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 (NCT01225211) enrolled 311 participants across four groups (called cohorts), testing different doses of two investigational drugs — lumacaftor (LUM) and ivacaftor (IVA) — alone and in combination. The trial was designed to look at how well the drugs were tolerated (by tracking unwanted medical events), and also to measure changes in two physical markers: sweat chloride levels (a marker used in cystic fibrosis monitoring) and a lung function test called percent predicted FEV1, which measures how much air a person can forcefully breathe out in one second. The reported data shows the following numbers. For adverse events (any unwanted medical occurrence during the study), the figures varied across groups: in Cohort 1, 12 placebo participants and 29 LUM-only participants reported adverse events during the first period, with 14–15 reporting events during the combination period. In Cohorts 2 and 3, between 7 and 37 participants per group reported adverse events depending on the dose and period. In Cohort 4, 53 of 63 placebo participants and 52 of 62 active-drug participants reported adverse events, with serious adverse events reported in 5 placebo and 9 active-drug participants. For sweat chloride, changes from the comparison point ranged from approximately +0.3 to −9.1 mmol/L across the different dose combinations, compared with small increases (around +0.5 to +1.6 mmol/L) in placebo groups. For the lung function measure in Cohort 4, the placebo group showed a change of −1.23 percent predicted FEV1, while the active drug group showed a change of −0.62 percent predicted FEV1 — both figures representing small decreases from baseline. 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 2 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 have a confirmed diagnosis of cystic fibrosis (CF)
- You carry a specific CF gene change called F508del on at least one copy of your CF gene
- Your lung function test result (called FEV1) meets the required level for the group (cohort) you would be joining — either at or above 40% of the expected normal value, or between 40–90% depending on the group (confirm with trial site)
- If you are able to become pregnant and are sexually active, you must be willing and able to use an approved method of contraception during the trial
Who may not be able to join:
- You have another health condition that, in the doctor's opinion, could interfere with the study results or make the study drug unsafe for you (for example, severe liver disease)
- You have had a recent illness, chest infection, flare-up of your CF symptoms, or a change in your CF medications (including antibiotics) within the last 14 or 28 days before starting the trial, depending on the group
- You have previously had an organ transplant (such as a lung, liver, or kidney) or a bone marrow/blood cell transplant
- You have had a problem with alcohol or drug misuse (including cannabis, cocaine, or opiates) in the past year
- You are currently taking part in another clinical drug trial, or recently took part in one without enough time having passed since it ended
- You are pregnant, breastfeeding, or able to become pregnant but unwilling or unable to use an approved non-hormonal contraception method
- You have already taken part in certain earlier groups (cohorts) of this trial, which may affect which group you can join (confirm with trial site)
- You have signs of cloudiness in the lens of your eye (cataracts), if you are being considered for the fourth group (Cohort 4)
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
6 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Cohort 1: Safety and Tolerability Based on Adverse Events (AEs); Cohort 2 and 3: Safety and Tolerability Based on Adverse Events (AEs); Cohort 4: Safety and Tolerability Assessed by Number of Participants With AEs and SAEs; Cohort 1: Absolute Change From Day 14 in Sweat Chloride at Day 21; Cohort 2 And 3: Absolute Change From Day 28 in Sweat Chloride at Day 56; Cohort 4: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 56
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.