Could a blood vessel drug boost exercise capacity in cystic fibrosis?

NCT ID NCT04039087

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

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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

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)

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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

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