An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy
Condition: Muscular Dystrophy, Duchenne; Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne; Musculoskeletal Diseases; Muscular Diseases; Muscular Disorders, Atrophic; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked
Intervention: Ataluren
Intervention description: Ataluren will be administered per the dose and schedule specified in the arm.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 219.0
Sex: Male
Minimum age: 7 Years
Maximum age: 15 Years
Healthy volunteers: No
Keywords: Duchenne muscular dystrophy; Dystrophinopathy; Nonsense mutation; Premature stop codon; Becker muscular dystrophy; DMD/BMD; PTC124; Ataluren

Summary:

      The primary objective of this study is to obtain long term safety data of ataluren in male
      participants with nonsense mutation dystrophinopathy (who participated and completed a
      previous Phase 3 study of ataluren [PTC124-GD-020-DMD {NCT01826487}]) to augment the overall
      safety database. Screening and baseline procedures are structured to avoid a gap in treatment
      between the double-blind study (PTC124-GD-020-DMD) and this extension study.

      This study may be further extended by amendment until either ataluren becomes commercially
      available or the clinical development of ataluren in duchenne muscular dystrophy (DMD) is
      discontinued.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Completion of study treatment in the previous Phase 3, double-blind study
             (PTC124-GD-020-DMD).

          -  Evidence of signed and dated informed consent/assent document(s) indicating that the
             participant (and/or his parent/legal guardian) has been informed of all pertinent
             aspects of the trial. Note: If the study candidate is considered a child under local
             regulation, a parent or legal guardian must provide written consent prior to
             initiation of study screening procedures and the study candidate may be required to
             provide written assent. The rules of the responsible Institutional Review
             Board/Independent Ethic Committee (IRB/IEC) regarding whether 1 or both parents must
             provide consent and the appropriate ages for obtaining consent and assent from the
             participant should be followed.

          -  In participants who are sexually active, willingness to abstain from sexual
             intercourse or employ a barrier or medical method of contraception during the period
             of study drug administration and 6-week follow-up period.

          -  Willingness and ability to comply with scheduled visits, ataluren administration plan,
             study procedures, laboratory tests, and study restrictions.

        Exclusion Criteria:

          -  Known hypersensitivity to any of the ingredients or excipients of the study drug
             (Litesse® UltraTM [refined polydextrose], polyethylene glycol 3350, Lutrol® micro F127
             [poloxamer 407], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla,
             Cab-O-Sil® M5P [colloidal silica], and magnesium stearate).

          -  Ongoing participation in any other therapeutic clinical trial.

          -  Prior or ongoing medical condition (for example, concomitant illness, psychiatric
             condition, behavioral disorder, alcoholism, drug abuse), medical history, physical
             findings, electrocardiogram (ECG) findings, or laboratory abnormality that, in the
             investigator's opinion, could adversely affect the safety of the participant, makes it
             unlikely that the course of treatment or follow-up would be completed, or could impair
             the assessment of study results.