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(EDITED) Vertex kicks off phase 3 trial of triple combination regimen for people with the most common genetic form of Cystic Fibrosis

Edit: The first paragraph used to say: Vertex is conducting the global ECLIPSE Phase 3 clinical trial program to evaluate VX-659 in triple combination with tezacaftor and ivacaftor for people with the most common genetic form of cystic fibrosis (CF), after which the company wants to submit it for regulatory approval in the US. 

To clarify, one of the studies (ECLIPSE F/F) is evaluating VX-659, tezacaftor and ivacaftor as a triple combination regimen in patients with two F508del mutations, which is the most common genetic form of CF. The other trial (ECLIPSE F/MF) is evaluating the regimen in patients with one F508del mutation and one minimal function mutation. The program currently consists of two trials, ECLIPSE F/MF and ECLIPSE F/F, with additional studies planned as part of this clinical trial program.

As Vertex has noted, the most common form of CF is with the patients who have two copies of the F508del mutation.

The primary endpoint of the study ECLIPSE F/F that will enroll about 100 patients is the mean absolute change from baseline in percent predicted forced expiratory volume in one second (ppFEV1) at week four of treatment. ECLIPSE F/MF has 360 patients.

Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex, said, “We continue to make rapid and significant progress in our efforts to advance our two triple combination regimens into Phase 3 development. The first Phase 3 study we announced in February is designed to support approval of the VX-659 triple combination in patients with one F508delmutation and one minimal function mutation who currently have no treatment that addresses the underlying cause of disease. This second study is designed to enable us to broaden the potential label for this regimen to include those with the most common genetic form of cystic fibrosis.”

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