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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01685801
• Date of registration: 06/09/2012
• Prospective Registration: No
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function
• Scientific title: A Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis, Residual CFTR Function, and FEV1 =40% Predicted
• Date of first enrolment: September 2012
• Target sample size: 24
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01685801
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Jerry Nick, MD
• Affiliation: National Jewish Health

Key inclusion & exclusion criteria

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



Age minimum: 12 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Ivacaftor

Drug: Placebo-matched-to-ivacaftor tablet

Primary Outcome(s)

Cycle 1 and Cycle 2: Absolute Change From Cycle Baseline In Percent Predicted Forced Expiratory Volume In 1 Second (FEV1) After 2 Weeks of Treatment [Time Frame: Cycle 1 baseline, Cycle 1 Day 15 (for Cycle 1 reporting arms); Cycle 2 baseline, Cycle 2 Day 15 (for Cycle 2 reporting arms)]

Secondary Outcome(s)

Open-label Period: Absolute Change From Open-label Baseline In Percent Predicted Forced Expiratory Volume In 1 Second (FEV1) at Day 57 [Time Frame: Open-label Baseline, Open-label Day 57]

Open-label Period: Absolute Change From Open-label Baseline In Lung Clearance Index (LCI) at Day 57 [Time Frame: Open-label Baseline, Open-label Day 57]

Open-label Period: Absolute Change From Study Baseline In Sweat Chloride at Day 57 [Time Frame: Study Baseline, Open-label Day 57]

Cycle 1 and Cycle 2: Absolute Change From Cycle Baseline In Lung Clearance Index (LCI) After 2 Weeks of Treatment [Time Frame: Cycle 1 baseline, Cycle 1 Day 15 (for Cycle 1 reporting arms); Cycle 2 baseline, Cycle 2 Day 15 (for Cycle 2 reporting arms)]

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [Time Frame: From first dose of study drug through completion of follow-up visit (up to 26 weeks)]

Open-label Period: Absolute Change From Open-label Baseline In Weight at Day 57 [Time Frame: Open-label Baseline, Open-label Day 57]

Secondary ID(s)

VX12-770-113

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 19/05/2015
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01685801