VX -770 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:
Recruiting 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:
-
FEV1:
- 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
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.
