Phase 2
Completed N=24
Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function
Source: ClinicalTrials.gov NCT01685801 ↗Enrolled (actual)
24
Serious AEs
1.5%
Results posted
May 2015
Primary outcomePrimary: 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 — 0.5828; 2.0898; 0.8495; 3.6868 Percent predicted of FEV1
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY 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 |
0.5828; 2.0898; 0.8495; 3.6868; -0.5710; 0.8196 | — |
| SECONDARY Cycle 1 and Cycle 2: Absolute Change From Cycle Baseline In Lung Clearance Index (LCI) After 2 Weeks of Treatment |
0.4596; 0.2822; 0.4111; 0.0770; 0.4321; -0.5306 | — |
| SECONDARY Open-label Period: Absolute Change From Open-label Baseline In Percent Predicted Forced Expiratory Volume In 1 Second (FEV1) at Day 57 |
4.6815; 4.3072; 4.9622 | — |
| SECONDARY Open-label Period: Absolute Change From Open-label Baseline In Lung Clearance Index (LCI) at Day 57 |
-1.5687; -1.3459; -1.7358 | — |
| SECONDARY Open-label Period: Absolute Change From Study Baseline In Sweat Chloride at Day 57 |
-15.74; -14.13; -16.91 | — |
| SECONDARY Open-label Period: Absolute Change From Open-label Baseline In Weight at Day 57 |
1.77; 2.32; 1.35 | — |
| SECONDARY Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
17; 18; 20; 0; 1; 0 | — |
Eligibility 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 ( =) 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
Data sourced from ClinicalTrials.gov (NCT01685801). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.