Vertex-770 in People with CF with Homozygous Delta F508 Mutation (DISCOVER)
Number of Participants Being Recruited:
Single / Multi-Center:
STUDY BACKGROUND INFORMATION:
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.
>= 12 Years
>= 40 Percent Predicted
P. aeruginosa status:
B. cepacia status:
Other Primary Eligibility Requirements:
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Sponsor Contact Information:
Medical Monitor, Vertex (617) 444-6777 firstname.lastname@example.org
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 (KalydecoÂ®, 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.