Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function

Study Results

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-09-30

Study Completion Date

2014-04-30

Brief Summary

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This study is a multiple within participant crossover study to evaluate the effect of ivacaftor on lung function in participants aged 12 years and older with cystic fibrosis (CF) who have phenotypic or molecular evidence of residual CF transmembrane conductance regulator (CFTR) function.

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Detailed Description

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CFTR Mutations associated with residual function or defective messenger ribonucleic acid (mRNA) splicing include the following:

R117H, E56K, P67L, D110E, D110H, R117C, R347H, R352Q, A455E, D579G, S945L, L206W, R1070W, F1074L, D1152H, S1235R, D1270N, 2789+5G-\>A, 3849+10kbC-\>T, 3272-26A-\>G, 711+5G-\>A, 3120G-\>A, 1811+1.6kbA-\>G, 711+3A-\>G, 1898+3A-\>G, 1898+1G-\>A, 1717-1G-\>A, 1717-8G-\>A, 1342-2A-\>C, 405+3A-\>C, 1716G/A 1811+1G-\>C, 1898+5G-\>T, 3850-3T-\>G, IVS14b+5G-\>A, 1898+1G-\>T, 4005+2T-\>C, 621+3A-\>G, 621+1G-\>T.

Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Caregivers

Study Groups

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Ivacaftor, Placebo, Ivacaftor, Placebo (IPIP)

During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and Cycle 2 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor.

Group Type EXPERIMENTAL

Ivacaftor

Intervention Type DRUG

150 milligram (mg) tablet orally every 12 hours up to 12 weeks.

Placebo-matched-to-ivacaftor tablet

Intervention Type DRUG

Orally every 12 hours up to 4 weeks.

Ivacaftor, Placebo, Placebo, Ivacaftor (IPPI)

During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and Cycle 2 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor.

Group Type EXPERIMENTAL

Ivacaftor

Intervention Type DRUG

150 milligram (mg) tablet orally every 12 hours up to 12 weeks.

Placebo-matched-to-ivacaftor tablet

Intervention Type DRUG

Orally every 12 hours up to 4 weeks.

Placebo, Ivacaftor, Ivacaftor, Placebo (PIIP)

During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and Cycle 2 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor.

Group Type EXPERIMENTAL

Ivacaftor

Intervention Type DRUG

150 milligram (mg) tablet orally every 12 hours up to 12 weeks.

Placebo-matched-to-ivacaftor tablet

Intervention Type DRUG

Orally every 12 hours up to 4 weeks.

Placebo, Ivacaftor, Placebo, Ivacaftor (PIPI)

During the Crossover Period, study drug was administered in 2-week alternating cycles with a minimum of 4-week washout period between Cycle 1 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and Cycle 2 \[(Period 1 - Day 1 to 14) (Period 2 - Day 15 to 29)\] and between Cycle 2 and the Open-label Period (Day 1 to 57). During the Open-label Period, all participants received ivacaftor.

Group Type EXPERIMENTAL

Ivacaftor

Intervention Type DRUG

150 milligram (mg) tablet orally every 12 hours up to 12 weeks.

Placebo-matched-to-ivacaftor tablet

Intervention Type DRUG

Orally every 12 hours up to 4 weeks.

Interventions

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Ivacaftor

150 milligram (mg) tablet orally every 12 hours up to 12 weeks.

Intervention Type DRUG

Placebo-matched-to-ivacaftor tablet

Orally every 12 hours up to 4 weeks.

Intervention Type DRUG

Other Intervention Names

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Kalydeco, VX-770

Eligibility Criteria

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Inclusion Criteria

* Male or female participants with confirmed diagnosis of CF
* Clinical evidence of residual CFTR function based on any 1 of the following: 1) Clinically documented residual exocrine pancreatic function, 2) Sweat chloride value less than equal to (\<=) 80 millimole per liter (mmol/L) at screening, or 3) Age of diagnosis greater than equal to (\>=) 12 years and at least 1 copy of a CFTR mutation associated with residual CFTR function or defective mRNA splicing
* FEV1 \>= 40 percent (%)
* 12 years of age or older
* Willing to agree to meet the contraception requirements
* Able to swallow tablets

Exclusion Criteria

* A copy of any of the following CFTR mutations: G551D, G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, or G1349D
* Unable to perform spirometry
* An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1
* Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within the 30 days prior to screening

Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No