Cystic Fibrosis (CF) Clinical Trials and Treatments

Cystic fibrosis (CF) is an autosomal recessive genetic disorder caused by mutations in the CFTR (cystic fibrosis transmembrane conductance regulator) gene. CFTR encodes a chloride channel protein essential for regulating ion transport across epithelial cells. When CFTR is absent or dysfunctional, chloride transport is impaired, causing thick, sticky secretions in the lungs, pancreas, intestines, and other organs.

The most common symptoms of Cystic Fibrosis include Persistent cough with sputum production, Recurrent respiratory infections, Wheezing and shortness of breath, Failure to thrive and malabsorption, Fatty, foul-smelling stools (steatorrhea), among others. Nearly 40,000 children and adults in the U.S.; 1 in 2,500 to 3,500 births among Caucasians. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Cystic Fibrosis from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. CFTR modulators have already transformed cystic fibrosis care for many patients, extending projected lifespans dramatically, and the next wave of research is pushing even further. A once-daily modulator combination, vanzacaftor, tezacaftor and deutivacaftor (Alyftrek), was FDA approved in December 2024 for people 6 and older with at least one F508del mutation or another responsive CFTR mutation.

Yes. As of October 21, 2026, 10 FDA-approved medicines have a current label that names Cystic Fibrosis: Alyftrek (vanzacaftor / tezacaftor / deutivacaftor), first approved in 2024; Kalydeco (ivacaftor), first approved in 2012; Orkambi (lumacaftor / ivacaftor), first approved in 2015; Symdeko (tezacaftor / ivacaftor), first approved in 2018; Trikafta (elexacaftor / tezacaftor / ivacaftor), first approved in 2019; Acetylcysteine inhalation solution (acetylcysteine (inhalation)); Bronchitol (mannitol (inhalation powder)); Cayston (aztreonam (inhalation)); Pulmozyme (dornase alfa); and Tobi (tobramycin (inhalation)). Corticosteroids and immune globulins count once each.

Cystic Fibrosis is caused by variants in the CFTR gene. Biallelic CFTR variants cause CF; the specific variants determine eligibility for CFTR modulator drugs.

It is inherited in an autosomal recessive pattern: both parents are usually unaffected carriers, and each pregnancy has a 1 in 4 chance of an affected child. Nearly 40,000 children and adults in the U.S.; 1 in 2,500 to 3,500 births among Caucasians. Genetic testing confirms the diagnosis.

Yes. Genetic testing for Cystic Fibrosis looks at the CFTR gene. Biallelic CFTR variants cause CF; the specific variants determine eligibility for CFTR modulator drugs.

It is inherited in an autosomal recessive pattern: both parents are usually unaffected carriers, and each pregnancy has a 1 in 4 chance of an affected child.

A confirmed variant establishes the diagnosis and is often required to enroll in gene-specific trials. The labs currently offering the test are listed in the Genetic Testing section of this page, pulled live from the NIH Genetic Testing Registry. A genetic counselor can help interpret the result and decide which relatives should be tested.

Eligibility for Cystic Fibrosis clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Cystic Fibrosis clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.