Based on: Cystic Fibrosis — gene targets, candidate compounds and trials, OSMF Research Tracker (DGIdb, Open Targets, ChEMBL, PubMed and ClinicalTrials.gov data). Numbers are a snapshot taken 8 October 2026; the tracker page is the live version.
Short answer: as of October 2026, the OSMF Research Tracker lists 56 candidate interventions for cystic Fibrosis that are either approved medicines or have at least one registered clinical trial, built on 11 druggable gene targets, with 14 recent trials on ClinicalTrials.gov. 4 are already approved medicines, for this condition or another one; where the approval is for a different condition, the trial is testing a new use, which is what drug repurposing means. Being in a trial says an intervention is being studied, not that it works; that is what the trial is for.
Which candidates have the most trial activity?
Ranked by the number of registered trials the tracker found. Each trial ID links to the registry entry, where you can read the design, status and sponsor.
- mannitol — 4 registered trials · approved medicine · NCT00251056
- IVA — 3 registered trials · investigational · NCT05331183 NCT02951195 NCT04537793
- Ivacaftor — 2 registered trials · approved medicine · NCT01705145 NCT03783286
- ELX/TEZ/IVA — 2 registered trials · investigational · NCT05331183 NCT04537793
- EUR-1008 (APT-1008) — 2 registered trials · investigational · NCT00297167
- FDL169 — 2 registered trials · investigational · NCT03424252 NCT02359357
- Hyperpolarized Xe129 — 2 registered trials · investigational · NCT06339593 NCT04624490
- Saline — 2 registered trials · investigational · NCT03078088 NCT00749866
- SB656933 — 2 registered trials · investigational · NCT00903201
- spirometry — 2 registered trials · investigational · NCT03839992 NCT05639556
- interferon gamma-1b — 1 registered trial · approved medicine · NCT00043316
- tobramycin — 1 registered trial · approved medicine · NCT00004829
Which gene targets are these built on?
The tracker starts from genes with evidence of being druggable in cystic Fibrosis and asks which compounds act on them. Each symbol links to its DGIdb entry, which lists the interacting drugs and the papers behind them: ANO1, CFTR, CTSC, CXCR2, DNASE1, ELANE, MUC5B, SCNN1A, SCNN1B, SCNN1G, SLC26A9.
Which trials are registered?
- Role of Genetic Factors in the Development of Lung Disease NCT00001532 · Recruiting · started 1996-09-13
- Comparing Expectorated and Induced Sputum and Pharyngeal Swabs for Cultures, AFB Smears, and Cytokines in Pulmonary Nontuberculous Mycobacterial Infection NCT01150721 · Completed · started 2010-11-10
- Bacteriophages for Adults With Cystic Fibrosis and Chronic Achromobacter Lung Infection NCT07275905 · Recruiting · Phase PHASE1 · started 2026-08-20
- Regional Phenotyping of CF and Non-CF Bronchiectasis NCT04793867 · Recruiting · started 2021-02-08
- Health Outcomes of Parents With Cystic Fibrosis-Aim 2 NCT06296394 · Recruiting · started 2024-05-29
- Hyperpolarized Imaging for New Treatments NCT04259970 · Completed · Phase PHASE4 · started 2020-01-13
- A Proof-of-Concept Study to Assess the Efficacy, Safety and Tolerability of Itepekimab (Anti-IL-33 mAb) in Participants With Non-cystic Fibrosis Bronchiectasis NCT06280391 · Completed · Phase PHASE2 · started 2024-02-20
- Phase 2 Study to Assess the Safety and Efficacy of ANG003 NCT07450547 · Recruiting · Phase PHASE2 · started 2026-04-24
- Development of Assist Tool for Breast Examination Using the Principle of Ultrasonic Sensor NCT06255808 · Completed · started 2022-10-05
- Acoustic Waveform Respiratory Evaluation NCT06512064 · Recruiting · Phase NA · started 2024-07-23
How to read this
Evidence is graded on the tracker page in three tiers: clinical (tested in people), mechanistic (a documented action on the target) and correlative (an association in the literature). Most candidates sit in the lower tiers, and many trials are small or early-phase. The purpose of this list is to show where the research is, with sources, so that patients, clinicians and researchers can look further; it is not a ranking of what to take.
Related on this site
Research information, not medical advice. Decisions about any medicine belong with you and your clinician. The underlying data are open (CC BY 4.0) and refreshed daily; this note was generated from the tracker page on 8 October 2026.