Respiratory

Cystic Fibrosis

Also known as CF, CFTR mutation, mucoviscidosis

Cystic fibrosis is caused by mutations in the CFTR gene encoding a chloride channel, leading to thick, sticky mucus in the lungs, digestive tract, and other organs. The F508del mutation accounts for ~70% of alleles.

ORPHA:586 ↗Gene SERPINA1Gene SLC26A9Gene SLC6A14Gene SLC9A3Gene CEACAM3Gene CEACAM6Prevalence 1-5 per 10,000 (Orphanet)Onset All agesGenetic (autosomal recessive)

148

studies recruiting now

as of 7 Sept 2026

1,767

studies registered in total

as of 7 Sept 2026

2

countries with a recruiting site

as of 7 Sept 2026

4 Sept 2026

most recent study posted

among recruiting studies

Recruiting trials

Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.

See all 148 recruiting studies

Where recruiting studies are running

Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.

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Support

Patient organisations

Cystic Fibrosis FoundationPatient association
Visit website ↗

Registry: CF Foundation Patient Registry · Join ↗. Registries connect patients to researchers and often hear about trials first.

About Cystic Fibrosis

Cystic fibrosis is caused by mutations in the CFTR gene encoding a chloride channel, leading to thick, sticky mucus in the lungs, digestive tract, and other organs. The F508del mutation accounts for ~70% of alleles. CFTR modulators (ivacaftor, lumacaftor, tezacaftor, and the triple combination Trikafta/Kaftrio) are now approved for ~90% of people with CF, dramatically improving outcomes. Trials focus on remaining patients, lung transplant, and next-generation modulators.

Common clinical features

Elevated sweat chlorideAirway obstructionRecurrent respiratory infectionsBronchiectasisExocrine pancreatic insufficiencyMalabsorptionMale infertilityFailure to thrive

From Orphanet’s phenotype annotations (CC BY 4.0). Not a complete list.

Treatments being studied

18 approved treatments and 103 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Approved: Phytonadione (Aquamephyton)Approved: Aztreonam (Azactam)Approved: Colistimethate Sodium (Colistimethate sodium)Approved: Vanzacaftor (Vanzacaftor component of alyftrek)Approved: Dornase Alfa (Pulmozyme)Approved: Aztreonam Lysine (Cayston)Approved: Azithromycin (Azasite)Approved: Ivacaftor (Ivacaftor component of orkambi)Approved: Levofloxacin (Evoxil)Approved: Elexacaftor (Elexacaftor component of trikafta)Approved: Tobramycin-TobramycinApproved: Tobramycin (Aktob)Approved: Lumacaftor (Lumacaftor component of orkambi)Approved: Acetylcysteine (A-cys)Approved: Mannitol (Aridol kit)Approved: Deutivacaftor (Deutivacaftor component of alyftrek)Approved: Tezacaftor (Tezacaftor component of symkevi)Approved: Interferon Gamma-1b (Actimmune)
Phase 3Insulin Glargine (Abasaglar)
Phase 3Sitagliptin (Sitagliptin component of zituvimet)
Phase 3Liprotamase (Theraclec)
Phase 3Insulin Aspart (Fiasp)
Phase 3Colistin (Sogecoli)
Phase 3Insulin Detemir (Ins levemir)
Phase 3Repaglinide (Enyglid)
Phase 3Cephalexin (Cephalexin)

+ 95 more in development

Before you apply

Things trial teams commonly ask about for Cystic Fibrosis. Not eligibility rules; those are set by each study.

  • CFTR mutation class (F508del homozygous, F508del heterozygous, or rare mutation) determines modulator eligibility and trial access
  • ppFEV1 (percent predicted FEV1) is the primary lung function measure used in eligibility
  • Pseudomonas aeruginosa infection status and exacerbation history are standard screening elements

Related conditions

Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).