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

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ClinicalTrials.gov Identifier: NCT02626182
Recruitment Status : Completed
First Posted : December 10, 2015
Results First Posted : July 15, 2019
Last Update Posted : July 24, 2019
Sponsor:
Information provided by (Responsible Party):
Jennifer Taylor-Cousar, National Jewish Health

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Cystic Fibrosis
Interventions Drug: sildenafil
Drug: placebo
Enrollment 14
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Sildenafil Placebo
Hide Arm/Group Description

Subjects will be randomized in a 3:1 (sildenafil:placebo) fashion. Subjects randomized to the treatment arm will receive sildenafil 20 mg p.o. t.i.d for 1 week followed by 40 mg p.o. t.i.d. for 11 weeks.

sildenafil: active sildenafil

Subjects randomized to the placebo arm will receive placebo p.o. t.i.d for 1 week followed by 2 placebo tablets p.o. t.i.d. for 11 weeks.

placebo: sugar pill that looks like sildenafil tablets

Period Title: Overall Study
Started 11 3
Completed 9 3
Not Completed 2 0
Reason Not Completed
Adverse Event             2             0
Arm/Group Title Sildenafil Placebo Total
Hide Arm/Group Description

Subjects will be randomized in a 3:1 (sildenafil:placebo) fashion. Subjects randomized to the treatment arm will receive sildenafil 20 mg p.o. t.i.d for 1 week followed by 40 mg p.o. t.i.d. for 11 weeks.

sildenafil: active sildenafil

Subjects randomized to the placebo arm will receive placebo p.o. t.i.d for 1 week followed by 2 placebo tablets p.o. t.i.d. for 11 weeks.

placebo: sugar pill that looks like sildenafil tablets

Total of all reporting groups
Overall Number of Baseline Participants 9 3 12
Hide Baseline Analysis Population Description
All patients who completed all 12 weeks of study drug or placebo are included in the baseline analysis.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 9 participants 3 participants 12 participants
32.5  (10.6) 31.3  (1.5) 32.2  (9.4)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants 3 participants 12 participants
Female
4
  44.4%
2
  66.7%
6
  50.0%
Male
5
  55.6%
1
  33.3%
6
  50.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 9 participants 3 participants 12 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
White
9
 100.0%
3
 100.0%
12
 100.0%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 9 participants 3 participants 12 participants
9 3 0
1.Primary Outcome
Title 6 Minute Walk Distance
Hide Description Change in distance walked between week 1 and week 13 were compared. The difference between the two time points is reported.
Time Frame Weeks 1, 13
Hide Outcome Measure Data
Hide Analysis Population Description
All subjects who received 12 weeks of sildenafil 40 mg po tid or placebo po tid with the exception of 1 extreme outlier in the placebo group who was excluded.
Arm/Group Title Sildenafil Placebo
Hide Arm/Group Description:
Subjects who received sildenafil 40 mg po tid
Subjects who received placebo for 12 weeks
Overall Number of Participants Analyzed 9 2
Mean (Standard Deviation)
Unit of Measure: meters
25.2  (18.8) 0.75  (5.44)
2.Primary Outcome
Title Cardiopulmonary Exercise Test Work Rate
Hide Description Work rate (the amount of energy being expended to cycle) was assessed at weeks 1 and 13. The change in maximum work measured during CPET between weeks 1 and 13 is reported.
Time Frame Weeks 1 and 13
Hide Outcome Measure Data
Hide Analysis Population Description
Patients who received sildenafil 40 mg po tid or placebo po tid for 12 weeks
Arm/Group Title Sildenafil Placebo
Hide Arm/Group Description:
Subjects who received sildenafil 40 mg po tid
Subjects who received placebo for 12 weeks
Overall Number of Participants Analyzed 9 3
Mean (Standard Deviation)
Unit of Measure: watts
-0.20  (2.96) -0.27  (4.15)
3.Secondary Outcome
Title Cystic Fibrosis Quality of Life-Revised Respiratory Domain Score
Hide Description The CFQ-R Respiratory domain score (scale 0-100 with higher scores indicating better quality of life) was assessed at weeks 1 and 13. The change in the score between week 1 and week 13 is reported.
Time Frame Assessed at weeks 1 and 13
Hide Outcome Measure Data
Hide Analysis Population Description
Subjects who received sildenafil 40 mg po tid or placebo tid for 12 weeks
Arm/Group Title Sildenafil Placebo
Hide Arm/Group Description:
Subjects who received sildenafil 40 mg po tid
Subjects who received placebo for 12 weeks
Overall Number of Participants Analyzed 9 3
Mean (Standard Deviation)
Unit of Measure: units on a scale
8.62  (18.23) -9.23  (3.23)
Time Frame Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Sildenafil Placebo
Hide Arm/Group Description

Subjects will be randomized in a 3:1 (sildenafil:placebo) fashion. Subjects randomized to the treatment arm will receive sildenafil 20 mg p.o. t.i.d for 1 week followed by 40 mg p.o. t.i.d. for 11 weeks.

sildenafil: active sildenafil

Subjects randomized to the placebo arm will receive placebo p.o. t.i.d for 1 week followed by 2 placebo tablets p.o. t.i.d. for 11 weeks.

placebo: sugar pill that looks like sildenafil tablets

All-Cause Mortality
Sildenafil Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/9 (0.00%)      0/3 (0.00%)    
Hide Serious Adverse Events
Sildenafil Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/9 (22.22%)      0/3 (0.00%)    
Reproductive system and breast disorders     
Pulmonary exacerbation *  2/9 (22.22%)  2 0/3 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Sildenafil Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   9/9 (100.00%)      3/3 (100.00%)    
Eye disorders     
Conjuctivitis * [1]  1/9 (11.11%)  1 0/3 (0.00%)  0
Gastrointestinal disorders     
Stomach discomfort * [2]  2/9 (22.22%)  2 0/3 (0.00%)  0
Diarrhea *  1/9 (11.11%)  1 0/3 (0.00%)  0
General disorders     
Sinus congestion * [3]  6/9 (66.67%)  6 1/3 (33.33%)  1
Epistaxis *  1/9 (11.11%)  2 0/3 (0.00%)  0
Fever *  2/9 (22.22%)  2 0/3 (0.00%)  0
Tooth pain * [4]  1/9 (11.11%)  1 0/3 (0.00%)  0
Fatigue *  1/9 (11.11%)  1 0/3 (0.00%)  0
Infections and infestations     
Shingles *  1/9 (11.11%)  1 0/3 (0.00%)  0
Musculoskeletal and connective tissue disorders     
Musculoskeletal pain * [5]  2/9 (22.22%)  7 0/3 (0.00%)  0
Nervous system disorders     
Parasthesia * [6]  1/9 (11.11%)  2 0/3 (0.00%)  0
Renal and urinary disorders     
Dysuria * [7]  1/9 (11.11%)  1 0/3 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Cough/congestion *  4/9 (44.44%)  4 3/3 (100.00%)  3
Increased sputum *  2/9 (22.22%)  2 0/3 (0.00%)  0
Hemoptysis *  1/9 (11.11%)  1 0/3 (0.00%)  0
Wheeze *  1/9 (11.11%)  1 0/3 (0.00%)  0
Chest pain *  1/9 (11.11%)  1 0/3 (0.00%)  0
Skin and subcutaneous tissue disorders     
Flushing * [8]  2/9 (22.22%)  2 0/3 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
[1]
red eyes
[2]
Heartburn/stomach upset
[3]
Sinus congestion
[4]
pericoronitis
[5]
Pain
[6]
Numbness/tingling
[7]
UTI
[8]
Facial flushing
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Jennifer Taylor-Cousar, MD, MSCS, ATSF
Organization: National Jewish Health
Phone: 3032702764
EMail: Taylor-CousarJ@NJHealth.org
Publications:
Layout table for additonal information
Responsible Party: Jennifer Taylor-Cousar, National Jewish Health
ClinicalTrials.gov Identifier: NCT02626182    
Other Study ID Numbers: NationalJewishHealth
First Submitted: December 3, 2015
First Posted: December 10, 2015
Results First Submitted: May 8, 2019
Results First Posted: July 15, 2019
Last Update Posted: July 24, 2019