What the trial was testing
The trial enrolled 87 patients with cystic fibrosis. The study was sponsored by Vertex Pharmaceuticals Incorporated and tracked outcomes across the full group of patients who matched the trial's eligibility profile.
It was initial testing (phase 2). 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
Lung function improved by up to 16 percentage points in some patients taking the new triple therapy.
The Lancet. Respiratory medicine · 2023 · NCT03912233
These findings — that people with certain gene types saw their breathing capacity increase substantially on the new once-daily pill — were published in the The Lancet. Respiratory medicine and represent the headline result of the study.
Researchers tracked outcomes across 87 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
This was an initial-stage study and the treatment is not yet available. The combination is being tested in larger trials compared to the standard treatment (elexacaftor-tezacaftor-ivacaftor). If you have cystic fibrosis, ask your doctor about approved triple therapies like Trikafta or whether you might qualify for ongoing studies of this new option.
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
Studying the Presence of CFRD Complications With Thoughtful Recruitment (SPeCTRuM)
This multicenter cross-sectional study will include a diverse population of adolescents and adults with CF. The overall Aim is to describe prevalence of diabetes microvascular complications and macrovascular surrogates in people with established CFRD.
A Phase II Study of Ensifentrine in Non-Cystic Fibrosis Bronchiectasis
This study is a randomized, double-blind, placebo-controlled study designed to assess the efficacy and safety of ensifentrine inhalation suspension (3 mg) delivered twice daily via standard jet nebulizer over at least 24 weeks, compared to placebo, in subjects with non-cystic fibrosis bronchiectasis (NCFBE).