CTP-656 Clinical Trial Initiated

CTP-656 for cystic fibrosis patients with CFTR gating mutations is now loaded in the clinicaltrials.gov website here NCT02971839.  The phase 2 trial is expected to start enrollment in January of 2017 to avoid the busy holiday season approaching.  The readout completion date is by the end of 2017.  Sweat chloride will be the primary endpoint of the study with FEV1 secondary.  With only 6-8 patients in each arm, the trial is not specifically powered for efficacy, but more likely to compare the three different dosages, and decide which one will be taken further into a phase 3 clinical trial, where FEV1 will be the primary endpoint.

Estimated Enrollment:	40
Study Start Date:	January 2017
Estimated Primary Completion Date:	December 2017 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: CTP-656, Dose 1, QD Oral tablet dosed once-daily for 28 days	Drug: CTP-656, Dose 1 (QD)
Experimental: CTP-656, Dose 2, QD Oral tablet dosed once-daily for 28 days	Drug: CTP-656, Dose 2 (QD)
Experimental: CTP-656, Dose 3, QD Oral tablet dosed once-daily for 28 days	Drug: CTP-656, Dose 3 (QD)
Active Comparator: Kalydeco, 150 mg Tablet (open label) 150 mg, oral tablet dosed twice-daily for 28 days	Drug: Kalydeco, 150 mg Tablet (BID; open-label)
Placebo Comparator: Placebo, Oral Tablet, QD Oral tablet dosed once-daily for 28 days	Drug: Placebo (QD)

Detailed Description:

This is a randomized, parallel-group, double-blind, placebo controlled multicenter study to evaluate the safety and efficacy of CTP-656 in CF patients with CFTR gating mutations, compared to Kalydeco, for a total of 28 days. Subjects will be randomized to receive either double-blind CTP-656 or placebo, or open-label Kalydeco.