Trial Outcomes & Findings for Evaluation and Treatment of Pulmonary Vascular Disease in Moderate to Severe CF (NCT NCT02626182)

NCT ID: NCT02626182

Last Updated: 2019-07-24

Results Overview

Change in distance walked between week 1 and week 13 were compared. The difference between the two time points is reported.

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 14 participants
• Primary outcome timeframe: Weeks 1, 13
• Results posted on: 2019-07-24

Participant Flow

Participant milestones

Measure	Sildenafil 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	Placebo 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
Overall Study STARTED	11	3
Overall Study COMPLETED	9	3
Overall Study NOT COMPLETED	2	0

Reasons for withdrawal

Measure	Sildenafil 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	Placebo 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
Overall Study Adverse Event	2	0

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Sildenafil n=9 Participants 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	Placebo n=3 Participants 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 n=12 Participants Total of all reporting groups
Age, Continuous	32.5 years STANDARD_DEVIATION 10.6 • n=5 Participants	31.3 years STANDARD_DEVIATION 1.5 • n=7 Participants	32.2 years STANDARD_DEVIATION 9.4 • n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants	2 Participants n=7 Participants	6 Participants n=5 Participants
Sex: Female, Male Male	5 Participants n=5 Participants	1 Participants n=7 Participants	6 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	9 Participants n=5 Participants	3 Participants n=7 Participants	12 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	9 participants n=5 Participants	3 participants n=7 Participants	0 participants n=5 Participants

PRIMARY outcome

Timeframe: Weeks 1, 13

Population: 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.

Change in distance walked between week 1 and week 13 were compared. The difference between the two time points is reported.

Outcome measures

Measure	Sildenafil n=9 Participants Subjects who received sildenafil 40 mg po tid	Placebo n=2 Participants Subjects who received placebo for 12 weeks
6 Minute Walk Distance	25.2 meters Standard Deviation 18.8	0.75 meters Standard Deviation 5.44

PRIMARY outcome

Timeframe: Weeks 1 and 13

Population: Patients who received sildenafil 40 mg po tid or placebo po tid for 12 weeks

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.

Outcome measures

Measure	Sildenafil n=9 Participants Subjects who received sildenafil 40 mg po tid	Placebo n=3 Participants Subjects who received placebo for 12 weeks
Cardiopulmonary Exercise Test Work Rate	-0.20 watts Standard Deviation 2.96	-0.27 watts Standard Deviation 4.15

SECONDARY outcome

Timeframe: Assessed at weeks 1 and 13

Population: Subjects who received sildenafil 40 mg po tid or placebo tid for 12 weeks

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.

Outcome measures

Measure	Sildenafil n=9 Participants Subjects who received sildenafil 40 mg po tid	Placebo n=3 Participants Subjects who received placebo for 12 weeks
Cystic Fibrosis Quality of Life-Revised Respiratory Domain Score	8.62 units on a scale Standard Deviation 18.23	-9.23 units on a scale Standard Deviation 3.23

Adverse Events

Sildenafil

Serious events: 2 serious events

Other events: 9 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Measure	Sildenafil n=9 participants at risk 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	Placebo n=3 participants at risk 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
Reproductive system and breast disorders Pulmonary exacerbation	22.2% 2/9 • Number of events 2 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)

Other adverse events

Measure	Sildenafil n=9 participants at risk 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	Placebo n=3 participants at risk 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
General disorders Sinus congestion	66.7% 6/9 • Number of events 6 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	33.3% 1/3 • Number of events 1 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
Skin and subcutaneous tissue disorders Flushing	22.2% 2/9 • Number of events 2 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
Respiratory, thoracic and mediastinal disorders Cough/congestion	44.4% 4/9 • Number of events 4 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	100.0% 3/3 • Number of events 3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
General disorders Epistaxis	11.1% 1/9 • Number of events 2 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
Musculoskeletal and connective tissue disorders Musculoskeletal pain	22.2% 2/9 • Number of events 7 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
General disorders Fever	22.2% 2/9 • Number of events 2 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
Gastrointestinal disorders Stomach discomfort	22.2% 2/9 • Number of events 2 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
Gastrointestinal disorders Diarrhea	11.1% 1/9 • Number of events 1 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
Renal and urinary disorders Dysuria	11.1% 1/9 • Number of events 1 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
General disorders Tooth pain	11.1% 1/9 • Number of events 1 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
Eye disorders Conjuctivitis	11.1% 1/9 • Number of events 1 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
Respiratory, thoracic and mediastinal disorders Increased sputum	22.2% 2/9 • Number of events 2 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
Respiratory, thoracic and mediastinal disorders Hemoptysis	11.1% 1/9 • Number of events 1 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
Respiratory, thoracic and mediastinal disorders Wheeze	11.1% 1/9 • Number of events 1 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
General disorders Fatigue	11.1% 1/9 • Number of events 1 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
Respiratory, thoracic and mediastinal disorders Chest pain	11.1% 1/9 • Number of events 1 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
Infections and infestations Shingles	11.1% 1/9 • Number of events 1 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)
Nervous system disorders Parasthesia	11.1% 1/9 • Number of events 2 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)	0.00% 0/3 • Adverse events were collected from the time of signing of consent until 2 weeks after the subject's final clinic visit (approximately 5 months)

Additional Information

Jennifer Taylor-Cousar, MD, MSCS, ATSF

National Jewish Health

Phone: 3032702764

Email: Taylor-CousarJ@NJHealth.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place