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Study Name: Vertex-770 (104) in People with CF with Homozygous Delta F508 Mutation (DISCOVER)
Study Type: Interventional
Intervention Category: CFTR Modulation
Study Sponsor: Vertex
Study Phase: 2
Recruitment Status: Trial Completed
Study Drug(s): Kalydeco
Number of Participants Being Recruited: 130
Single / Multi-Center: Multi-Center
Cystic Fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. CFTR is a protein in the cell that makes the channel where chloride moves in and out, and is believed to be important in maintaining the normal hydration of lung secretions. VX 770 is a study drug that effects CFTR. This study looked at the safety and effectiveness of VX-770 taken by mouth twice a day in people with CF who are 12 years and older and homozygous with Delta F508 mutation. The study drug was taken by participants for 16 weeks.
Age: >= 12 Years
FEV1: >= 40 Percent Predicted
P. aeruginosa status: Not applicable
B. cepacia status: Not applicable
Other Primary Eligibility Requirements:

Note: Detailed eligibility criteria information may be available on If a specific trial listing for this trial is available, a link to the specific listing will be present in the "More Information" section below.
Sponsor Contact Information: Medical Monitor, Vertex
(617) 444-6777
Trial Specific Link on
Clinical Research Terms Glossary: Click here
Primary Efficacy:

Not applicable

Secondary Efficacy:

The difference in change of FEV1 % predicted from baseline through Week 16 (primary endpoint) between the ivacaftor and placebo groups was 1.7% (P=0.15). The change in sweat chloride from baseline through Week 16 showed a small reduction in the ivacaftor versus placebo groups of â??2.9 mmol/L that was statistically significant (P=0.04).
There were no significant differences in either the ivacaftor or placebo group in the CFQR respiratory domain scores, pulmonary exacerbations, and antibiotic treatment for sinopulmonary signs or symptoms. Change from baseline in weight and BMI were not significantly different between arms.


One hundred and forty subjects aged =12 years with CF, homozygous for the F508del-CFTR mutation were enrolled in this two-part, Phase 2, placebo-controlled study of ivacaftor (Kalydeco®, formerly known as VX-770). Subjects received 150 mg ivacaftor or matched placebo orally administered every 12 h for 16 weeks (Part A- DISCOVER), followed by an open-label 96-week extension for subjects who met pre-specified eligibility criteria (Part B described in VX-770-104 OPEN LABEL Follow-on).
The primary study endpoint was safety. The safety profile of ivacaftor was comparable to placebo. The overall adverse event frequency was similar between ivacaftor (87.5%) and placebo (89.3%) through 16 weeks. The most common events were respiratory-related.
There were no clinically important trends identified in clinical laboratory measures (serum chemistry, hematology, coagulation studies, and urinalysis), vital sign, digital ECG, ambulatory ECG, or physical examination results.


Chest 2012;10(1378):11-2672

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