Cystic Fibrosis — RepurpOS | OpenSourceMedicine

Disease overview

Alterations

176

Molecular (A)

100

Clinical (B–E)

Direct drugs

154

Via biomarker

180

Natural agents

Natural products

Merged ranked

332

With trial/literature evidence

Sources: Open Targets, HPO, ClinicalTrials.gov, DGIdb, ChEMBL, PubMed Literature, Examine.com, PubMed NP Literature · Pipeline phase 7 · Updated 2026-07-06

Remission & chronicity

Spontaneous remission

Progressive lung function decline; no spontaneous remission; historically median survival 30 years; now >50 years with CFTR modulators for eligible genotypes

Best-intervention remission

Elexacaftor-tezacaftor-ivacaftor (ETI, Trikafta): FEV1 improvement 14-15 percentage points, sweat chloride normalisation in F508del homozygous patients (AURORA trial); 80-90% of CF patients in US have eligible genotypes; 'functional cure' in many patients — near-normal lung function in those starting young

Drug-free remission

—

Relapse after remission

—

Chronicity

—

Gap size

Near-zero biological gap for CFTR-modulator-eligible patients (10-15% have non-eligible mutations); very large access gap globally

Primary barrier

access

Barrier detail: Trikafta is among the most expensive drugs ever approved (~$330K/year); most middle-income countries have no access; 10-15% of CF patients (class I nonsense mutations) are not eligible for CFTR modulators and remain without disease-modifying therapy; expanded mRNA/gene therapy approach needed for ineligible patients

Last SoC change: 2019 — Trikafta (elexacaftor-tezacaftor-ivacaftor) FDA approved — arguably the most transformative disease-modifying approval in CF history; 2015 lumacaftor-ivacaftor; 2012 ivacaftor (G551D only); lung transplantation as sole 'curative' option for decades; DNase, tobramycin inhaled, hypertonic saline as symptomatic standard since 1990s-2000s

Remission data layers: disease_db_100 · source-locked manual data · confidence: high

Identifiers: MONDO: MONDO:0009061 · ORPHA: ORPHA:2575

Right to Try Relevance Critical

Eligibility: This disease meets the federal and state right-to-try legal standard (21 U.S.C. § 360bbb-0a and Montana SB 535): it lacks disease-modifying therapy with indication-specific FDA approval. Patients with Cystic Fibrosis may be eligible to access investigational therapies outside of clinical trials under right-to-try frameworks.

Investigational Agents

Phase 3: 25, Phase 2: 15

Most Advanced Phase

Phase 3

Highest dev. stage

Gene Targets

176

Actionable

Pipeline Compounds (Top 6)

Amikacin, Ataluren, Bamocaftor, Cephalexin, Colistin, Dexamethasone

Legal Standard: Right-to-try requires the disease lacks "adequate approved therapy for that specific disease" (not symptom-management drugs). This distinction means a disease with antidepressants available still qualifies if no FDA approval exists for THAT disease indication.

View Right-to-Try Compendium

Alterations

Showing 80 of 176 · Types A (molecular) through E (functional)

Name	Type	Subtype	Direction	Frequency	Evidence	Sources	Links
Abnormal erythrocyte morphology	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Abnormality of the liver	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Absent vas deferens	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Airway obstruction	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Anxiety	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Asthma	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Biliary cirrhosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Bronchiectasis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Chronic lung disease	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Chronic sinusitis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cirrhosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Clubbing of fingers	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Cor pulmonale	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Decreased body mass index	Scales & PROs	Clinical scale	—	—	Moderate	HPO, Monarch	HPO
Decreased forced expiratory flow 25-75%	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Deeply set eye	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Dehydration	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Depression	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Diarrhea	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Elevated circulating hepatic transaminase concentration	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Elevated sweat chloride	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Exocrine pancreatic insufficiency	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Failure to thrive	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Folate-unresponsive megaloblastic anemia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Frontal bossing	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Gastritis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Gastroesophageal reflux	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Glanular hypospadias	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hearing impairment	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hemoptysis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hepatomegaly	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hepatosplenomegaly	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hypercalciuria	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hyperpolarized transepithelial nasal potential difference	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hypertelorism	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Hypospadias	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Ileus	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Intellectual disability	Functional	Functional test	—	—	Moderate	HPO, Monarch	HPO
Macrotia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Malabsorption	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Male infertility	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Meconium ileus	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Megaloblastic anemia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Micrognathia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Nasal polyposis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Nephrolithiasis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Nephrotic syndrome	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Nontuberculous mycobacterial pulmonary infection	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Osteopenia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Osteoporosis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Pancreatitis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Pneumothorax	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Rectal prolapse	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Recurrent Aspergillus infections	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Recurrent Burkholderia cepacia infections	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Recurrent Haemophilus influenzae infections	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Recurrent Staphylococcus aureus infections	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Recurrent bronchopulmonary infections	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Recurrent lower respiratory tract infections	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Recurrent pneumonia	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Recurrent respiratory infections	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Reduced forced expiratory volume in one second	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Reduced forced vital capacity	Lab / Clinical	Lab value	—	—	Moderate	HPO, Monarch	HPO
Salty tasting skin	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Shawl scrotum	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Sinusitis	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Steatorrhea	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Telecanthus	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
Wide nasal bridge	Pathology	Pathological finding	—	—	Moderate	HPO, Monarch	HPO
CFTR CF transmembrane conductance regulator	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Change in Absolute FEV1 From Baseline Over 26 Weeks	Scales & PROs	Clinical scale	—	—	Emerging	ClinicalTrials.gov	—
DERL1 derlin 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DERL2 derlin 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
DERL3 derlin 3	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ERLEC1 endoplasmic reticulum lectin 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ERLIN1 ER lipid raft associated 1	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
ERLIN2 ER lipid raft associated 2	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
Exercise capacity	Functional	Functional test	—	—	Emerging	ClinicalTrials.gov	—
FAU FAU ubiquitin like and ribosomal protein S30 fusion	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets
HFE homeostatic iron regulator	Molecular	Gene	—	—	Emerging	Open Targets	UniProt Ensembl Open Targets

FDA-Approved Therapies

Phase 4 agents with regulatory approval for this indication

Drug Name	Mechanism	Type
ACETAZOLAMIDE	None	Gene-targeted
ADALIMUMAB		Gene-targeted
ALCLOMETASONE		Gene-targeted
ALCLOMETASONE DIPROPIONATE	agonist	Gene-targeted
AMCINONIDE	agonist	Gene-targeted
AMPHETAMINE	None	Gene-targeted
APIGENIN	activator	Gene-targeted
ASPIRIN		Gene-targeted
ATALUREN	modulator	Gene-targeted
AZACITIDINE		Gene-targeted
BECLOMETHASONE		Gene-targeted
BECLOMETHASONE DIPROPIONATE	agonist; agonist	Gene-targeted
BETAMETHASONE	agonist; agonist	Gene-targeted
BETAMETHASONE ACETATE	agonist	Gene-targeted
BETAMETHASONE BENZOATE	agonist	Gene-targeted
BETAMETHASONE DIPROPIONATE	agonist	Gene-targeted
BETAMETHASONE SODIUM PHOSPHATE	agonist	Gene-targeted
BETAMETHASONE VALERATE	agonist	Gene-targeted
BICALUTAMIDE	None	Gene-targeted
BUDESONIDE	agonist; agonist	Gene-targeted
CAPSAICIN	activator	Gene-targeted
CARBACHOL	None	Gene-targeted
CARBAMAZEPINE		Gene-targeted
CHLORZOXAZONE	potentiator; activator	Gene-targeted
CICLESONIDE	agonist; agonist	Gene-targeted
CIPROFLOXACIN	None	Gene-targeted
CLIOQUINOL		Gene-targeted
CLOBETASOL		Gene-targeted
CLOBETASOL PROPIONATE	agonist; agonist	Gene-targeted
CLOCORTOLONE		Gene-targeted
CLOCORTOLONE PIVALATE	agonist	Gene-targeted
CLOMIPRAMINE	None	Gene-targeted
CLOTRIMAZOLE	blocker	Gene-targeted
CORTISONE ACETATE	agonist	Gene-targeted
CORTIVAZOL	agonist	Gene-targeted
CROFELEMER	inhibitor; inhibitor	Gene-targeted
CURCUMIN		Gene-targeted
CYSTEAMINE HYDROCHLORIDE		Gene-targeted
DACTINOMYCIN		Gene-targeted
DAUNORUBICIN LIPOSOMAL		Gene-targeted

Therapeutics

Direct disease associations, biomarker-linked drugs, OSMF narrative-review natural agents, and merged ranking. Only agents with registry trials or published clinical literature are shown by default.

Show agents without direct trial evidence

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
CIPROFLOXACIN	Small molecule	Approved	Strong	100	8 trials 17 Cochrane 12 Meta-analysis 60 Trial pubs 35 RCT View evidence Registry trials NCT00910351 — Cipro Inhaler for Cystic Fibrosis Children Ages 6-12 (COMPLETED) PHASE1 NCT01400750 — Comparison of 2 Treatment Regimens for Eradication of P Aeruginosa Infection in Children With Cystic Fibrosis (COMPLETED) PHASE4 NCT00889967 — Safety and Efficacy Study of Ciprofloxacin for Inhalation in Patients With Non-Cystic Fibrosis Bronchiectasis "ORBIT-1" (COMPLETED) PHASE2 NCT00645788 — Study to Evaluate the Safety and Efficacy of Ciprofloxacin (Inhaled) in Patients With Cystic Fibrosis (COMPLETED) PHASE2 NCT03262142 — Targeted AntiBiotics for Chronic Pulmonary Diseases (TERMINATED) PHASE4 NCT01090908 — Evaluation of Ciprofloxacin for Inhalation to Cystic Fibrosis Patients With P. Aeruginosa (WITHDRAWN) NCT00097773 — Comparison of Two Treatment Regimens to Reduce PA Infection in Children With Cystic Fibrosis (COMPLETED) PHASE2 NCT06368804 — Comparison of Two Antibiotic Regimens for the Treatment of Early Airways Infection With PA in Adults With Bronchiectasis (RECRUITING) PHASE2 Published literature Cochrane review Comparative efficacy and safety of inhaled antibiotics in managing chronic Pseudomonas aeruginosa infection in patients with cystic fibrosis and bronchiectasis: a systematic review and network meta-analysis (Journal of thoracic disease, 2025) Cochrane review Antibiotic treatment for non-tuberculous mycobacteria lung infection in people with cystic fibrosis (The Cochrane database of systematic reviews, 2025) Cochrane review Antibiotic strategies for eradicating Pseudomonas aeruginosa in people with cystic fibrosis (The Cochrane database of systematic reviews, 2023) Cochrane review Safety and Efficacy of Devices Delivering Inhaled Antibiotics among Adults with Non-Cystic Fibrosis Bronchiectasis: A Systematic Review and a Network Meta-Analysis (Antibiotics (Basel, Switzerland), 2022) Cochrane review Intermittent prophylactic antibiotics for bronchiectasis (The Cochrane database of systematic reviews, 2022) Clinical trial publication Targeted AntiBiotics for Chronic pulmonary diseases (TARGET ABC): can targeted antibiotic therapy improve the prognosis of Pseudomonas aeruginosa-infected patients with chronic pulmonary obstructive disease, non-cystic fibrosis bronchiectasis, and asthma? A multicenter, randomized, controlled, open-label trial (Trials, 2022) Clinical trial publication Intravenous or oral antibiotic treatment in adults and children with cystic fibrosis and Pseudomonas aeruginosa infection: the TORPEDO-CF RCT (Health technology assessment (Winchester, England), 2021) Clinical trial publication Intravenous versus oral antibiotics for eradication of Pseudomonas aeruginosa in cystic fibrosis (TORPEDO-CF): a randomised controlled trial (The Lancet. Respiratory medicine, 2020) Clinical trial publication Microbiological changes observed over 48 weeks of treatment with inhaled liposomal ciprofloxacin in individuals with non-cystic fibrosis bronchiectasis and chronic Pseudomonas aeruginosa lung infection (Clinical microbiology and infection : the official publication of the European Society of Clinical Microbiology and Infectious Diseases, 2019) Clinical trial publication Ciprofloxacin Dry Powder for Inhalation: Inspiratory Flow in Patients with Non-cystic Fibrosis Bronchiectasis (Journal of aerosol medicine and pulmonary drug delivery, 2019) Randomized controlled trial Inhaled liposomal ciprofloxacin in patients with non-cystic fibrosis bronchiectasis and chronic lung infection with Pseudomonas aeruginosa (ORBIT-3 and ORBIT-4): two phase 3, randomised controlled trials (The Lancet. Respiratory medicine, 2019) CT.gov search · Cochrane · PubMed	Open Targets, ChEMBL	ChEMBL Open Targets
ACETAZOLAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ADALIMUMAB Repurposing	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ALCLOMETASONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ALCLOMETASONE DIPROPIONATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMCINONIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AMPHETAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
APIGENIN Repurposing	Small molecule	Approved	Strong	95	20 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ASPIRIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
ATALUREN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
AZACITIDINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BECLOMETHASONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BECLOMETHASONE DIPROPIONATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BETAMETHASONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
BETAMETHASONE ACETATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE BENZOATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE DIPROPIONATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE SODIUM PHOSPHATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE VALERATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BICALUTAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
BUDESONIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CAPSAICIN Repurposing	Small molecule	Approved	Strong	95	14 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CARBACHOL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBAMAZEPINE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CHLORZOXAZONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CICLESONIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CLIOQUINOL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CLOBETASOL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CLOBETASOL PROPIONATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CLOCORTOLONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CLOCORTOLONE PIVALATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
CLOMIPRAMINE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CLOTRIMAZOLE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CORTISONE ACETATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
CORTIVAZOL Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
CROFELEMER Repurposing	Small molecule	Approved	Strong	95	1 Cochrane 1 Trial pubs 1 RCT View evidence Published literature Cochrane review Crofelemer: In HIV Associated Diarrhea and Secretory Diarrhea - A Patent Perspective (Recent patents on anti-infective drug discovery, 2014) Clinical trial publication HALT-D: A Phase II Evaluation of Crofelemer for the Prevention and Prophylaxis of Diarrhea in Patients With Breast Cancer on Pertuzumab-Based Regimens (Clinical breast cancer, 2017) CT.gov search · Cochrane · PubMed	DGIdb	—
CURCUMIN Repurposing	Small molecule	Approved	Strong	95	1 trials 1 Trial pubs View evidence Registry trials NCT00219882 — Safety Study of Orally Administered Curcuminoids in Adult Subjects With Cystic Fibrosis (COMPLETED) PHASE1 Published literature Systematic review Curcumin supplementation in pediatric patients: A systematic review of current clinical evidence (Phytotherapy research : PTR, 2022) Clinical trial publication Clinical effects of the three CFTR potentiator treatments curcumin, genistein and ivacaftor in patients with the CFTR-S1251N gating mutation (Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2020) CT.gov search · Cochrane · PubMed	DGIdb	—
CYSTEAMINE HYDROCHLORIDE Repurposing	Small molecule	Approved	Strong	95	2 trials View evidence Registry trials NCT03000348 — A Study of the Dosing, Efficacy, and Safety of Oral Cysteamine in Adult Patients With Cystic Fibrosis Exacerbations (COMPLETED) PHASE2 NCT02212431 — First Study of Oral Cysteamine in Cystic Fibrosis (COMPLETED) PHASE1, PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—
DACTINOMYCIN Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DAUNORUBICIN LIPOSOMAL Repurposing	Small molecule	Approved	Strong	95	1 trials View evidence Registry trials NCT06526923 — A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF) (RECRUITING) PHASE1, PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—
DEFLAZACORT Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DESONIDE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DESOXIMETASONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DEUTIVACAFTOR Repurposing	Small molecule	Approved	Strong	95	8 trials 2 Cochrane 1 Meta-analysis 4 Trial pubs 2 RCT View evidence Registry trials NCT05844449 — Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older (ENROLLING_BY_INVITATION) PHASE3 NCT05033080 — A Phase 3 Study of VX-121 Combination Therapy in Participants With Cystic Fibrosis (CF) Heterozygous for F508del and a Minimal Function Mutation (F/MF) (COMPLETED) PHASE3 NCT04732910 — Modulate-CF: Cystic Fibrosis Transmembrane Regulator (CFTR) Biomarker Study to Evaluate the Rescue of Mutant CFTR in Patients With Cystic Fibrosis Treated With CFTR-modulators (RECRUITING) 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 (COMPLETED) PHASE3 NCT05444257 — A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy (ACTIVE_NOT_RECRUITING) PHASE3 NCT06683092 — Restarting Triple Therapy With Robust Monitoring for Adverse Events (RETRIAL) (RECRUITING) NCT07349394 — Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy Participants (COMPLETED) PHASE1 NCT06154447 — Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis (ACTIVE_NOT_RECRUITING) PHASE1 Published literature Cochrane review Comparative efficacy and safety of CFTR modulators for people with cystic fibrosis with phe508del mutation: a systematic review and bayesian network meta-analysis (EClinicalMedicine, 2025) Cochrane review Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del) (The Cochrane database of systematic reviews, 2023) Meta-analysis Comparative Efficacy of CFTR Modulators: A Network Meta-analysis (Lung, 2025) Clinical trial publication Improvements in health-related quality of life in people with cystic fibrosis ≥6 years of age treated with vanzacaftor/tezacaftor/deutivacaftor (Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2026) Clinical trial publication Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial (The Lancet. Respiratory medicine, 2025) Clinical trial publication Vanzacaftor-tezacaftor-deutivacaftor versus elexacaftor-tezacaftor-ivacaftor in individuals with cystic fibrosis aged 12 years and older (SKYLINE Trials VX20-121-102 and VX20-121-103): results from two randomised, active-controlled, phase 3 trials (The Lancet. Respiratory medicine, 2025) Clinical trial publication Safety and efficacy of vanzacaftor-tezacaftor-deutivacaftor in adults with cystic fibrosis: randomised, double-blind, controlled, phase 2 trials (The Lancet. Respiratory medicine, 2023) CT.gov search · Cochrane · PubMed	DGIdb	—
DEXAMETHASONE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DEXAMETHASONE ACETATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
DEXAMETHASONE SODIUM PHOSPHATE Repurposing	Small molecule	Approved	Strong	95	—	DGIdb	—
DIAZEPAM Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DICHLORPHENAMIDE Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
DIETHYLSTILBESTROL Repurposing	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Sources	Links
AMIKACIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
ATALUREN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
BAMOCAFTOR	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
CEPHALEXIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
COLISTIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
DEXAMETHASONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
GLUTATHIONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
HYDROCORTISONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
INSULIN DETEMIR	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
INSULIN GLARGINE	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
INSULIN HUMAN	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
LIPROTAMASE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
METHYLPREDNISOLONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
METHYLPREDNISOLONE ACETATE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
MYCOPHENOLATE MOFETIL	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
PANCREATIN	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
PANCRELIPASE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
PREDNISOLONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
PREDNISONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
REPAGLINIDE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
SODIUM CHLORIDE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
SOMATROPIN	Biologic	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
TIOTROPIUM	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
TRIAMCINOLONE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
TRIAMCINOLONE ACETONIDE	Small molecule	Phase III	Moderate	80	Open Targets	ChEMBL Open Targets
.ALPHA.1-PROTEINASE INHIBITOR HUMAN	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
16.ALPHA.-BROMOEPIANDROSTERONE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ACEBILUSTAT	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ALGINIC ACID	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ALVELESTAT	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
AMELUBANT	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
AMILORIDE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
AMITRIPTYLINE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ANAKINRA	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ANDECALIXIMAB	Biologic	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
ARGININE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
AZD-1236	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
BRENSOCATIB	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CAMOSTAT	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets
CARBON MONOXIDE	Small molecule	Phase II	Emerging	60	Open Targets	ChEMBL Open Targets

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
ACETAZOLAMIDE Repurposing via CFTR	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
ADALIMUMAB Repurposing via HFE	Small molecule	Approved	Strong	95	No indexed hits View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ALCLOMETASONE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	—
ALCLOMETASONE DIPROPIONATE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
AMCINONIDE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	—
AMPHETAMINE Repurposing via CFTR	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
APIGENIN Repurposing via CFTR	Small molecule	Approved	Strong	95	20 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
ASPIRIN Repurposing via HMOX1	Small molecule	Approved	Strong	95	—	DGIdb	—
ATALUREN Repurposing via FAU	Small molecule	Approved	Strong	95	—	DGIdb	—
AZACITIDINE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	—
BECLOMETHASONE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	—
BECLOMETHASONE DIPROPIONATE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	—
BETAMETHASONE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	—
BETAMETHASONE ACETATE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE BENZOATE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE DIPROPIONATE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE SODIUM PHOSPHATE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BETAMETHASONE VALERATE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
BICALUTAMIDE Repurposing via CFTR	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
BUDESONIDE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	—
CAPSAICIN Repurposing via CFTR	Small molecule	Approved	Strong	95	14 pubs View evidence CT.gov search · Cochrane · PubMed	DGIdb	—
CARBACHOL Repurposing via CFTR	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CARBAMAZEPINE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	—
CHLORZOXAZONE Repurposing via KCNN4	Small molecule	Approved	Strong	95	—	DGIdb	—
CICLESONIDE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	—
CIPROFLOXACIN Repurposing via CFTR	Small molecule	Approved	Strong	95	8 trials 17 Cochrane 12 Meta-analysis 60 Trial pubs 35 RCT View evidence Registry trials NCT00910351 — Cipro Inhaler for Cystic Fibrosis Children Ages 6-12 (COMPLETED) PHASE1 NCT01400750 — Comparison of 2 Treatment Regimens for Eradication of P Aeruginosa Infection in Children With Cystic Fibrosis (COMPLETED) PHASE4 NCT00889967 — Safety and Efficacy Study of Ciprofloxacin for Inhalation in Patients With Non-Cystic Fibrosis Bronchiectasis "ORBIT-1" (COMPLETED) PHASE2 NCT00645788 — Study to Evaluate the Safety and Efficacy of Ciprofloxacin (Inhaled) in Patients With Cystic Fibrosis (COMPLETED) PHASE2 NCT03262142 — Targeted AntiBiotics for Chronic Pulmonary Diseases (TERMINATED) PHASE4 NCT01090908 — Evaluation of Ciprofloxacin for Inhalation to Cystic Fibrosis Patients With P. Aeruginosa (WITHDRAWN) NCT00097773 — Comparison of Two Treatment Regimens to Reduce PA Infection in Children With Cystic Fibrosis (COMPLETED) PHASE2 NCT06368804 — Comparison of Two Antibiotic Regimens for the Treatment of Early Airways Infection With PA in Adults With Bronchiectasis (RECRUITING) PHASE2 Published literature Cochrane review Comparative efficacy and safety of inhaled antibiotics in managing chronic Pseudomonas aeruginosa infection in patients with cystic fibrosis and bronchiectasis: a systematic review and network meta-analysis (Journal of thoracic disease, 2025) Cochrane review Antibiotic treatment for non-tuberculous mycobacteria lung infection in people with cystic fibrosis (The Cochrane database of systematic reviews, 2025) Cochrane review Antibiotic strategies for eradicating Pseudomonas aeruginosa in people with cystic fibrosis (The Cochrane database of systematic reviews, 2023) Cochrane review Safety and Efficacy of Devices Delivering Inhaled Antibiotics among Adults with Non-Cystic Fibrosis Bronchiectasis: A Systematic Review and a Network Meta-Analysis (Antibiotics (Basel, Switzerland), 2022) Cochrane review Intermittent prophylactic antibiotics for bronchiectasis (The Cochrane database of systematic reviews, 2022) Clinical trial publication Targeted AntiBiotics for Chronic pulmonary diseases (TARGET ABC): can targeted antibiotic therapy improve the prognosis of Pseudomonas aeruginosa-infected patients with chronic pulmonary obstructive disease, non-cystic fibrosis bronchiectasis, and asthma? A multicenter, randomized, controlled, open-label trial (Trials, 2022) Clinical trial publication Intravenous or oral antibiotic treatment in adults and children with cystic fibrosis and Pseudomonas aeruginosa infection: the TORPEDO-CF RCT (Health technology assessment (Winchester, England), 2021) Clinical trial publication Intravenous versus oral antibiotics for eradication of Pseudomonas aeruginosa in cystic fibrosis (TORPEDO-CF): a randomised controlled trial (The Lancet. Respiratory medicine, 2020) Clinical trial publication Microbiological changes observed over 48 weeks of treatment with inhaled liposomal ciprofloxacin in individuals with non-cystic fibrosis bronchiectasis and chronic Pseudomonas aeruginosa lung infection (Clinical microbiology and infection : the official publication of the European Society of Clinical Microbiology and Infectious Diseases, 2019) Clinical trial publication Ciprofloxacin Dry Powder for Inhalation: Inspiratory Flow in Patients with Non-cystic Fibrosis Bronchiectasis (Journal of aerosol medicine and pulmonary drug delivery, 2019) Randomized controlled trial Inhaled liposomal ciprofloxacin in patients with non-cystic fibrosis bronchiectasis and chronic lung infection with Pseudomonas aeruginosa (ORBIT-3 and ORBIT-4): two phase 3, randomised controlled trials (The Lancet. Respiratory medicine, 2019) CT.gov search · Cochrane · PubMed	ChEMBL	ChEMBL Open Targets
CLIOQUINOL Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	—
CLOBETASOL Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	—
CLOBETASOL PROPIONATE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	—
CLOCORTOLONE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	—
CLOCORTOLONE PIVALATE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
CLOMIPRAMINE Repurposing via CFTR	Small molecule	Approved	Strong	95	—	ChEMBL	ChEMBL Open Targets
CLOTRIMAZOLE Repurposing via KCNN4	Small molecule	Approved	Strong	95	—	DGIdb	—
CORTISONE ACETATE Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	ChEMBL Open Targets
CORTIVAZOL Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	—
CROFELEMER Repurposing via CFTR	Small molecule	Approved	Strong	95	1 Cochrane 1 Trial pubs 1 RCT View evidence Published literature Cochrane review Crofelemer: In HIV Associated Diarrhea and Secretory Diarrhea - A Patent Perspective (Recent patents on anti-infective drug discovery, 2014) Clinical trial publication HALT-D: A Phase II Evaluation of Crofelemer for the Prevention and Prophylaxis of Diarrhea in Patients With Breast Cancer on Pertuzumab-Based Regimens (Clinical breast cancer, 2017) CT.gov search · Cochrane · PubMed	DGIdb	—
CURCUMIN Repurposing via CFTR	Small molecule	Approved	Strong	95	1 trials 1 Trial pubs View evidence Registry trials NCT00219882 — Safety Study of Orally Administered Curcuminoids in Adult Subjects With Cystic Fibrosis (COMPLETED) PHASE1 Published literature Systematic review Curcumin supplementation in pediatric patients: A systematic review of current clinical evidence (Phytotherapy research : PTR, 2022) Clinical trial publication Clinical effects of the three CFTR potentiator treatments curcumin, genistein and ivacaftor in patients with the CFTR-S1251N gating mutation (Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2020) CT.gov search · Cochrane · PubMed	DGIdb	—
CYSTEAMINE HYDROCHLORIDE Repurposing via CFTR	Small molecule	Approved	Strong	95	2 trials View evidence Registry trials NCT03000348 — A Study of the Dosing, Efficacy, and Safety of Oral Cysteamine in Adult Patients With Cystic Fibrosis Exacerbations (COMPLETED) PHASE2 NCT02212431 — First Study of Oral Cysteamine in Cystic Fibrosis (COMPLETED) PHASE1, PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—
DACTINOMYCIN Repurposing via NR3C1	Small molecule	Approved	Strong	95	—	DGIdb	—
DAUNORUBICIN LIPOSOMAL Repurposing via HFE	Small molecule	Approved	Strong	95	1 trials View evidence Registry trials NCT06526923 — A Phase 1/2 Trial of SP-101 for the Treatment of Cystic Fibrosis (CF) (RECRUITING) PHASE1, PHASE2 CT.gov search · Cochrane · PubMed	DGIdb	—

Natural Products

64 natural products with the same evidence schema as repurposed drugs — registry trials, PubMed literature, and reference links (pipeline-ranked). Only agents with direct clinical evidence are shown by default.

Reference lookups: Examine.com

Show agents without direct trial evidence

Drug	Type	Phase	Evidence	Score	Trials & literature	Sources	Links
Probiotics Natural product Cochrane review Dietary and complementary oral supplements for the management of chroni… Cochrane review Gut microbiome dysbiosis in chronic lung disease: a systematic review a… Cochrane review Effectiveness of Probiotics, Prebiotics, and Symbiotic Supplementation…	Probiotic	Approved	Moderate	26	7 trials 9 Cochrane 4 Meta-analysis 7 Trial pubs 7 RCT View evidence Registry trials NCT06057558 — Airway Microbiome of Cystic Fibrosis Patients (UNKNOWN) NA NCT05523180 — A Study to Evaluate the Effect of Probiotic Supplement on Quality of Life (COMPLETED) NA NCT06284577 — Quality of Life and Gut Health in Pediatric Patients With Cystic Fibrosis (RECRUITING) NA NCT01956916 — Probiotics in Cystic Fibrosis (COMPLETED) PHASE3 NCT01737983 — Effect of Lactobacillus Reuteri in Cystic Fibrosis (COMPLETED) PHASE4 NCT01201434 — Effect of Probiotics on Sputum Inflammation and Pulmonary Infections in Patients With Cystic Fibrosis (TERMINATED) NA NCT01961661 — Probiotics on Intestinal Inflammation in Cystic Fibrosis (COMPLETED) PHASE3 Published literature Cochrane review Dietary and complementary oral supplements for the management of chronic diseases in children: a systematic review (Frontiers in pediatrics, 2025) Cochrane review Gut microbiome dysbiosis in chronic lung disease: a systematic review and meta-analysis (Frontiers in cellular and infection microbiology, 2025) Cochrane review Effectiveness of Probiotics, Prebiotics, and Symbiotic Supplementation in Cystic Fibrosis Patients: A Systematic Review and Meta-Analysis of Clinical Trials (Medicina (Kaunas, Lithuania), 2025) Cochrane review A systematic cochrane review of probiotics for people with cystic fibrosis (Paediatric respiratory reviews, 2021) Cochrane review Probiotics for people with cystic fibrosis (The Cochrane database of systematic reviews, 2020) Meta-analysis Microbiota manipulation for weight change (Microbial pathogenesis, 2017) Systematic review Improving nutrition in cystic fibrosis: A systematic literature review (Nutrition (Burbank, Los Angeles County, Calif.), 2022) Systematic review Systematic Review of Probiotics for Cystic Fibrosis Patients: Moving Forward (Journal of pediatric gastroenterology and nutrition, 2019) Clinical trial publication The impact of probiotics on pulmonary, gastrointestinal, and growth outcomes in pediatric cystic fibrosis: a randomized controlled trial (BMC pediatrics, 2025) Clinical trial publication Probiotics in cystic fibrosis patients: A double blind crossover placebo controlled study: Pilot study from the ESPGHAN Working Group on Pancreas/CF (Clinical nutrition ESPEN, 2018) Clinical trial publication Lack of efficacy of Lactobacillus GG in reducing pulmonary exacerbations and hospital admissions in children with cystic fibrosis: A randomised placebo controlled trial (Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2018) Clinical trial publication Improvement of digestive health and reduction in proteobacterial populations in the gut microbiota of cystic fibrosis patients using a Lactobacillus reuteri probiotic preparation: a double blind prospective study (Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2014) CT.gov search · Cochrane · PubMed	Examine.com	Examine.com
Omega-3 Fatty Acids Natural product Cochrane review Dietary and complementary oral supplements for the management of chroni… Cochrane review Omega-3 fatty acid supplementation for cystic fibrosis Cochrane review Omega-3 fatty acids for cystic fibrosis	Nutraceutical	Phase III	Moderate	20	6 trials 9 Cochrane 7 Meta-analysis 8 RCT View evidence Registry trials NCT00530244 — Use of Formula Fortified With DHA in Infants With Cystic Fibrosis (COMPLETED) NA NCT02690857 — Study of Docosahexanoic Acid in Patients With Cystic Fibrosis (CF) (COMPLETED) PHASE2 NCT01329172 — Nasal Mucin Oral Supplementation Treatment Respiratory Inflammation Using PUFA in Cystic Fibrosis (CF) Patients (TERMINATED) NA NCT02239874 — VITamin D and OmegA-3 TriaL: Effects on Mammographic Density and Breast Tissue (UNKNOWN) NA NCT00959010 — Omega 3 Supplementation in Cystic Fibrosis Patients (COMPLETED) PHASE3 NCT02750501 — Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study (COMPLETED) NA Published literature Cochrane review Dietary and complementary oral supplements for the management of chronic diseases in children: a systematic review (Frontiers in pediatrics, 2025) Cochrane review Omega-3 fatty acid supplementation for cystic fibrosis (The Cochrane database of systematic reviews, 2020) Cochrane review Omega-3 fatty acids for cystic fibrosis (The Cochrane database of systematic reviews, 2016) Cochrane review Dietary interventions (plant sterols, stanols, omega-3 fatty acids, soy protein and dietary fibers) for familial hypercholesterolaemia (The Cochrane database of systematic reviews, 2014) Cochrane review Omega-3 fatty acids for cystic fibrosis (The Cochrane database of systematic reviews, 2013) CT.gov search · Cochrane · PubMed	Examine.com	PubChem Examine.com
Omega-3 Fatty Acids Natural product Cochrane review Dietary and complementary oral supplements for the management of chroni… Cochrane review Omega-3 fatty acid supplementation for cystic fibrosis Cochrane review Omega-3 fatty acids for cystic fibrosis	Nutraceutical	Phase III	Emerging	19	6 trials 9 Cochrane 7 Meta-analysis 8 RCT View evidence Registry trials NCT00530244 — Use of Formula Fortified With DHA in Infants With Cystic Fibrosis (COMPLETED) NA NCT02690857 — Study of Docosahexanoic Acid in Patients With Cystic Fibrosis (CF) (COMPLETED) PHASE2 NCT01329172 — Nasal Mucin Oral Supplementation Treatment Respiratory Inflammation Using PUFA in Cystic Fibrosis (CF) Patients (TERMINATED) NA NCT02239874 — VITamin D and OmegA-3 TriaL: Effects on Mammographic Density and Breast Tissue (UNKNOWN) NA NCT00959010 — Omega 3 Supplementation in Cystic Fibrosis Patients (COMPLETED) PHASE3 NCT02750501 — Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study (COMPLETED) NA Published literature Cochrane review Dietary and complementary oral supplements for the management of chronic diseases in children: a systematic review (Frontiers in pediatrics, 2025) Cochrane review Omega-3 fatty acid supplementation for cystic fibrosis (The Cochrane database of systematic reviews, 2020) Cochrane review Omega-3 fatty acids for cystic fibrosis (The Cochrane database of systematic reviews, 2016) Cochrane review Dietary interventions (plant sterols, stanols, omega-3 fatty acids, soy protein and dietary fibers) for familial hypercholesterolaemia (The Cochrane database of systematic reviews, 2014) Cochrane review Omega-3 fatty acids for cystic fibrosis (The Cochrane database of systematic reviews, 2013) CT.gov search · Cochrane · PubMed	Examine.com	PubChem Examine.com
N-Acetylcysteine Natural product Cochrane review Efficacy of N-acetylcysteine on idiopathic or postinfective non-cystic… Cochrane review Antioxidant supplementation for lung disease in cystic fibrosis Cochrane review Mucolytic agents versus placebo for chronic bronchitis or chronic obstr…	Amino acid	Approved	Emerging	19	5 trials 6 Cochrane 6 Meta-analysis 10 Trial pubs 5 RCT View evidence Registry trials NCT00996424 — The Effect of Inhaled N-Acetylcysteine Compared to Normal Saline on Sputum Rheology and Lung Function (TERMINATED) PHASE4 NCT03843541 — A Clinical Trial to Compare the Efficacy and Safety of 1-week Treatment of Intravenous N-acetylcysteine (NAC) 600 mg Twice Daily, Ambroxol Hydrochloride 30 mg Twice Daily and Placebo as Expectorant Therapies in Adult Chinese Patients With Respiratory Tract Diseases and Abnormal Mucus Secretions (COMPLETED) PHASE3 NCT02088216 — Effect of Long-term, High-dose N-acetylcysteine on Exacerbations of Bronchiectaisis (COMPLETED) NA NCT04798118 — Role of Toilet Bronchoscopy in RICU (UNKNOWN) NA NCT00809094 — NAC Phase IIB: A Multi-Center, Phase IIB, Randomized, Placebo-controlled, Double-Blind Study Of The Effects Of N-Acetylcysteine On Redox Changes and Lung Inflammation In Cystic Fibrosis Patients (COMPLETED) PHASE2 Published literature Cochrane review Efficacy of N-acetylcysteine on idiopathic or postinfective non-cystic fibrosis bronchiectasis: a systematic review and meta-analysis protocol (BMJ open, 2022) Cochrane review Antioxidant supplementation for lung disease in cystic fibrosis (The Cochrane database of systematic reviews, 2019) Cochrane review Mucolytic agents versus placebo for chronic bronchitis or chronic obstructive pulmonary disease (The Cochrane database of systematic reviews, 2019) Cochrane review Nebulized and oral thiol derivatives for pulmonary disease in cystic fibrosis (The Cochrane database of systematic reviews, 2013) Cochrane review Nebulized and oral thiol derivatives for pulmonary disease in cystic fibrosis (The Cochrane database of systematic reviews, 2009) Meta-analysis Mucoactive agents in bronchiectasis: a systematic review and meta-analysis (European respiratory review : an official journal of the European Respiratory Society, 2026) Meta-analysis Mucoactive agents for chronic, non-cystic fibrosis lung disease: A systematic review and meta-analysis (Respirology (Carlton, Vic.), 2017) Meta-analysis Efficacy and safety of acetylcysteine in "non-acetaminophen" acute liver failure: A meta-analysis of prospective clinical trials (Clinics and research in hepatology and gastroenterology, 2015) Systematic review Pharmacologic Agents That Promote Airway Clearance in Hospitalized Subjects: A Systematic Review (Respiratory care, 2015) Clinical trial publication The effect of short-term, high-dose oral N-acetylcysteine treatment on oxidative stress markers in cystic fibrosis patients with chronic P. aeruginosa infection -- a pilot study (Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2015) Clinical trial publication Long-term treatment with oral N-acetylcysteine: affects lung function but not sputum inflammation in cystic fibrosis subjects. A phase II randomized placebo-controlled trial (Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2015) Clinical trial publication A phase II study on safety and efficacy of high-dose N-acetylcysteine in patients with cystic fibrosis (European journal of medical research, 2009) CT.gov search · Cochrane · PubMed		PubChem
Creatine Natural product Cochrane review Deferasirox for managing transfusional iron overload in people with sic… Clinical trial publication Once versus three-times daily regimens of tobramycin treatment for pulm…	Amino acid	Preclinical	Moderate	17	1 Cochrane 1 Trial pubs 1 RCT View evidence Published literature Cochrane review Deferasirox for managing transfusional iron overload in people with sickle cell disease (The Cochrane database of systematic reviews, 2010) Clinical trial publication Once versus three-times daily regimens of tobramycin treatment for pulmonary exacerbations of cystic fibrosis--the TOPIC study: a randomised controlled trial (Lancet (London, England), 2005) CT.gov search · Cochrane · PubMed	Examine.com	PubChem Examine.com
Lactobacillus Reuteri Natural product Cochrane review Systematic Review of Probiotics for Cystic Fibrosis Patients: Moving Fo… Clinical trial publication The impact of probiotics on pulmonary, gastrointestinal, and growth out… Clinical trial publication The effect of Lactobacillus reuteri on pulmonary function test and grow…	Nutraceutical	Approved	Moderate	13	1 trials 1 Cochrane 4 Trial pubs 4 RCT View evidence Registry trials NCT01737983 — Effect of Lactobacillus Reuteri in Cystic Fibrosis (COMPLETED) PHASE4 Published literature Cochrane review Systematic Review of Probiotics for Cystic Fibrosis Patients: Moving Forward (Journal of pediatric gastroenterology and nutrition, 2019) Clinical trial publication The impact of probiotics on pulmonary, gastrointestinal, and growth outcomes in pediatric cystic fibrosis: a randomized controlled trial (BMC pediatrics, 2025) Clinical trial publication The effect of Lactobacillus reuteri on pulmonary function test and growth of cystic fibrosis patients (Journal of the science of food and agriculture, 2024) Clinical trial publication Improvement of digestive health and reduction in proteobacterial populations in the gut microbiota of cystic fibrosis patients using a Lactobacillus reuteri probiotic preparation: a double blind prospective study (Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2014) Clinical trial publication Lactobacillus reuteri ATCC55730 in cystic fibrosis (Journal of pediatric gastroenterology and nutrition, 2014) CT.gov search · Cochrane · PubMed	Examine.com	Examine.com
Synbiotics Natural product Cochrane review Effectiveness of Probiotics, Prebiotics, and Symbiotic Supplementation… Cochrane review Microbiota manipulation for weight change	Nutraceutical	Preclinical	Moderate	10	2 Cochrane 2 Meta-analysis View evidence Published literature Cochrane review Effectiveness of Probiotics, Prebiotics, and Symbiotic Supplementation in Cystic Fibrosis Patients: A Systematic Review and Meta-Analysis of Clinical Trials (Medicina (Kaunas, Lithuania), 2025) Cochrane review Microbiota manipulation for weight change (Microbial pathogenesis, 2017) CT.gov search · Cochrane · PubMed	Examine.com	Examine.com
Vitamin D3 Natural product Human Study Bolus weekly vitamin D3 supplementation impacts gut and airway microbio… Human Study Daily vitamin D supplementation was associated with reduced Aspergillus… Human Study 5-methyltehydrofolate (folic acid) and vitamin B12 ameliorate red blood…	Nutraceutical	Approved	Emerging	10	8 trials View evidence Registry trials NCT00788138 — Effects of Vitamin D Supplementation on Lung Function in an Acute Pulmonary Exacerbation of Cystic Fibrosis (COMPLETED) NA NCT01880346 — Comparison of Absorption of Vitamin D in Cystic Fibrosis (COMPLETED) PHASE4 NCT02225899 — BioEnergetics and Metabolomics in Cystic Fibrosis (COMPLETED) NCT03734744 — PK/PD of Vitamin D3 in Adults With CF (TERMINATED) NA NCT04411901 — The Role of Vitamin D3 in Pediatric Bronchiectasis Severity (COMPLETED) PHASE2, PHASE3 NCT02043717 — Increased Vitamin D Reduces Pulmonary Exacerbations in CF (COMPLETED) NCT02613884 — Safety, Efficacy, and Feasibility of High-dose Cholecalciferol in Pediatric Patients With Cystic Fibrosis (COMPLETED) PHASE2 NCT02239874 — VITamin D and OmegA-3 TriaL: Effects on Mammographic Density and Breast Tissue (UNKNOWN) NA CT.gov search · Cochrane · PubMed		PubChem
Vitamin C Natural product Cochrane review Interventions for chronic kidney disease in people with sickle cell dis… Cochrane review Antioxidant supplementation for lung disease in cystic fibrosis Cochrane review Antioxidant supplementation for lung disease in cystic fibrosis	Nutraceutical	Phase II	Emerging	10	5 trials 4 Cochrane 5 Trial pubs 3 RCT View evidence Registry trials NCT02690857 — Study of Docosahexanoic Acid in Patients With Cystic Fibrosis (CF) (COMPLETED) PHASE2 NCT05495243 — Phase 2a, 28-day Investigational Use Study of ARINA-1 in Non-Cystic Fibrosis Bronchiectasis (NCFBE) With Excess Mucus and Cough (COMPLETED) PHASE2 NCT01772758 — Cystic Fibrosis and Endothelial Function: At Rest and During Exercise (COMPLETED) PHASE2 NCT02690064 — Mechanisms for Vascular Dysfunction and Exercise Tolerance in CF (ACTIVE_NOT_RECRUITING) NA NCT06875050 — Antioxidant Supplements in Cystic Fibrosis Children (COMPLETED) NA Published literature Cochrane review Interventions for chronic kidney disease in people with sickle cell disease (The Cochrane database of systematic reviews, 2023) Cochrane review Antioxidant supplementation for lung disease in cystic fibrosis (The Cochrane database of systematic reviews, 2019) Cochrane review Antioxidant supplementation for lung disease in cystic fibrosis (The Cochrane database of systematic reviews, 2014) Cochrane review Antioxidant micronutrients for lung disease in cystic fibrosis (The Cochrane database of systematic reviews, 2010) Clinical trial publication Supplementation with red palm oil increases β-carotene and vitamin A blood levels in patients with cystic fibrosis (Mediators of inflammation, 2015) Clinical trial publication The effect of short-term, high-dose oral N-acetylcysteine treatment on oxidative stress markers in cystic fibrosis patients with chronic P. aeruginosa infection -- a pilot study (Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2015) Clinical trial publication Can changes in hydrophobicity increase the bioavailability of alpha-tocopherol? (European journal of nutrition, 2006) Clinical trial publication Improved antioxidant and fatty acid status of patients with cystic fibrosis after antioxidant supplementation is linked to improved lung function (The American journal of clinical nutrition, 2003) Clinical trial publication Evidence for increased oxidative damage in patients with cystic fibrosis (Pediatric research, 1994) CT.gov search · Cochrane · PubMed		PubChem
N-Acetylcysteine Natural product Cochrane review Efficacy of N-acetylcysteine on idiopathic or postinfective non-cystic… Cochrane review Antioxidant supplementation for lung disease in cystic fibrosis Cochrane review Mucolytic agents versus placebo for chronic bronchitis or chronic obstr…	Amino acid	Approved	Emerging	10	5 trials 6 Cochrane 6 Meta-analysis 10 Trial pubs 5 RCT View evidence Registry trials NCT00996424 — The Effect of Inhaled N-Acetylcysteine Compared to Normal Saline on Sputum Rheology and Lung Function (TERMINATED) PHASE4 NCT03843541 — A Clinical Trial to Compare the Efficacy and Safety of 1-week Treatment of Intravenous N-acetylcysteine (NAC) 600 mg Twice Daily, Ambroxol Hydrochloride 30 mg Twice Daily and Placebo as Expectorant Therapies in Adult Chinese Patients With Respiratory Tract Diseases and Abnormal Mucus Secretions (COMPLETED) PHASE3 NCT02088216 — Effect of Long-term, High-dose N-acetylcysteine on Exacerbations of Bronchiectaisis (COMPLETED) NA NCT04798118 — Role of Toilet Bronchoscopy in RICU (UNKNOWN) NA NCT00809094 — NAC Phase IIB: A Multi-Center, Phase IIB, Randomized, Placebo-controlled, Double-Blind Study Of The Effects Of N-Acetylcysteine On Redox Changes and Lung Inflammation In Cystic Fibrosis Patients (COMPLETED) PHASE2 Published literature Cochrane review Efficacy of N-acetylcysteine on idiopathic or postinfective non-cystic fibrosis bronchiectasis: a systematic review and meta-analysis protocol (BMJ open, 2022) Cochrane review Antioxidant supplementation for lung disease in cystic fibrosis (The Cochrane database of systematic reviews, 2019) Cochrane review Mucolytic agents versus placebo for chronic bronchitis or chronic obstructive pulmonary disease (The Cochrane database of systematic reviews, 2019) Cochrane review Nebulized and oral thiol derivatives for pulmonary disease in cystic fibrosis (The Cochrane database of systematic reviews, 2013) Cochrane review Nebulized and oral thiol derivatives for pulmonary disease in cystic fibrosis (The Cochrane database of systematic reviews, 2009) Meta-analysis Mucoactive agents in bronchiectasis: a systematic review and meta-analysis (European respiratory review : an official journal of the European Respiratory Society, 2026) Meta-analysis Mucoactive agents for chronic, non-cystic fibrosis lung disease: A systematic review and meta-analysis (Respirology (Carlton, Vic.), 2017) Meta-analysis Efficacy and safety of acetylcysteine in "non-acetaminophen" acute liver failure: A meta-analysis of prospective clinical trials (Clinics and research in hepatology and gastroenterology, 2015) Systematic review Pharmacologic Agents That Promote Airway Clearance in Hospitalized Subjects: A Systematic Review (Respiratory care, 2015) Clinical trial publication The effect of short-term, high-dose oral N-acetylcysteine treatment on oxidative stress markers in cystic fibrosis patients with chronic P. aeruginosa infection -- a pilot study (Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2015) Clinical trial publication Long-term treatment with oral N-acetylcysteine: affects lung function but not sputum inflammation in cystic fibrosis subjects. A phase II randomized placebo-controlled trial (Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2015) Clinical trial publication A phase II study on safety and efficacy of high-dose N-acetylcysteine in patients with cystic fibrosis (European journal of medical research, 2009) CT.gov search · Cochrane · PubMed		PubChem
Vitamin E Natural product Randomized controlled trial Fat-Soluble Vitamin Supplementation Using Liposomes, Cyclodextrins, or… Randomized controlled trial Moderate intake of docosahexaenoic acid raises plasma and platelet vita… Randomized controlled trial Bioavailability of oral vitamin E formulations in adult volunteers and…	Nutraceutical	Phase II	Emerging	10	6 trials 14 RCT View evidence Registry trials NCT02690857 — Study of Docosahexanoic Acid in Patients With Cystic Fibrosis (CF) (COMPLETED) PHASE2 NCT00889434 — Efficacy and Safety Study of EGCG/Tocotrienol in 18 Patients With Splicing-mutation-mediated Cystic Fibrosis (CF) (COMPLETED) NA NCT01859390 — Effects of an Antioxidant-Enriched Multivitamin Supplement on Inflammation and Oxidative Stress in Cystic Fibrosis (COMPLETED) PHASE2 NCT01772758 — Cystic Fibrosis and Endothelial Function: At Rest and During Exercise (COMPLETED) PHASE2 NCT02690064 — Mechanisms for Vascular Dysfunction and Exercise Tolerance in CF (ACTIVE_NOT_RECRUITING) NA NCT06875050 — Antioxidant Supplements in Cystic Fibrosis Children (COMPLETED) NA Published literature Randomized controlled trial Fat-Soluble Vitamin Supplementation Using Liposomes, Cyclodextrins, or Medium-Chain Triglycerides in Cystic Fibrosis: A Randomized Controlled Trial (Nutrients, 2021) Randomized controlled trial Moderate intake of docosahexaenoic acid raises plasma and platelet vitamin E levels in cystic fibrosis patients (Prostaglandins, leukotrienes, and essential fatty acids, 2016) Randomized controlled trial Bioavailability of oral vitamin E formulations in adult volunteers and children with chronic cholestasis or cystic fibrosis (Journal of clinical pharmacy and therapeutics, 2009) Randomized controlled trial Bioavailability of a novel, water-soluble vitamin E formulation in malabsorbing patients (Digestive diseases and sciences, 2007) Randomized controlled trial Dietary supplementation with multiple micronutrients: no beneficial effects in pediatric cystic fibrosis patients (Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2007) CT.gov search · Cochrane · PubMed		PubChem
Taurine Natural product Meta-analysis The effects of taurine supplementation on obesity, blood pressure and l… Randomized controlled trial Ursodeoxycholic acid for liver disease associated with cystic fibrosis:… Randomized controlled trial Serum bile acids and ursodeoxycholic acid treatment in cystic fibrosis-…	Amino acid	Preclinical	Emerging	10	1 Meta-analysis 5 RCT View evidence Published literature Meta-analysis The effects of taurine supplementation on obesity, blood pressure and lipid profile: A meta-analysis of randomized controlled trials (European journal of pharmacology, 2020) Randomized controlled trial Ursodeoxycholic acid for liver disease associated with cystic fibrosis: a double-blind multicenter trial. The Italian Group for the Study of Ursodeoxycholic Acid in Cystic Fibrosis (Hepatology (Baltimore, Md.), 1996) Randomized controlled trial Serum bile acids and ursodeoxycholic acid treatment in cystic fibrosis-related liver disease (European journal of gastroenterology & hepatology, 1996) Randomized controlled trial Effect of a medium dose of ursodeoxycholic acid with or without taurine supplementation on the nutritional status of patients with cystic fibrosis: a randomized, placebo-controlled, crossover trial (Journal of pediatric gastroenterology and nutrition, 1994) Randomized controlled trial Taurine decreases fecal fatty acid and sterol excretion in cystic fibrosis. A randomized double-blind trial (American journal of diseases of children (1960), 1991) Randomized controlled trial Taurine improves the absorption of a fat meal in patients with cystic fibrosis (Pediatrics, 1987) CT.gov search · Cochrane · PubMed		PubChem
Zinc Natural product Meta-analysis Pancreatic islet autoantibodies and their association with glycemic sta… Meta-analysis Drugs for preventing red blood cell dehydration in people with sickle c… Meta-analysis Treatment for osteoporosis in people with ß-thalassaemia	Nutraceutical	Phase II	Emerging	10	5 trials 16 Cochrane 11 Meta-analysis 6 RCT View evidence Registry trials NCT00104494 — Zinc Homeostasis and Kinetics in Children With Cystic Fibrosis (CF) (COMPLETED) NA NCT01859390 — Effects of an Antioxidant-Enriched Multivitamin Supplement on Inflammation and Oxidative Stress in Cystic Fibrosis (COMPLETED) PHASE2 NCT04302428 — Zinc Status and Growth in Cystic Fibrosis (COMPLETED) NCT00591864 — Comparison of MRI and Molecular Breast Imaging in Breast Diagnostic Evaluation (COMPLETED) NA NCT06875050 — Antioxidant Supplements in Cystic Fibrosis Children (COMPLETED) NA Published literature Meta-analysis Pancreatic islet autoantibodies and their association with glycemic status in cystic fibrosis patients: A comprehensive meta-analysis (Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2025) Meta-analysis Drugs for preventing red blood cell dehydration in people with sickle cell disease (The Cochrane database of systematic reviews, 2018) Meta-analysis Treatment for osteoporosis in people with ß-thalassaemia (The Cochrane database of systematic reviews, 2016) Meta-analysis Drugs for preventing red blood cell dehydration in people with sickle cell disease (The Cochrane database of systematic reviews, 2016) Systematic review Antioxidant Micronutrients and Essential Fatty Acids Supplementation on Cystic Fibrosis Outcomes: A Systematic Review (Journal of the Academy of Nutrition and Dietetics, 2020) Randomized controlled trial Zinc Supplementation for One Year Among Children with Cystic Fibrosis Does Not Decrease Pulmonary Infection (Respiratory care, 2016) Randomized controlled trial Effect of zinc supplementation on respiratory tract infections in children with cystic fibrosis (Pediatric pulmonology, 2008) Randomized controlled trial Copper enzyme activities in cystic fibrosis before and after copper supplementation plus or minus zinc (Metabolism: clinical and experimental, 2004) Randomized controlled trial Improved antioxidant and fatty acid status of patients with cystic fibrosis after antioxidant supplementation is linked to improved lung function (The American journal of clinical nutrition, 2003) Randomized controlled trial Effect of pancreatic enzymes on zinc absorption in cystic fibrosis (Journal of pediatric gastroenterology and nutrition, 1998) CT.gov search · Cochrane · PubMed		PubChem
Folate Natural product Cochrane review Folate supplementation in people with sickle cell disease Cochrane review Folate supplementation in people with sickle cell disease Human Study 5-methyltehydrofolate (folic acid) and vitamin B12 ameliorate red blood…	Nutraceutical	Preclinical	Emerging	10	1 trials 2 Cochrane View evidence Registry trials NCT00730509 — Effects of 5-Methyltetrahydrofolate and Vitamin B12 Supplemetation on Red Cell Membrane in Children With Cystic Fibrosis (COMPLETED) NA Published literature Cochrane review Folate supplementation in people with sickle cell disease (The Cochrane database of systematic reviews, 2018) Cochrane review Folate supplementation in people with sickle cell disease (The Cochrane database of systematic reviews, 2016) CT.gov search · Cochrane · PubMed		PubChem
Vitamin B12 Natural product Human Study 5-methyltehydrofolate (folic acid) and vitamin B12 ameliorate red blood… Human Study An antioxidant formula increases important fat-soluble nutrients and an… Human Study Bolus weekly vitamin D3 supplementation impacts gut and airway microbio…	Nutraceutical	Preclinical	Emerging	10	1 trials View evidence Registry trials NCT00730509 — Effects of 5-Methyltetrahydrofolate and Vitamin B12 Supplemetation on Red Cell Membrane in Children With Cystic Fibrosis (COMPLETED) NA CT.gov search · Cochrane · PubMed		PubChem
Vitamin K Natural product Randomized controlled trial Effect of vitamin K1 supplementation on vitamin K status in cystic fibr… Human Study High dose vitamin K is efficacious in correcting vitamin K deficiency i… Human Study 5-methyltehydrofolate (folic acid) and vitamin B12 ameliorate red blood…	Nutraceutical	Phase II	Emerging	10	5 trials 6 Trial pubs 4 RCT View evidence Registry trials NCT03285100 — The Effects of Discontinuation of Vitamin K Antagonists on the Rate of Elastin Degradation (UNKNOWN) NCT02043717 — Increased Vitamin D Reduces Pulmonary Exacerbations in CF (COMPLETED) NCT01859390 — Effects of an Antioxidant-Enriched Multivitamin Supplement on Inflammation and Oxidative Stress in Cystic Fibrosis (COMPLETED) PHASE2 NCT06875050 — Antioxidant Supplements in Cystic Fibrosis Children (COMPLETED) NA NCT04496921 — Vitamin K Supplementation to Reduce Deficiencies in Adults With Cystic Fibrosis (WITHDRAWN) NA Published literature Randomized controlled trial Effect of vitamin K1 supplementation on vitamin K status in cystic fibrosis patients (Journal of pediatric gastroenterology and nutrition, 1997) CT.gov search · Cochrane · PubMed		PubChem
Magnesium Natural product Clinical trial publication Changes in Mineral Micronutrient Status During and After Pulmonary Exac… Clinical trial publication Oral magnesium supplementation in children with cystic fibrosis improve… Clinical trial publication Once-daily tobramycin in the treatment of adult patients with cystic fi…	Nutraceutical	Phase III	Emerging	10	5 trials 4 Cochrane 3 Trial pubs 3 RCT View evidence Registry trials NCT05523180 — A Study to Evaluate the Effect of Probiotic Supplement on Quality of Life (COMPLETED) NA NCT01956916 — Probiotics in Cystic Fibrosis (COMPLETED) PHASE3 NCT03988816 — Effect of Roflumilast on Quality of Life, Lung Function and Mucus Properties in Patients With Bronchiectasis (UNKNOWN) PHASE2 NCT07022327 — Ivacaftor Impact in an Adolescent Without a Cystic Fibrosis-Associated CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) Mutation for Chronic Pancreatitis: Single-Patient Study Tracking Flare Frequency and Health Data Compared to Historical Records. (ACTIVE_NOT_RECRUITING) NCT01961661 — Probiotics on Intestinal Inflammation in Cystic Fibrosis (COMPLETED) PHASE3 Published literature Clinical trial publication Changes in Mineral Micronutrient Status During and After Pulmonary Exacerbation in Adults With Cystic Fibrosis (Nutrition in clinical practice : official publication of the American Society for Parenteral and Enteral Nutrition, 2015) Clinical trial publication Oral magnesium supplementation in children with cystic fibrosis improves clinical and functional variables: a double-blind, randomized, placebo-controlled crossover trial (The American journal of clinical nutrition, 2012) Clinical trial publication Once-daily tobramycin in the treatment of adult patients with cystic fibrosis (The European respiratory journal, 2002) CT.gov search · Cochrane · PubMed		PubChem
Creatine Natural product Cochrane review Deferasirox for managing transfusional iron overload in people with sic… Clinical trial publication Once versus three-times daily regimens of tobramycin treatment for pulm…	Amino acid	Preclinical	Emerging	7	1 Cochrane 1 Trial pubs 1 RCT View evidence Published literature Cochrane review Deferasirox for managing transfusional iron overload in people with sickle cell disease (The Cochrane database of systematic reviews, 2010) Clinical trial publication Once versus three-times daily regimens of tobramycin treatment for pulmonary exacerbations of cystic fibrosis--the TOPIC study: a randomised controlled trial (Lancet (London, England), 2005) CT.gov search · Cochrane · PubMed	Examine.com	PubChem Examine.com
Curcumin Natural product Systematic review Curcumin supplementation in pediatric patients: A systematic review of… Clinical trial publication Clinical effects of the three CFTR potentiator treatments curcumin, gen… Human Study Silibinins and curcumin as promising ligands against mutant cystic fibr…	Food compound	Phase I	Emerging	7	1 trials 1 Trial pubs View evidence Registry trials NCT00219882 — Safety Study of Orally Administered Curcuminoids in Adult Subjects With Cystic Fibrosis (COMPLETED) PHASE1 Published literature Systematic review Curcumin supplementation in pediatric patients: A systematic review of current clinical evidence (Phytotherapy research : PTR, 2022) Clinical trial publication Clinical effects of the three CFTR potentiator treatments curcumin, genistein and ivacaftor in patients with the CFTR-S1251N gating mutation (Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2020) CT.gov search · Cochrane · PubMed		PubChem
Lactobacillus Natural product Human Study Gut Bifidobacteria enrichment following oral Lactobacillus-supplementat… Human Study Lactobacillus GG supplementation reduces pulmonary exacerberations and… Human Study Probiotics might ameliorate the dysbiosis of cystis fibrosis gut microb…	Probiotic	Approved	Emerging	7	5 trials 5 Cochrane 9 RCT View evidence Registry trials NCT05523180 — A Study to Evaluate the Effect of Probiotic Supplement on Quality of Life (COMPLETED) NA NCT01837355 — Modulation of Intestinal and Pulmonary Inflammation by Lactobacillus Diet Supplementation in Pediatric Cystic Fibrosis (TERMINATED) NA NCT01956916 — Probiotics in Cystic Fibrosis (COMPLETED) PHASE3 NCT01737983 — Effect of Lactobacillus Reuteri in Cystic Fibrosis (COMPLETED) PHASE4 NCT01961661 — Probiotics on Intestinal Inflammation in Cystic Fibrosis (COMPLETED) PHASE3 CT.gov search · Cochrane · PubMed		—
Coenzyme Q10 Natural product Human Study An antioxidant formula increases important fat-soluble nutrients and an… Human Study Coenzyme Q10 levels are low in children with cystic fibrosis, indicatin…	Nutraceutical	Preclinical	Emerging	7	—		PubChem
L-Arginine Natural product Human Study L-arginine improves exhaled nitric oxide and pulmonary function in pati… Human Study L-arginine supplementation improve nitric oxide formation in cystic fib…	Amino acid	Preclinical	Emerging	7	—		PubChem
Resveratrol Natural product Human Study Silibinins and curcumin as promising ligands against mutant cystic fibr… Animal Study Resveratrol partially rescues the refractory CFTR dependent β adrenergi… In Vitro Study Curcumin opens cystic fibrosis transmembrane conductance regulator chan…	Food compound	Preclinical	Emerging	7	—		PubChem
Selenium Natural product Human Study Antioxidant supplementation may have therapeutic value in the treatment… Human Study Antioxidant supplementation may have therapeutic value in the treatment…	Nutraceutical	Preclinical	Emerging	7	—		PubChem
Sulforaphane Natural product Human Study Oral sulforaphane safely and effectively induces mucosal Phase II enzym…	Food compound	Preclinical	Emerging	7	—		PubChem
Feverfew Natural product Animal Study Parthenolide (from Feverfew) may down-regulate excessive inflammation i…	Botanical	Preclinical	Emerging	7	—		PubChem
Cordyceps Natural product In Vitro Study Cordyceps may play a therapeutic role in improving pulmonary function i…	Mushroom	Preclinical	Emerging	7	—		—
Genistein Natural product In Vitro Study Compounds found within Coleus (forskolin) and Soy (genistein) may have… In Vitro Study Genistein has a genocorrective effect in Cystic Fibrosis In Vitro Study Genistein may be useful in patients with cystic fibrosis and the G551D-…	Food compound	Preclinical	Emerging	0	—		PubChem
Glutathione Natural product Human Study Glutathione improves clinical markers in cystic fibrosis patients. Human Study Inhaled glutathione improves symptoms in patients with cystic fibrosis. Human Study Improved glutathione status in young adult patients with cystic fibrosi…	Nutraceutical	Preclinical	Emerging	0	—		—
Lactoferrin Natural product Human Study The inability of patients with cystic fibrosis to produce adequate lact… Animal Study, In Vitro Study Lactoferrin Decreases Inflammatory Response by Cystic Fibrosis Bronchia… In Vitro Study Lactoferrin inhibits Pseudomonas aeruginosa and Burkholderia cenocepia,…	Food compound	Preclinical	Emerging	0	—		PubChem
Flavonoids Natural product Animal Study Pinostrobin inhibits renal CFTR-mediated Cl- secretion and retards cyst… In Vitro Study Flavonoids may contribute to correction of the expression of the CFTR g… In Vitro Study Flavonoids may improve the expression of the defective CFTR gene in Cys…	Nutraceutical	Preclinical	Emerging	0	—		—
Antioxidant Formulas Natural product Human Study An antioxidant formula increases important fat-soluble nutrients and an… Human Study Antioxidant supplementation may have therapeutic value in the treatment… Human Study Antioxidant supplementation may have therapeutic value in the treatment…	Nutraceutical	Preclinical	Emerging	0	—		—
Beta-Carotene Natural product Human Study Antioxidant supplementation may have therapeutic value in the treatment… Human Study Antioxidant supplementation may have therapeutic value in the treatment… Human Study Beta-carotene supplementation ameliorates deficiencies and reduces oxid…	Nutraceutical	Preclinical	Emerging	0	—		—
Whey Natural product Human Study Improved glutathione status in young adult patients with cystic fibrosi… Human Study Oral supplementation with pressurized whey improves nutritional status…	Nutraceutical	Preclinical	Emerging	0	—		—
Salt Natural product Human Study Inhaled hypertonic saline accelerates mucus clearance and improves lung… Human Study Inhaled hypertonic saline significantly reduces the incidence of pulmon…	Nutraceutical	Preclinical	Emerging	0	—		—
Manuka Honey Natural product Human Study Manuka honey versus saline sinus irrigation in the treatment of cystic… Human Study Fractional exhalation nitric oxide changes in cystic fibrosis patients… In Vitro Study The use of honey as a combination agent may be possible for the managem…	Nutraceutical	Preclinical	Emerging	0	—		—
Ursodeoxycholic Acid Natural product Human Study Ursodeoxycholic acid and taurine have a therapeutic role in treating li… Human Study Ursodeoxycholic acid is associated with improvement in hepatobiliary sy…	Nutraceutical	Preclinical	Emerging	0	—		—
Lipase Natural product Human Study A high lipase pancreatic enzyme is superior to regular pancreatin in ad…	Nutraceutical	Preclinical	Emerging	0	—		—
Pancreatic Enzymes Natural product Human Study A high lipase pancreatic enzyme is superior to regular pancreatin in ad… Human Study Potato crisp consumption without pancreatic enzyme supplementation has… Plant source enzymes may provide ideal acid-stable adjunct to pancreati…	Nutraceutical	Preclinical	Emerging	0	—		—
Carotenoids Natural product Human Study An antioxidant formula increases important fat-soluble nutrients and an…	Nutraceutical	Preclinical	Emerging	0	—		—
Choline Natural product Human Study Choline-related supplements may improve status of children with Cystic…	Nutraceutical	Preclinical	Emerging	0	—		—
EGCG (Epigallocatechin Gallate) Natural product Animal Study, Human Study, In Vitro Study Cysteamine plus epigallocatechin gallate can be used for autophagy-depe…	Nutraceutical	Preclinical	Emerging	0	—		—
Flaxseed Natural product Human Study Flaxseed modulates inflammatory and oxidative stress biomarkers in cyst…	Nutraceutical	Preclinical	Emerging	0	—		—
Lignans Natural product Human Study Flaxseed modulates inflammatory and oxidative stress biomarkers in cyst…	Nutraceutical	Preclinical	Emerging	0	—		—
Honey Natural product Human Study Fractional exhalation nitric oxide changes in cystic fibrosis patients… Human Study Manuka honey versus saline sinus irrigation in the treatment of cystic… In Vitro Study The use of honey as a combination agent may be possible for the managem…	Nutraceutical	Preclinical	Emerging	0	—		—
Ketone Bodies Natural product Human Study Low-dose ketone monoester administration in adults with cystic fibrosis.	Nutraceutical	Preclinical	Emerging	0	—		—
Gamma-Linoleic Acid (GLA) Natural product Human Study Omega-3 and gamma-linolenic fatty acids improve pulmonary status (lung…	Nutraceutical	Preclinical	Emerging	0	—		—
Vitamin a Natural product Human Study 5-methyltehydrofolate (folic acid) and vitamin B12 ameliorate red blood… Human Study An antioxidant formula increases important fat-soluble nutrients and an… Human Study Bolus weekly vitamin D3 supplementation impacts gut and airway microbio…	Food compound	Preclinical	Emerging	0	—		PubChem
Ganoderic Acid Natural product Animal Study, In Vitro Study The SIRT3 activator ganoderic acid D regulates airway mucin MUC5AC expr…	Nutraceutical	Preclinical	Emerging	0	—		—
St. John's Wort Natural product Animal Study Hyperforin, a compound found within St. John's wort, has powerful…	Botanical	Preclinical	Emerging	0	—		PubChem
Trichilia Rubescens Natural product In Vitro Study A natural compound derived from Trichilia rubescens, corrects the defec…	Nutraceutical	Preclinical	Emerging	0	—		—
Amla Fruit Natural product In Vitro Study Amla (Emblica officinalis) extract contains a compound known as pyrogal… In Vitro Study Bergamot fruit extracts and identified components alter expression of i…	Nutraceutical	Preclinical	Emerging	0	—		—
Bergamot Orange Natural product In Vitro Study Bergamot fruit extracts and identified components alter expression of i…	Nutraceutical	Preclinical	Emerging	0	—		—
Cayenne Natural product In Vitro Study Capsaicin may help correct defective expression of the CFTR gene in Cys…	Nutraceutical	Preclinical	Emerging	0	—		—
Coleus Forskohlii Natural product In Vitro Study Compounds found within Coleus (forskolin) and Soy (genistein) may have…	Nutraceutical	Preclinical	Emerging	0	—		—
Evening Primrose Oil Natural product Review Evening primrose oil may partially correct the theoretical delta-6-desa…	Nutraceutical	Preclinical	Emerging	0	—		—
Curcuminoids Natural product Human Study Silibinins and curcumin as promising ligands against mutant cystic fibr… Animal Study Curcumin has a corrective effect on the expression of genetic defects a… In Vitro Study Curcumin enhances the functional competence of the cystic-fibrosis asso…	Nutraceutical	Preclinical	Emerging	0	—		—
Isoflavones Natural product In Vitro Study Genistein may help correct defective CFTR gene in Cystic Fibrosis	Nutraceutical	Preclinical	Emerging	0	—		—
Ginseng (Korean) Natural product In Vitro Study Korean red ginseng aqueous extract improves markers of mucociliary clea…	Nutraceutical	Preclinical	Emerging	0	—		—
Sodium Bicarbonate Natural product In Vitro Study Novel therapy of bicarbonate, glutathione and ascorbic acid improves cy…	Food compound	Preclinical	Emerging	0	—		PubChem
Olive Leaf Extract Natural product In Vitro Study Olive leaf extract as a novel antioxidant that ameliorates the inflamma…	Nutraceutical	Preclinical	Emerging	0	—		—
Breast Milk Natural product Review Prolonged breastfeeding is beneficial in children with cystic fibrosis…	Nutraceutical	Preclinical	Emerging	0	—		—
Caffeic Acid Phenethyl Ester Natural product Human Study Low-dose ketone monoester administration in adults with cystic fibrosis. Human Study There is an association between cholesterol oxidation and Cystic Fibros… Review Role of inflammation and oxidative stress in tissue damage associated w…	Food compound	Preclinical	Emerging	0	—		PubChem
Enzyme Therapy Natural product Human Study A high lipase pancreatic enzyme is superior to regular pancreatin in ad… Human Study Coenzyme Q10 levels are low in children with cystic fibrosis, indicatin… Human Study Omega-3 supplementation offers several clinical benefits as to the numb…	Nutraceutical	Preclinical	Emerging	0	—		—

Alterations and therapeutics are aggregated from public databases (Open Targets, HPO, ChEMBL, DGIdb, and others). Natural agents are candidates from OSMF narrative reviews on opensourcemed.info/chronic-disease.html — not approved treatments. This page is for research orientation only — not medical advice.