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Study Name: VX -770 (104) in People with CF age 12 and older with 2 copies of the F508del-cftr mutation
Study Type: Interventional
Intervention Category: CFTR Modulation
Study Sponsor: Vertex
Study Phase: 3
Recruitment Status: Trial Completed
Study Drug(s): Kalydeco
Number of Participants Being Recruited: 40
Single / Multi-Center: Multi-Center
STUDY BACKGROUND INFORMATION:
This is a 2-part Phase 2 study assessing VX 770 in people ages 12 yrs and older with two copies of the F508del-cftr mutation. In Part A subjects take VX-770 or placebo by mouth twice daily (morning and evening) for 16 weeks. Subjects who complete Part A and meet certain response criteria will be invited to enroll in Part B. Part B is an open-label extension and eligible subjects will receive VX-770 twice daily for up to 96 weeks.
ELIGIBILITY
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 clinicaltrials.gov. If a specific trial listing for this trial is available, a link to the specific clinicaltrials.gov listing will be present in the "More Information" section below.
FOR MORE INFORMATION:
Sponsor Contact Information: Medical Monitor, Vertex
(617) 444-6777
medicalinfo@vrtx.com
Trial Specific Link on ClinicalTrials.gov: http://www.clinicaltrials.gov/ct2/show/NCT00953706?term=vertex+and+cystic+fibrosis&rank=7
Clinical Research Terms Glossary: Click here
TRIAL RESULTS:
Primary Efficacy:

Not applicable

Secondary Efficacy:

The changes in FEV1 or sweat chloride in Part A were not sustained with ivacaftor treatment from Week 16 to Week 40.

Safety:

Forty-two (37.5%) ivacaftor subjects and 6 (21.4%) placebo subjects from the VX-770-104 (Discover) trial (Part A) qualified for Part B, based on pre-specified eligibility criteria including either =10% change relative to baseline in FEV1 % predicted at any time point through Week 16, or a sweat chloride concentration reduction from baseline =15 mmol/L at both the Day 15 and Week 8 visits. Thirty-three subjects entered the open label follow-on: 28 of these subjects met the FEV1 criterion and 5 met the sweat chloride criterion. Of 6 placebo subjects eligible for Part B, 5 subjects enrolled; all on the basis of FEV1 changes.
No new safety signals were identified.

Citation:

Chest 2012;10(1378):11-2672

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