Disease · MONDO_0009061
cystic fibrosis
6 sourcesdiseases-2026-09-18-0.1.0
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ConductScience Library. cystic fibrosis. Diseases, record MONDO_0009061. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0009061
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Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Other names
CF, CF - Cystic fibrosis, CYSTIC FIBROS W/O ILEUS, Cystic fibrosis (disorder), Cystic fibrosis NOS, Cystic fibrosis NOS (disorder), Cystic fibrosis without mention of meconium ileus, Fibrocystic disease, Meconium ileus in cystic fibrosis (disorder), Meconium obstruction of intestine in mucoviscidosis, cystic fibrosis lung disease, modifier of, cystic fibrosis with combined manifestations, cystic fibrosis with gastrointestinal manifestations, cystic fibrosis with meconium ileus, cystic fibrosis with meconium ileus (disorder), cystic fibrosis with other manifestations, cystic fibrosis with other manifestations (disorder), cystic fibrosis with pulmonary manifestations, cystic fibrosis with pulmonary manifestations (disorder), mucoviscidosis, pseudomonas aeruginosa, susceptibility to chronic infection by, in cystic fibrosis
- Open Targets
- Highest stage of its drugs
- Approval, for 17 of its 148 drugs, from ClinicalTrials.gov, DailyMed, EMA Human Drugs, FDA, PMDA and TTD
- Open Targets
Drugs148
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| IVACAFTOR | CHEMBL2010601 | Small molecule | Approval | ClinicalTrials.gov, DailyMed, EMA Human Drugs, FDA and TTD | 132 | 121 | 121 | Drugs and targets |
| TEZACAFTOR | CHEMBL3544914 | Small molecule | Approval | ClinicalTrials.gov, EMA Human Drugs and FDA | 76 | 71 | 71 | Drugs and targets |
| ELEXACAFTOR | CHEMBL4298128 | Small molecule | Approval | ClinicalTrials.gov and EMA Human Drugs | 42 | 40 | 40 | Drugs and targets |
| TOBRAMYCIN | CHEMBL1747 | Small molecule | Approval | DailyMed, EMA Human Drugs and PMDA | 47 | 36 | 36 | Drugs and targets |
| LUMACAFTOR | CHEMBL2103870 | Small molecule | Approval | DailyMed, EMA Human Drugs and TTD | 34 | 30 | 30 | Drugs and targets |
| DORNASE ALFA | CHEMBL1201431 | Enzyme | Approval | DailyMed, PMDA and TTD | 17 | 12 | 12 | Drugs and targets |
| AZTREONAM | CHEMBL158 | Small molecule | Approval | DailyMed and EMA Human Drugs | 12 | 10 | 10 | Drugs and targets |
| DEUTIVACAFTOR | CHEMBL4297603 | Small molecule | Approval | EMA Human Drugs | 12 | 10 | 10 | Drugs and targets |
| MANNITOL | CHEMBL689 | Small molecule | Approval | DailyMed and EMA Human Drugs | 11 | 9 | 9 | Drugs and targets |
| VANZACAFTOR | CHEMBL5314934 | Small molecule | Approval | EMA Human Drugs | 10 | 8 | 8 | Drugs and targets |
1 to 10 of 148
See all 148 drugsTargets154
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| CFTR | Cystic fibrosis transmembrane conductance regulator | 11 | Cystic fibrosis transmembrane conductance regulator activator; Cystic fibrosis transmembrane conductance regulator positive modulator; Cystic fibrosis transmembrane conductance regulator stabiliser | Approval | https://conductscience.org/library/genes/1080 | Drugs and targets |
| INSR | Insulin receptor | 3 | Insulin receptor agonist | Phase 3 | https://conductscience.org/library/genes/3643 | Drugs and targets |
| SCNN1A | Amiloride-sensitive sodium channel, ENaC | 3 | Amiloride-sensitive sodium channel, ENaC blocker; Amiloride-sensitive sodium channel, ENaC mRNA antisense inhibitor | Phase 2 | https://conductscience.org/library/genes/6337 | Drugs and targets |
| SCNN1B | Amiloride-sensitive sodium channel, ENaC | 3 | Amiloride-sensitive sodium channel, ENaC blocker; Amiloride-sensitive sodium channel, ENaC mRNA antisense inhibitor | Phase 2 | https://conductscience.org/library/genes/6338 | Drugs and targets |
| SCNN1G | Amiloride-sensitive sodium channel, ENaC | 3 | Amiloride-sensitive sodium channel, ENaC blocker; Amiloride-sensitive sodium channel, ENaC mRNA antisense inhibitor | Phase 2 | https://conductscience.org/library/genes/6340 | Drugs and targets |
| ADH5 | Alcohol dehydrogenase class-3 | 2 | Alcohol dehydrogenase class III inhibitor | Phase 2 | https://conductscience.org/library/genes/128 | Drugs and targets |
| ADRB2 | Beta-2 adrenergic receptor | 2 | Beta-2 adrenergic receptor agonist | Unknown | https://conductscience.org/library/genes/154 | Drugs and targets |
| CHRM3 | Muscarinic acetylcholine receptor M3 | 2 | Muscarinic acetylcholine receptor M3 antagonist | Phase 3 | https://conductscience.org/library/genes/1131 | Drugs and targets |
| ELANE | Neutrophil elastase | 2 | Leukocyte elastase inhibitor | Phase 2 | https://conductscience.org/library/genes/1991 | Drugs and targets |
| ESR1 | Estrogen receptor | 2 | Estrogen receptor alpha agonist; Estrogen receptor alpha modulator | Phase 2 | https://conductscience.org/library/genes/2099 | Drugs and targets |
1 to 10 of 154
See all 154 targetsTrials395
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 7 more are not shown, since the checks do not confirm them.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT01931839 | A Phase 3 Rollover Study of Lumacaftor in Combination With Ivacaftor in Subjects 12 Years and Older With Cystic Fibrosis | Phase 3 | 1,164 | 2017-05-12 | Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation | IVACAFTOR, LUMACAFTOR | Its record names the disease | ClinicalTrials.gov |
| NCT02565914 | A Study to Evaluate the Safety and Efficacy of Long Term Treatment With VX-661 in Combination With Ivacaftor in Participants With Cystic Fibrosis Who Have an F508del-CFTR Mutation | Phase 3 | 1,131 | 2020-06-16 | Cystic Fibrosis | IVACAFTOR, TEZACAFTOR | Its record names the disease | ClinicalTrials.gov |
| NCT05076149 | A Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Who Are Homozygous for F508del, Heterozygous for F508del and a Gating (F/G) or Residual Function (F/RF) Mutation, or Have At Least 1 Other Triple Combination Responsive (TCR) CFTR Mutation and No F508del Mutation | Phase 3 | 597 | 2024-06-13 | Cystic Fibrosis | DEUTIVACAFTOR, ELEXACAFTOR, IVACAFTOR, TEZACAFTOR, VANZACAFTOR | Its record names the disease | ClinicalTrials.gov |
| NCT01807949 | A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation | Phase 3 | 563 | 2015-09-01 | Cystic Fibrosis, Homozygous for the F508del CFTR Mutation | IVACAFTOR, LUMACAFTOR | Its record names the disease | ClinicalTrials.gov |
| NCT01807923 | A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation | Phase 3 | 559 | 2015-08-31 | Cystic Fibrosis, Homozygous for the F508del CFTR Mutation | IVACAFTOR, LUMACAFTOR | Its record names the disease | ClinicalTrials.gov |
| NCT00388505 | Safety of Tobramycin Inhalation Powder (TIP) vs Tobramycin Solution for Inhalation in Patients With Cystic Fibrosis | Phase 3 | 517 | 2012-07-24 | Cystic Fibrosis | TOBRAMYCIN | Its record names the disease | ClinicalTrials.gov |
| NCT00737100 | Safety and Efficacy of 12-wk Treatment With Two Doses of Tiotropium Respimat in Cystic Fibrosis | Phase 2 | 510 | 2011-04-13 | Cystic Fibrosis | TIOTROPIUM BROMIDE | Its record names the disease | ClinicalTrials.gov |
| NCT02347657 | A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor | Phase 3 | 510 | 2018-06-12 | Cystic Fibrosis | IVACAFTOR, TEZACAFTOR | Its record names the disease | ClinicalTrials.gov |
| NCT03525574 | A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy | Phase 3 | 507 | 2024-05-08 | Cystic Fibrosis | ELEXACAFTOR, IVACAFTOR, TEZACAFTOR | Its record names the disease | ClinicalTrials.gov |
| NCT03447262 | A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy | Phase 3 | 484 | 2022-01-25 | Cystic Fibrosis | BAMOCAFTOR, IVACAFTOR, TEZACAFTOR | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 395
See all 395 trialsComparisons in its trials165
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| ELX/TEZ/IVA vs VX-121/TEZ/D-IVA: deaths from any cause | 0 of 289 | 0 of 284 | 573 | NCT05076149 | Trial comparisons |
| ELX/TEZ/IVA vs VX-121/TEZ/D-IVA: serious adverse events | 40 of 289 | 40 of 284 | 573 | NCT05076149 | Trial comparisons |
| Part A: ELX/TEZ/IVA vs Part B: ELX/TEZ/IVA: deaths from any cause | 0 of 457 | 0 of 66 | 523 | NCT04043806 | Trial comparisons |
| Part A: ELX/TEZ/IVA vs Part B: ELX/TEZ/IVA: serious adverse events | 75 of 457 | 4 of 66 | 523 | NCT04043806 | Trial comparisons |
| Tobramycin Inhalation Powder (TIP) vs Tobramycin Solution for Inhalation (TOBI): serious adverse events | 85 of 308 | 61 of 209 | 517 | NCT00388505 | Trial comparisons |
| Treatment Period: ELX/TEZ/IVA vs Extension Period: ELX/TEZ/IVA: deaths from any cause | 1 of 506 | 0 of 11 | 517 | NCT03525574 | Trial comparisons |
| Treatment Period: ELX/TEZ/IVA vs Extension Period: ELX/TEZ/IVA: serious adverse events | 175 of 506 | 0 of 11 | 517 | NCT03525574 | Trial comparisons |
| VX-661/IVA vs Placebo: deaths from any cause | 0 of 251 | 0 of 258 | 509 | NCT02347657 | Trial comparisons |
| VX-661/IVA vs Placebo: serious adverse events | 31 of 251 | 47 of 258 | 509 | NCT02347657 | Trial comparisons |
| VX-661/IVA vs Placebo: Number of Participants With at Least One Pulmonary Exacerbation Pulmonary Exacerbation Through Week 24 | 62 of 248 | 88 of 256 | 504 | NCT02347657 | Trial comparisons |
1 to 10 of 165
See all 165 comparisons in its trialsPapers from its trials197
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0009061
GET https://conductscience.org/api/diseases/v1/MONDO_0009061/drugs?page=2