Cystic Fibrosis Therapy with VX-661 and VX-770 – Phase II

Evaluating the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-661 Monotherapy and VX-661/VX-770 Cotherapy in Patients with Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

Protocol Description

This international multicenter study is being done to learn more about the safety and effects of the drug VX-661 when given alone and in combination with VX-770 to people with cystic fibrosis who have the most common CFTR gene mutation. These medicines-in-development aim to treat the underlying cause of cystic fibrosis by improving the function of the defective protein known to cause the disease.
Study Level: Phase II

Eligibility Criteria

Subject to certain exclusion criteria, adults of both genders who have cystic fibrosis with the F508del-CFTR gene mutation are eligible to participate in this study.  
Males and Females: Ages 18 and older

Participants will be given an initial screening examination inclusive of an overnight stay for pulmonary function testing, sampling and instruction. After a 4-week screening period, participants will be assigned to take some combination of VX-661 alone or a placebo, or with VX-770 or a placebo, at dosages prescribed by the study protocol for a period of 28 days.
Visits: 9
Duration: 12 weeks

Status: Open for Enrollment

Source(s) of Support
Vertex Pharmaceuticals Inc.
Cystic Fibrosis Foundation Therapeutics, Inc.

Primary Investigator(s)

Joseph M. Pilewski, MD

Contact Information

For information or to enroll, please contact:
Elizabeth Hartigan, MPH, RN

Last Update
December 29, 2013
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Last Update
December 29, 2013