What the trial was testing
The trial enrolled 57 patients with cystic fibrosis. The study was sponsored by Vertex Pharmaceuticals and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was a large trial designed to confirm whether the treatment works well enough for wider use. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.
What the results showed
Sweat chloride dropped 29.6 mmol/L on lumacaftor-ivacaftor and stayed lower.
The Lancet Respiratory Medicine · 2021 · NCT03125395
These findings — that in sweat chloride sustained over 96 weeks on lumacaftor-ivacaftor in young children — were published in the The Lancet Respiratory Medicine and represent the headline result of the study.
Researchers tracked outcomes across 57 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.
What this means for patients
For patients with cystic fibrosis, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.
What you can do now
Lumacaftor-ivacaftor (Orkambi) is FDA-approved and available now for children 1 and older with two F508del mutations. However, most CF clinics now use the newer triple combo (Trikafta) when eligible since it works better. Ask your CF team which CFTR modulator is best for your child's genotype and age.
Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.
Open cystic fibrosis trials
Xenon-129 and Inert Fluorinated Gas Lung MRI: Study of Healthy Volunteers and Participants With Pulmonary Disease
Aim of this study is to evaluate image quality and reproducibility of Xenon-129 and Inert fluorinated (19F) gas Magnetic Resonance Imaging (MRI) and to evaluate changes in lung structure and function in participants with cystic fibrosis (CF) and asthma compared to healthy controls.
Restarting Triple Therapy With Robust Monitoring for Adverse Events (RETRIAL)
RETRIAL is a multi-site observational study of people with Cystic Fibrosis (PWCF) ages 6 and up starting the new triple-therapy modulator (vanzacaftor/tezacaftor/deutivacaftor (VTD)), after having experienced neuropsychiatric events and/or liver injury while taking elexacaftor/tezacaftor/ivacaftor (ETI) that resulted in a modification or discontinuation of standard ETI dosing.