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news.PTC Therapeutics has concluded patient recruitment in Phase 3 clinical trial of Ataluren, an investigational new drug, in patients with nonsense mutation cystic fibrosis (nmCF).
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Ataluren is a protein restoration therapy designed to enable the formation of full-length, functional cystic fibrosis transmembrane regulator (CFTR) protein in patients with cystic fibrosis due to a nonsense mutation.
The 48-week study is designed to determine whether ataluren can improve lung function in patients with nmCF.
PTC has enrolled 238 patients at 36 sites in North America, Europe and Israel for the trial.
The primary objective of the registration-directed, double-blind, placebo-controlled study is to determine whether Ataluren can improve lung function in patients with nmCF, as measured by forced expiratory volume in 1 second (FEV1).
Additional secondary endpoints are evaluating other aspects of patient function, drug activity, and safety.
The patients who complete the treatment phase of the Phase 3 trial are eligible to participate in a 48-week, open-label extension study, which has begun enrolling patients, said PTC.
PTC president and CEO Stuart Peltz said they are pleased to have completed enrollment of the second pivotal clinical trial of ataluren in patients with a nonsense mutation genetic disorder.
"This is a tremendous achievement and a testament to the commitment of clinical trial patients and their families, as well as study investigators and trial site staff," Peltz said.