  • Gender: Male

Inclusion Criteria:

  • Male sex.
  • Age 5 years.
  • Phenotypic evidence of Duchenne Muscular Dystrophy
  • Nonsense point mutation in the dystrophin gene
  • Use of systemic corticosteroids (prednisone/prednisolone or deflazacort)for a minimum of 12 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen for a minimum of 3 months immediately prior to start of study treatment
  • 6MWD 150 meters
  • Ability to perform timed function tests within 30 seconds
  • Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.

Exclusion Criteria:

  • Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start of study treatment.
  • Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.
  • Prior or ongoing therapy with ataluren.
  • Known hypersensitivity to any of the ingredients or excipients of the study drug
  • Exposure to another investigational drug within 6 months prior to start of study treatment, or ongoing participation in any interventional clinical trial.
  • History of major surgical procedure within 12 weeks prior to start of study treatment, or expectation of major surgical procedure during the 72-week placebo-controlled treatment period.
  • Requirement for daytime ventilator assistance or any use of invasive mechanical ventilation via tracheostomy.
  • Uncontrolled clinical symptoms and signs of congestive heart failure
  • Elevated serum creatinine or cystatin C at screening.

Recruitment Status: Open


Brief Description Eligibility Contact Research Team


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