What the trial was testing
The ARRIVAL enrolled 57 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 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 by 73.5 mmol/L on average after 24 weeks of treatment.
The Lancet. Respiratory medicine · 2018 · NCT02725567
These findings — that a large reduction in sweat chloride shows the drug is working to improve CFTR function — 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
Ivacaftor is FDA-approved for young children with cystic fibrosis and certain gene mutations, including those as young as 12 months. If your child has cystic fibrosis, ask your doctor about genetic testing to see if ivacaftor is right for them. Starting treatment early may help protect the pancreas.
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).