Clinical trial NCT01009294 (phase 2, status: terminated)
Conditions: duchenne muscular dystrophy, becker muscular dystrophy
ICD codes: G71.01, G71.01
Drugs: ataluren, chronic corticosteroid therapy
SMILES: OC(=O)C1=CC=CC(=C1)C1=NOC(=N1)C1=CC=CC=C1F, [H][C@@]12CC[C@](O)(C(=O)CO)[C@@]1(C)C[C@H](O)[C@@]1([H])[C@@]2([H])CCC2=CC(=O)CC[C@]12C
Reason stopped: 
    terminated early because a similar study with ataluren exhibited lack of efficacy at the high
    dose (not due to safety concerns).
  
Eligibility criteria:

        Inclusion Criteria:

          -  Diagnosis of DMD or BMD

          -  Presence of a nonsense mutation in the dystrophin gene

          -  Unable to ambulate independently for ≥1 year due to DMD/BMD

          -  Presence of sufficient shoulder and elbow function to perform study-related functional
             procedures (for example, 9-hole peg test)

          -  Adequate hepatic, renal, and adrenal function

          -  Ability to provide evaluable pretreatment echocardiogram and lung function assessments

          -  Willingness and ability to comply with scheduled visits, drug administration plan,
             study procedures, laboratory tests, and study restrictions

          -  Ability to provide written informed consent (parental/guardian consent if
             applicable)/assent (if <18 years of age)

        Exclusion Criteria:

          -  Initiation of systemic corticosteroid therapy within 6 months prior to start of study
             treatment or use of systemic aminoglycoside antibiotic within 3 months prior to start
             of study treatment

          -  Use of any intermittent systemic corticosteroid therapy regimen (for example, 10 days
             on followed by 10 days off, weekend dosing, every-other-day dosing); note that
             participants must have either been receiving a daily dosing regimen of prednisone,
             prednisolone, or deflazacort at the time of enrollment into the study or must have not
             been receiving any systemic corticosteroids

          -  Any change in treatment for congestive heart failure within 3 months prior to start of
             study treatment

          -  Ongoing warfarin or phenytoin therapy

          -  Prior therapy with ataluren

          -  Known hypersensitivity to any of the ingredients or excipients of ataluren (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], magnesium stearate).

          -  Exposure to another investigational drug within 2 months prior to start of study
             treatment

          -  History of major surgical procedure within 1 month prior to start of study treatment
             or expectation of major surgical procedure (for example, scoliosis surgery) during the
             48-week treatment period of the study

          -  Ongoing immunosuppressive therapy (other than corticosteroids)

          -  Ongoing participation in any other clinical trial

          -  Requirement for daytime ventilator assistance

          -  Uncontrolled clinical symptoms and signs of congestive heart failure

          -  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