Evaluation and Treatment of Pulmonary Vascular Disease in Moderate to Severe CF

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02626182
• Date of registration: 03/12/2015
• Prospective Registration: No
• Primary sponsor: National Jewish Health
• Public title: Evaluation and Treatment of Pulmonary Vascular Disease in Moderate to Severe CF
• Scientific title: Evaluation and Treatment of Pulmonary Vascular Disease in Moderate to Severe Cystic Fibrosis Lung Disease
• Date of first enrolment: December 2015
• Target sample size: 14
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02626182
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Jennifer L Taylor-Cousar, MD
• Affiliation: National Jewish Health

Key inclusion & exclusion criteria

Inclusion Criteria:

1. 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

2. Male or female patients = 18 years of age

3. FEV1 = 20% predicted and = 70% predicted (Hankinson)

4. 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

5. Ability to reproducibly perform spirometry (according to ATS criteria)

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

7. Willingness to maintain chronic CF medication schedule (e.g. alternating month inhaled
antibiotics)

Exclusion Criteria:

1. History of hypersensitivity to sildenafil

2. Use of an investigational agent within the 4-week period prior to Visit 1 (Day 0)

3. 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.

4. History of significant hepatic disease (AST or ALT > 5 times the upper limit of normal
at screening, documented biliary cirrhosis, or portal hypertension),

5. History of significant cardiovascular disease (history of aortic stenosis, coronary
artery disease, or life-threatening arrhythmia),

6. History of severe neurological disease (e.g. history of stroke),

7. History of severe hematologic disease (e.g. history of bleeding diathesis; current INR
> 2.0

8. History of severe ophthalmologic disease (e.g. history of retinal impairment or
non-arteritic ischemic optic neuritis)

9. History of severe renal impairment (creatinine >1.8 mg/dL.)

10. Inability to swallow pills

11. Previous organ transplantation

12. Use of concomitant nitrates, a-blocker, or Ca channel blocker (currently or within one
month of Visit 1)

13. 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

14. History of sputum or throat swab culture yielding Burkholderia cepacia or
Mycobacterium massiliense within 2 years of screening

15. Weight less than 40 kg at Screening

16. History of migraine headaches.

17. Resting room air oxygen saturation <80% without supplemental oxygen

18. 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

19. Start of CFTR modulator therapy less than 1 month prior to first dose of sildenafil or
placebo

20. Use of anticoagulants

21. Frank pulmonary hypertension (RVSP >40 mmHg by ECHO)



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: placebo

Drug: sildenafil

Primary Outcome(s)

6 Minute Walk Distance [Time Frame: Weeks 1, 13]

Cardiopulmonary Exercise Test Work Rate [Time Frame: Weeks 1 and 13]

Secondary Outcome(s)

Cystic Fibrosis Quality of Life-Revised Respiratory Domain Score [Time Frame: Assessed at weeks 1 and 13]

Secondary ID(s)

NationalJewishHealth

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 15/07/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02626182