The primary objective of this registration-directed Phase IIb trial is to demonstrate the efficacy of PTC124 as measured by improvements in the walking ability of patients with this progressive genetic disease.
In this double-blind study, patients will be randomized to receive placebo, or one of two dose levels of PTC124, three times per day. Eligible patients will be boys with nonsense-mutation-mediated Duchenne or Becker muscular dystrophy (DMD/BMD) who are at least five years of age and are able to walk at least 75 meters or approximately 80 yards in six minutes.
PTC expects to enroll a total of 165 patients at approximately 35 investigational sites; all study subjects will undergo 48 weeks of blinded treatment. Thereafter, all participants, including those who have been receiving placebo, will be eligible to enroll in an open-label PTC124 extension study.
Stuart Peltz, president and CEO of PTC Therapeutics, said: “Our future plans for PTC124 include the initiation of longer-term studies in CF, as well as additional proof-of-concept studies in other indications.”