| Clinical trials identifier | NCT01707290 |
|---|---|
| Study codes | KONTINUE, VX11-770-112 |
| Study title | A Phase 3, Two-Arm, Rollover Study to Evaluate the Safety of Long Term Ivacaftor Treatment in Subjects 6 Years of Age and Older With Cystic Fibrosis and a Non-G551D CFTR Mutation |
| Sponsor | Vertex |
| Drug (brand name) | Ivacaftor (Kalydeco) |
| Drug administration | Tablets taken by mouth |
| Eligible CF patients | Aged at least 6 years old, at least one non G551D gating mutation; R117H CFTR mutation; or residual function mutation, finished feeder study |
| Controlled study? | No |
| Randomised study? | No |
| Blinded study? | No |
| Extension study? | Yes (feeder studies: KONDUCT /VX11-770-110, VX11-770-111, VX11-770-113). |
| Study timeframe | This study ran between February 2013 and April 2016 |
RESULTS
| What does this mean for me? | This study shows that Ivacaftor appears safe over 2 years in the 6 years and over age group. Effectiveness was maintained and was particularly clinically important in the group with non-G551D gating mutations (G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D). This information supports ongoing clinical use as per the existing approvals in the EU, USA, and Australia. |
|---|---|
| Study: | The publication did not list the sites and countries from which patients were enrolled. |
| Aim: | To assess the long term safety and efficacy of ivacaftor |
| Participants: | 65 participants were enrolled into the extension study KONTINUE. Of these participants, 30 had been treated with ivacaftor in the feeder study KONDUCT and 35 had been treated with placebo. |
| Treatment: | In KONTINUE, all patients were treated with ivacaftor 150 mg every 12 hours for up to 104 weeks. This summary presents results from the interim analysis of the extension study (KONTINUE), planned after 12 weeks of treatment. The information here concerns patients who joined the extension study from the feeder study KONDUCT /VX11-770-110. |
| Safety: | For the interim analysis of safety, the occurrence of serious adverse events was reported. Eight patients had SAEs, the majority of which were pulmonary exacerbations. |
| Efficacy: | After 12 weeks of treatment, percent predicted forced expiratory volume in 1 second (ppFEV1) increased from extension study start (baseline). The increase was 5% for patients who were in the placebo group in the feeder study (KONDUCT) and by 6% for patients who were in the ivacaftor group in the feeder study. The increases were statistically significant (p=0.0005 and p=0.006, respectively). Both adults and children had improved ppFEV1 after 12 weeks of ivacaftor. All patients showed improvement in the respiratory domain of the cystic fibrosis questionnaire revised (CFQ-R). |
| Data source: | The summary of data provided here is from a peer-reviewed publication, available here: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4641035/ |