Could a blood vessel drug boost exercise capacity in cystic fibrosis?
NCT ID NCT04039087
First seen Aug 28, 2026 · Last updated Aug 28, 2026
Summary
This trial tests whether sildenafil, a drug that relaxes blood vessels, can improve exercise tolerance in people with cystic fibrosis. Researchers think that vascular dysfunction may contribute to exercise intolerance in CF, and sildenafil might help reverse that. Participants take either sildenafil or a placebo three times daily, and the study measures how far they can walk in six minutes and their respiratory quality of life. The goal is to see if this drug can help people with CF be more active and feel better.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- sildenafil (an oral drug that relaxes blood vessels, taken as a 40 mg capsule three times daily)
- What this could lead to
- If it works, sildenafil could become a new way to improve exercise capacity and quality of life in people with cystic fibrosis, potentially improving long-term outcomes.
- What could go wrong
- This is a small, early-stage trial, so results may not apply to everyone with CF. Sildenafil can cause side effects like headache, flushing, and low blood pressure.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2/3
Runs two stages together: whether the treatment works, then large-scale confirmation.
- Participants
-
31 people
The number who actually took part.
- Started
-
Sep 2019
- Finished
-
Jun 2024
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
9 years and older
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Confirmed diagnosis of cystic fibrosis (CF) based on the following criteria: Positive sweat chloride concentration ≥60 milliequivalents (mEq)/liter (by pilocarpine iontophoresis) and/or genotype with two identifiable disease-causing mutations consistent with CF, and accompanied by one or more clinical features consistent with the CF phenotype * Male or female patients ≥ 9 years of age * forced expiratory volume at one second (FEV1) ≥ 30% predicted and ≤ 80% for patients ≥ 18 years of age and ≤ 85% for patients \< 18 years of age * Clinically stable without evidence of acute upper or lower respiratory tract infection or current pulmonary exacerbation within the 14 days prior to the screening visit * Resting oxygen saturation (room air) ≥ 85% * Patients with or without CF related diabetes * Ability to perform spirometry reproducibly (according to American Thoracic Society) criteria * Willingness to maintain chronic CF medication schedule (e.g. alternating month inhaled antibiotics) Exclusion Criteria: * Children 8 yrs. old and younger * Subjects who weigh \< 20 Kgs * History of hypersensitivity to sildenafil * Use of an investigational agent within the 4-week period prior to Visit 1 (Day 0) * Breastfeeding, pregnant, or verbal expression of unwillingness to practice an acceptable birth control method (abstinence, hormonal or barrier methods, partner sterilization or intrauterine device) during participation in the study for women of child-bearing potential. * History of significant hepatic disease (aspartate transaminase or alanine transaminase \> 3 times the upper limit of normal at screening, documented biliary cirrhosis, or portal hypertension), * History of significant cardiovascular disease (history of aortic stenosis, coronary artery disease, or life-threatening arrhythmia), * History of severe neurological disease (e.g. history of stroke), * History of severe hematologic disease (e.g. history of bleeding diathesis; current international normalized ratio (INR) \> 2.0 * History of severe ophthalmologic disease (e.g. history of retinal impairment or non-arteritic ischemic optic neuritis) * History of severe renal impairment (creatinine \>1.8 mg/dL.) * Inability to swallow pills * Previous organ transplantation * Use of concomitant nitrates, α-blocker, or Ca channel blocker (currently or within one month of Visit 1) * Use of concomitant medications known to be potent inhibitors of CYP3A4 \[e.g. ketoconazole, itraconazole, ritonavir, clarithromycin, erythromycin, rifampin (currently or within one month of initiation of study drug)\] (NOTE: use of azithromycin is NOT a cause for exclusion) * History of sputum or throat swab culture yielding Burkholderia cepacia or Mycobacteria massiliense within 2 years of screening * History of migraine headaches. * Presence of a condition or abnormality that in the opinion of the investigator would compromise the safety of the subject or the quality of the data * Initiation of a cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy less than 1 month prior to first dose of sildenafil or placebo * Use of anticoagulants * Frank pulmonary hypertension\[right ventricular systolic pressure (RVSP) \>40 mm Hg by echocardiography)
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Cystic fibrosis are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Augusta University
Augusta, Georgia, 30912, United States
-
National Jewish Health
Denver, Colorado, 80206, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Real world clinical outcomes with novel modulator therapy combinations in people with CF (RECOVER)
- Can video calls reveal how cystic fibrosis affects daily life?
- Could cystic fibrosis offer hidden protection against COVID-19?
- Can a 'Treatable Traits' approach unlock better care for all airway diseases?
- A smartphone tool may help cystic fibrosis patients survive the lung transplant waitlist
- Can a smartphone app reveal how CF treatments really work at home?