Blood Flow and Vascular Function in Cystic Fibrosis

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

19 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-04-30

Study Completion Date

2018-07-31

Brief Summary

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Cystic fibrosis (CF) has many health consequences. A reduction in the ability to perform exercise in patients with CF is related to greater death rates, steeper decline in lung function, and more frequent lung infections. However, the physiological mechanisms for this reduced exercise capacity are unknown. The investigators laboratory recently published the first evidence of systemic vascular dysfunction in patients with CF. Therefore, it is reasonable to suspect that the blood vessels are involved with exercise intolerance in CF. This study will look at how 1) blood flow and 2) artery function contribute to exercise capacity in CF.

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Detailed Description

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The most disturbing aspect of Cystic Fibrosis (CF) is the associated premature death. Low exercise capacity predicts death in patients with CF and is also associated with a steeper decline in lung function and more lung infections. A critical barrier to improving exercise tolerance in patients with CF is the investigators lack of knowledge regarding the different physiological mechanisms which contribute to their lower exercise capacity. We have compelling data to indicate that the blood vessels may contribute to the low exercise capacity in CF. The impact of this proof of concept investigation will test Phosphodiesterase Type 5 inhibitors (PDE5) inhibitors as a potential therapy in CF and will explore blood flow and endothelial function as potential mechanisms which contribute to exercise intolerance in CF. Improvements in exercise capacity will not only contribute to a better quality of live for patients with CF, it will also increase longevity in these patients.

Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Acute Study: Sildenafil first, then Placebo

In randomized order, on two separate days, endothelial function and exercise capacity will be determined 1 hour following a single dose of sildenafil (50 mg) or placebo.

Group Type EXPERIMENTAL

Sildenafil (Acute-1 hour)

Intervention Type DRUG

Vascular function will be assessed 1 hour following oral ingestion of sildenafil (50 mg)

Placebo

Intervention Type DRUG

Sugar pill designed to mimic the sildenafil treatment

Acute Study: Placebo first, then Sildenafil

In randomized order, on two separate days, endothelial function and exercise capacity will be determined 1 hour following a single dose of sildenafil (50 mg) or placebo.

Group Type EXPERIMENTAL

Sildenafil (Acute-1 hour)

Intervention Type DRUG

Vascular function will be assessed 1 hour following oral ingestion of sildenafil (50 mg)

Placebo

Intervention Type DRUG

Sugar pill designed to mimic the sildenafil treatment

Sub-Chronic Study Sildenafil

Following the acute study, patients will be instructed to take 20 mg of sildenafil, three times a day, for 4 weeks. Endothelial function will be determined within 48 hours following the last dose.

Group Type EXPERIMENTAL

Sildenafil (Subchronic-4 weeks)

Intervention Type DRUG

Vascular function will be assessed 4 weeks following 20 mg three times per day (TID) of sildenafil for four weeks

Interventions

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Sildenafil (Acute-1 hour)

Vascular function will be assessed 1 hour following oral ingestion of sildenafil (50 mg)

Intervention Type DRUG

Sildenafil (Subchronic-4 weeks)

Vascular function will be assessed 4 weeks following 20 mg three times per day (TID) of sildenafil for four weeks

Intervention Type DRUG

Placebo

Sugar pill designed to mimic the sildenafil treatment

Intervention Type DRUG

Other Intervention Names

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Viagra Revatio Viagra Revatio

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of CF and healthy controls
* Men and women (greater than 18 yrs. old)
* Resting oxygen saturation (room air) greater than 90%
* Forced expiratory volume (FEV1) percent predicted greater than 30%
* Patients with or without CF related diabetes
* Traditional CF-treatment medications
* Ability to perform reliable/reproducible pulmonary function tests (PFT)
* Clinically stable for 2 weeks (no exacerbations or need for antibiotic treatment within 2 weeks of testing or major change in medical status)

Exclusion Criteria

* Children less than 17 years old
* Body mass less than 20 kg
* A diagnosis of pulmonary arterial hypertension (PAH)
* FEV1 less than 30% of predicted
* Resting oxygen saturation (SpO2) less than 90%
* Self-reported to be a smoker
* Current use of any vaso-active medications
* History of migraine headaches
* Pregnant or nursing at the time of the investigation
* A clinical diagnosis of cardiovascular disease, hypertension, or CF related diabetes

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes