Date: 2016-12-21
Type of information: Initiation of the trial
phase: 2
Announcement: initiation of the trial
Company: Concert Pharmaceuticals (USA - MA)
Product: CTP-656 (D9-ivacaftor ) deuterated ivacaftor
Action
mechanism: CFTR potentiator. CTP-656 is a novel CFTR potentiator that was developed by Concert's novel application of deuterium chemistry to modify ivacaftor. Ivacaftor is marketed by Vertex Pharmaceuticals under the brand name Kalydeco®. Concert is initially developing CTP-656 as a potential monotherapy treatment for cystic fibrosis due to gating mutations of the gene that encodes for cystic fibrosis transmembrane conductance regulator (CFTR).
Disease: cystic fibrosis
Therapeutic area: Rare diseases - Genetic diseases
Country: USA
Trial
details: This phase 2 study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation. Patients enrolled in the 28-day study will receive one of three doses (20 mg, 100 mg and 150 mg) of CTP-656 once-daily or placebo. There will also be an open-label Kalydeco comparator arm in the trial. Approximately 30-40 patients will be enrolled in the Phase 2 trial which will be conducted in multiple centers in the U.S. The primary endpoint of the Phase 2 trial is a change from baseline in sweat chloride at Day 28. Secondary endpoints include change in percent predicted forced expiratory volume (FEV1) and change from baseline in CFQ-R Respiratory Domain. The study is being conducted within the Cystic Fibrosis Foundation's Therapeutic Development Network (TDN). (NCT02971839)
Latest
news: * On December 21, 2016, Concert Pharmaceuticals announced the initiation of a U.S.-based Phase 2 clinical trial evaluating CTP-656 (deuterated ivacaftor), a next generation CFTR potentiator being developed for the treatment of cystic fibrosis. Topline results of the Phase 2 trial of CTP-656 are expected by year-end 2017. In Phase 1 clinical evaluations, CTP-656 provided superior key exposure parameters compared to Kalydeco. CTP-656 was well-tolerated and its safety profile was comparable to that of Kalydeco. No serious adverse events were reported in Phase 1 studies.
