What the trial was testing
The WILLOW enrolled 256 patients with cystic fibrosis. The study was sponsored by Insmed 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
Patients taking brensocatib went 134 days before a flare-up, compared to 67 days on placebo.
The New England journal of medicine · 2020 · NCT03218917
These findings — that patients on the 10 mg pill had 42% fewer lung flare-ups than those on placebo — were published in the The New England journal of medicine and represent the headline result of the study.
Researchers tracked outcomes across 256 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 a mid-stage study and brensocatib is not yet FDA-approved for bronchiectasis. The trial showed promise in reducing flare-ups, but some patients had dental and skin side effects. Ask your doctor about open trials or approved treatments for bronchiectasis.
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
Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs
The purpose of this study is to look at how Alpha-1-antitrypsin (AAT) deficiency and Cystic Fibrosis (CF) affect white blood cells in the lungs, called macrophages, and their ability to work.
Dose Range Finding, Efficacy, and Safety Study of Nebulized CSL787 in Adults With Non-cystic Fibrosis Bronchiectasis (NCFB)
This study is a phase 2b, multicenter, randomized, double-blind, placebo-controlled, parallel-group, dose range finding study designed to explore the efficacy, safety, and tolerability of 2 active treatment regimens of CSL787 (immunoglobulin G \[IgG\] inhalation solution) compared with placebo over a period of 6 to 12 months independent of the occurrence of pulmonary exacerbations. The primary aim of the study is to characterize the overall effect of CSL787 as well as the dose response of 2 active treatment regimens of inhaled CSL787 administered to participants with NCFB toward prolonging the TTF exacerbation.