A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation



Status:Completed
Conditions:Pulmonary
Therapuetic Areas:Pulmonary / Respiratory Diseases
Healthy:No
Age Range:12 - Any
Updated:6/14/2018
Start Date:August 2015
End Date:June 7, 2016

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A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation and With a Second CFTR Mutation That Is Not Likely to Respond to VX-661 and/or Ivacaftor Therapy (F508del/NR)

Study to evaluate the efficacy of VX-661 in combination with ivacaftor (IVA, VX-770) through
Week 12 in participants with cystic fibrosis (CF) who are heterozygous for the F508del
mutation on the CF transmembrane conductance regulator (CFTR) gene and with a second CFTR
mutation that is not likely to respond to VX-661 and/or IVA therapy (F508del/not responsive
[NR]).


Inclusion Criteria:

- Confirmed diagnosis of CF defined as a sweat chloride value greater than or equal to
(>=)60 millimole per liter (mmol/L) by quantitative pilocarpine iontophoresis.

- Heterozygous for the F508del-CFTR mutation and with a second CFTR mutation that is not
likely to respond to VX-661 and/or ivacaftor therapy, genotype to be confirmed via
assessment at the Screening Visit.

- Forced Expiratory Volume in 1 Second (FEV1) >=40 percent (%) and less than or equal to
(<=)90% of predicted normal for age, sex, and height at Screening Visit.

Exclusion Criteria:

- History of any comorbidity that, in the opinion of the investigator, might confound
the results of the study or pose an additional risk in administering study drug to the
participant.

- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in
therapy (including antibiotics) for pulmonary disease within 28 days before Day 1
(first dose of study drug).

- History of solid organ or hematological transplantation.

- Ongoing or prior participation in an investigational drug study or use of commercially
available CFTR modulator within 30 days of screening.

- Pregnant or nursing females.
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