| Clinical trials identifier | NCT02516410 |
|---|---|
| Study codes | VX14-661-107 |
| Study title | A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation and With a Second CFTR Mutation That Is Not Likely to Respond to VX-661 and/or Ivacaftor Therapy (F508del/NR) |
| Sponsor | Vertex |
| Drug (brand name) | VX-661 |
| Drug administration | Tablet taken by mouth |
| Eligible CF patients | Aged 12 Years and Older, Heterozygous for the F508del-CFTR Mutation and With a Second CFTR Mutation That Is Not Likely to Respond to VX-661 and/or Ivacaftor Therapy (F508del/NR) |
| Controlled study? | Yes (placebo controlled) |
| Randomised study? | Yes |
| Blinded study? | Yes (double blind - neither medical staff, nor participants knew which treatment participants were taking) |
| Extension study? | No |
| Study timeframe | The study was conducted between August 2015 and May 2016 |
RESULTS
| What does this mean for me? | Unfortunately, for people with one F508del mutation, and a second “minimal function” mutation – such as class 1 or “stop”/”mis-sense” mutations, the combination of VX-661 (Tezacaftor) with Ivacaftor did not show any evidence of clinical benefit, although it did appear safe. People with this combination of mutations causing cystic fibrosis are going to need a different approach or combination of CFTR modulator drugs, and work is ongoing. |
|---|---|
| Study: | This Phase 3 trial was conducted in 38 sites in 7 countries. |
| Aim: | To evaluate the efficacy of VX-661 in combination with ivacaftor |
| Participants: | In total, 168 patients were enrolled and randomly assigned to either the treatment group or the placebo group. There were 30 participants from 4 ECFS-CTN sites in France and Israel. |
| Treatment: | In the treatment group, patients received VX-661 100 mg plus ivacaftor 150 mg in the morning and ivacaftor 150 mg in the evening every day for 12 weeks. In the placebo group, patients received placebo plus ivacaftor 150 mg in the morning and ivacaftor 150 mg in the evening every day for 12 weeks. |
| Safety: | Around three quarters of each treatment group had non-serious adverse events, the most common was Infective pulmonary exacerbation of cystic fibrosis. Serious adverse events were experienced by 16.47% of patients in the placebo group and 13.25% of patients in the VX-661+ivacaftor treatment group. The most common serious adverse event was Infective pulmonary exacerbation of cystic fibrosis (14.12% of patients in the placebo group and 7.23% of patients in the VX-661+ivacaftor treatment group |
| Efficacy: | After 12 weeks of treatment, the VX-661+ivacaftor combination was not superior to placebo+ivacaftor in improving percent predicted forced expiratory volume in 1 second (ppFEV1). |
| Data source: | The summary of data provided here is from a registry posting, available here: https://www.clinicaltrialsregister.eu/ctr-search/trial/2014-004787-37/results |