What the trial was testing
The trial enrolled 124 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 13 percentage points in patients with certain cystic fibrosis gene mutations.
The New England journal of medicine · 2018 · NCT03224351
These findings — that patients with certain gene mutations saw major gains in breathing ability — were published in the The New England journal of medicine and represent the headline result of the study.
Researchers tracked outcomes across 124 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 testing study. The triple combination later became FDA-approved as Trikafta in 2019 and is now a standard treatment for people with cystic fibrosis who have at least one Phe508del gene mutation. If you have cystic fibrosis, ask your doctor if Trikafta or similar treatments are right for you.
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
IV Gallium Study for Patients With Cystic Fibrosis Who Have NTM (ABATE Study)
The purpose of this study is to assess the safety and tolerability of two 5-day infusion cycles of IV gallium in adult patients with CF who are infected with NTM. Funding Source - FDA Office of Orphan Products Development (OOPD)
Bionic Pancreas in CFRD
This multi-center randomized controlled trial (RCT) will compare efficacy and safety endpoints using the insulin-only configuration of the iLet Bionic Pancreas System (BP) versus a control group using their usual care insulin delivery method and continuous glucose monitoring (CGM) during a 13-week study period in individuals ≥14 years old with cystic fibrosis-related diabetes (CFRD). After 13 weeks, participants will continue in a 13-week Extension Phase in which the BP group will continue to use the BP system and the Usual Care group will initiate use of the BP system.