NCT01685801

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

A Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis, Residual CFTR Function, and FEV1 ≥40% Predicted

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: Vertex Pharmaceuticals Incorporated · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	Ivacaftor	150 milligram (mg) tablet orally every 12 hours up to 12 weeks.
DRUG	Placebo-matched-to-ivacaftor tablet	Orally every 12 hours up to 4 weeks.

Timeline

• Start date: 2012-09-01
• Primary completion: 2014-04-01
• Completion: 2014-04-01
• First posted: 2012-09-14
• Last updated: 2015-05-19
• Results posted: 2015-05-19

Locations

1 site across 1 country: United States