Cystic fibrosis treatment has been transformed by CFTR modulators, particularly elexacaftor-tezacaftor-ivacaftor (Trikafta), which has dramatically improved outcomes for patients with at least one F508del mutation (about 90% of CF patients). Research now focuses on patients who don’t have CFTR mutations responsive to current modulators (about 10%), gene therapy approaches, and management of complications and comorbidities. For CF patients, clinical trials for cystic fibrosis offer access to investigational therapies. This guide covers how to find CF trials, active research areas, and resources.
Categories of CF trials
Active cystic fibrosis trials in 2026 include:
CFTR modulators for non-responsive mutations: Therapies for the ~10% of CF patients without F508del or with mutations not responsive to current modulators.
Gene therapy: AAV-based and other gene therapy approaches delivering functional CFTR.
mRNA therapies: Messenger RNA-based approaches to express functional CFTR protein.
Anti-inflammatory and infection-targeted therapies: Addressing the chronic infection and inflammation that drives CF lung disease.
Pseudomonas aeruginosa-targeted approaches: Novel antibiotic approaches for chronic CF infection.
Comorbidity management: CF-related diabetes, liver disease, and other complications.
Where to find CF trials
Resources:
- ClinicalTrials.gov
- Cystic Fibrosis Foundation Therapeutics Development Network
- Cystic Fibrosis Foundation
- Accredited CF care centers
Typical eligibility for CF trials
Common inclusion criteria:
- Confirmed CF diagnosis with documented CFTR mutations
- Specific mutation requirements (varies by trial)
- Lung function (FEV1) in specific range
- Stable medications for specified period
Common exclusion criteria:
- Recent lung transplant
- Active infection requiring intensive treatment
- Pregnancy
Frequently Asked Questions
Will I qualify for trials if I’m on Trikafta?
Many trials test add-on or alternative therapies in patients on Trikafta. Some specifically target the ~10% who don’t qualify for Trikafta.
Are gene therapy trials available?
Yes, in early-stage trials at major CF centers. Approaches continue to evolve.
Can I be in a trial after lung transplant?
Generally no for most CF-specific trials. Transplant-specific trials may apply.
Are pediatric CF trials available?
Yes. Pediatric CF research is active given the early onset of disease.
What’s the CF Therapeutics Development Network?
The CF Foundation’s research network of accredited CF centers conducting clinical research. Most major CF trials run through this network.
The bottom line on CF trials
CF research focuses on patients without F508del mutations, gene therapy, and managing the chronic complications. CF Foundation Therapeutics Development Network is the central resource. Discuss with your CF care team as part of comprehensive care.