Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial (NCT01931839) enrolled 1,163 participants in total across seven groups. It was split into two parts — Part A and Part B — and tested a combination of two investigational medicines, lumacaftor (LUM) and ivacaftor (IVA), given at different doses, against a placebo (a dummy treatment with no active ingredient). A small observational group of 19 people was also included. The trial was measuring, as its primary focus, how many participants experienced adverse events (unwanted medical occurrences during the study) and serious adverse events (more severe events such as hospitalisation or life-threatening situations) while taking the treatments. A key secondary focus was tracking changes in lung function, measured by a breathing test called FEV1 — essentially how much air a person can forcefully breathe out in one second. The reported data shows that in Part A, adverse events of any kind were recorded in 331 out of 335 participants in the LUM 600 mg once-daily/IVA group, 177 out of 178 in its placebo group, 333 out of 340 in the LUM 400 mg twice-daily/IVA group, and 176 out of 176 in its placebo group. Serious adverse events were recorded in 156, 77, 143, and 89 participants in those same groups respectively. In Part B, adverse events were recorded in 52 out of 55 participants in the active treatment group and 57 out of 60 in the placebo group, with serious adverse events in 18 and 21 participants respectively. For the lung function measure in Part A, the reported data shows small increases from baseline across all groups at the time points provided — for example, absolute changes at one measured point ranged from roughly +2.5 to +3.4 percentage points across the active and placebo groups. In Part B, the reported data shows small negative changes from baseline (slight decreases) at all measured time points across both the active and placebo groups, ranging from around −2 to −5.4 percentage points in the active group and −1.8 to −3.4 percentage points in the placebo group. The data for several of the later time points (weeks 36 through 72) was not reported in the submitted results. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 37983082) ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 3 Cystic Fibrosis Trial, Completed
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You must have signed a consent form agreeing to take part (and a separate assent form if applicable).
- To join the Part A Treatment Group: You must have completed 24 weeks of treatment in a related study called Study 103 or Study 104, and be willing to continue into this trial.
- To join the Part B Treatment Group: You must have completed 56 days of treatment in a specific group (Cohort 4) of a related study called Study 102, and be willing to continue into this trial.
- To join the Part A Observation-Only Group: You must have completed 24 weeks of treatment in Study 103 or Study 104, but either choose not to join the Part A Treatment Group or do not qualify for it.
- If joining the Part A or Part B Treatment Groups, you must be willing to keep your current cystic fibrosis medications unchanged throughout the study.
Who may not be able to join:
- People with other serious health conditions or abnormal lab results that the study doctor believes could affect the results or make the study medication unsafe for you (confirm with trial site).
- People who are pregnant or breastfeeding; women who could become pregnant must have a negative pregnancy test before starting.
- People who had a bad reaction or intolerance to the study medication in the previous related study, if the study doctor believes this would put you at further risk.
- People who did not reliably follow the medication schedule or study procedures in the previous related study, as judged by the study doctor.
- People who are currently taking part in another clinical trial involving investigational drugs, including studies of lumacaftor and/or ivacaftor, or any study requiring blood draws.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
8 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Part A Treatment Cohort: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs); Part B Treatment Cohort: Number of Participants With Treatment-Emergent AEs and SAEs
Can't join this trial?
Data last synced from ClinicalTrials.gov: 22 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.