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Safety and Efficacy of Sildenafil in Cystic Fibrosis (CF) Lung Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00659529
Recruitment Status : Completed
First Posted : April 16, 2008
Results First Posted : May 2, 2014
Last Update Posted : October 19, 2020
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Jennifer Taylor-Cousar, National Jewish Health

Tracking Information
First Submitted Date  ICMJE April 14, 2008
First Posted Date  ICMJE April 16, 2008
Results First Submitted Date  ICMJE April 1, 2014
Results First Posted Date  ICMJE May 2, 2014
Last Update Posted Date October 19, 2020
Actual Study Start Date  ICMJE August 2008
Actual Primary Completion Date August 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 1, 2014)
Sputum Elastase [ Time Frame: Pre/post therapy ]
Original Primary Outcome Measures  ICMJE
 (submitted: April 15, 2008)
Sputum IL-8 and elastase [ Time Frame: Pre/post therapy ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 15, 2008)
  • Exhaled Breath Condensate pH [ Time Frame: Pre/post therapy ]
  • CFQ-R [ Time Frame: Pre/post therapy ]
  • Serum Sildenafil Levels [ Time Frame: Pre/during therapy ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Safety and Efficacy of Sildenafil in Cystic Fibrosis (CF) Lung Disease
Official Title  ICMJE The Role of Phosphodiesterase Inhibitors in CF Lung Disease
Brief Summary The purpose of this study is to determine whether sildenafil can decrease inflammation in CF lung disease.
Detailed Description This study is an open-label study that examines the use of sildenafil (Revatio) in clinically stable patients with mild to moderate CF lung disease. The length of participation for each subject will be approximately 10 weeks, and will consist of a screening visit, a study visit for initiation of study drug if subject qualifies, interim visits to escalate drug dose, obtain drug levels, review concomitant medications and assess side effects, a visit at the end of the therapy period (to reassess inflammatory markers, laboratory studies and side effects,) and a follow-up assessment 2 weeks after subject completion.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE Drug: sildenafil
Sildenafil will be given 20mg po tid for 1 week, and then will be give 40mg po tid for 5 weeks.
Other Name: Revatio
Study Arms  ICMJE Experimental: 1
All subjects will receive oral sildenafil three times per day during the study. Study endpoints will be measured before the treatment period and at the end of the treatment period.
Intervention: Drug: sildenafil
Publications *

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 7, 2013)
Original Estimated Enrollment  ICMJE
 (submitted: April 15, 2008)
Actual Study Completion Date  ICMJE December 2012
Actual Primary Completion Date August 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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, α-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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 14 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT00659529
Other Study ID Numbers  ICMJE 851R14-8510M3
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Jennifer Taylor-Cousar, National Jewish Health
Study Sponsor  ICMJE National Jewish Health
Collaborators  ICMJE Cystic Fibrosis Foundation
Investigators  ICMJE
Principal Investigator: Jennifer L Taylor-Cousar, MD National Jewish Health
PRS Account National Jewish Health
Verification Date October 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP