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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Last refreshed on: 12 December 2020
Main ID:  NCT00659529
Date of registration: 14/04/2008
Prospective Registration: Yes
Primary sponsor: National Jewish Health
Public title: Safety and Efficacy of Sildenafil in Cystic Fibrosis (CF) Lung Disease
Scientific title: The Role of Phosphodiesterase Inhibitors in CF Lung Disease
Date of first enrolment: August 2008
Target sample size: 36
Recruitment status: Completed
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 1/Phase 2
Countries of recruitment
United States
Name:     Jennifer L Taylor-Cousar, MD
Affiliation:  National Jewish Health
Key inclusion & exclusion criteria

Inclusion Criteria:

- Confirmed diagnosis of CF based on the following criteria:

- Positive sweat chloride =60mEq/liter (by pilocarpine iontophoresis) and/or

- Genotype with two identifiable mutations consistent with CF, and accompanied by
one or more clinical features consistent with the CF phenotype

- Male or female patients = 12 years of age

- FEV1 = 50% predicted (Knudson) 31

- 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

- Ability to reproducibly perform spirometry (according to ATS criteria)

- Ability to produce at least 1mL of sputum spontaneously, or be willing to undergo
sputum induction

- Ability to understand and sign a written informed consent or assent and comply with
the requirements of the study

- Chronic bacterial colonization (3 documented positive cultures in the prior 2 years of
which at least one was obtained in the 3 months prior to randomization)

Exclusion Criteria:

- 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

- Daily use of systemic corticosteroids and/or NSAIDs within 4 weeks of the study or as
needed use within 72 hours prior to the screening visit

- History of significant hepatic (SGOT or SGPT > 3 times the upper limit of normal at
screening, documented biliary cirrhosis, or portal hypertension), cardiovascular
(history of aortic stenosis, coronary artery disease, pulmonary hypertension with
right ventricular systolic pressure >55 mmHg or life-threatening arrhythmia),
neurological (history of stroke), hematologic (history of bleeding diathesis),
ophthalmologic (history of retinal impairment or non-arteritic ischemic optic
neuritis) or renal impairment (creatinine >1.8 mg/dL.)

- Inability to swallow pills

- Previous lung transplantation

- Use of concomitant nitrates, a-blocker, or Ca channel blocker

- Use of concomitant medications known to be potent inhibitors of CYP3A4 (e.g.
ketoconazole, itraconazole, ritonavir, clarithromycin, erythromycin, rifampin)

- 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

- Weight less than 40 kg

- History of sputum or throat swab culture yielding Burkholderia cepacia within 2 years
of screening

- Resting room air oxygen saturation <93%

- History of migraines

Age minimum: 14 Years
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Cystic Fibrosis
Drug: sildenafil
Primary Outcome(s)
Sputum Elastase [Time Frame: Pre/post therapy]
Secondary Outcome(s)
CFQ-R [Time Frame: Pre/post therapy]
Exhaled Breath Condensate pH [Time Frame: Pre/post therapy]
Serum Sildenafil Levels [Time Frame: Pre/during therapy]
Secondary ID(s)
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Cystic Fibrosis Foundation
Ethics review
Results available: Yes
Date Posted: 02/05/2014
Date Completed:
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