Trial Outcomes & Findings for Safety and Efficacy of Sildenafil in Cystic Fibrosis (CF) Lung Disease (NCT NCT00659529)

NCT ID: NCT00659529

Last Updated: 2020-10-19

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 36 participants
• Primary outcome timeframe: Pre/post therapy
• Results posted on: 2020-10-19

Participant Flow

Participant milestones

Measure	Open-label (All Subjects) 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. sildenafil: Sildenafil will be given 20mg po tid for 1 week, and then will be give 40mg po tid for 5 weeks.
Overall Study STARTED	36
Overall Study Received at Least One Dose of Study Drug	27
Overall Study COMPLETED	20
Overall Study NOT COMPLETED	16

Reasons for withdrawal

Measure	Open-label (All Subjects) 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. sildenafil: Sildenafil will be given 20mg po tid for 1 week, and then will be give 40mg po tid for 5 weeks.
Overall Study Withdrawal by Subject	4
Overall Study Protocol Violation	3
Overall Study Screen failure	9

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	Open-label (All Subjects) n=27 Participants 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. sildenafil: Sildenafil will be given 20mg po tid for 1 week, and then will be give 40mg po tid for 5 weeks.
Age, Continuous	25.7 years STANDARD_DEVIATION 5.5 • n=93 Participants
Sex: Female, Male Female	16 Participants n=93 Participants
Sex: Female, Male Male	11 Participants n=93 Participants
FEV1% predicted	77.5 % predicted STANDARD_DEVIATION 19.6 • n=93 Participants

PRIMARY outcome

Timeframe: Pre/post therapy

Population: Subjects who completed 6 weeks of sildenafil and had available data for pre/post 6 weeks of sildenafil were analyzed. One subject presented to the final study visit with 1 week of previously unreported symptoms consistent with pulmonary exacerbation, and therefore, efficacy data was not analyzed on that subject as pre-specified in the protocol.

Outcome measures

Measure	Open-label (All Subjects) n=18 Participants 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. sildenafil: Sildenafil will be given 20mg po tid for 1 week, and then will be give 40mg po tid for 5 weeks.
Sputum Elastase	-57 micrograms/mL Interval -119.0 to -18.0

SECONDARY outcome

Timeframe: Pre/post therapy

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Pre/post therapy

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Pre/during therapy

Outcome measures

Outcome data not reported

Adverse Events

Open-label (All Subjects)

Serious events: 2 serious events

Other events: 24 other events

Deaths: 0 deaths

Serious adverse events

Measure	Open-label (All Subjects) n=27 participants at risk 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. sildenafil: Sildenafil will be given 20mg po tid for 1 week, and then will be give 40mg po tid for 5 weeks.
Respiratory, thoracic and mediastinal disorders CF pulmonary exacerbation	7.4% 2/27 • Number of events 2
Gastrointestinal disorders Distal Intestinal Obstruction Syndrome	3.7% 1/27 • Number of events 1

Other adverse events

Measure	Open-label (All Subjects) n=27 participants at risk 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. sildenafil: Sildenafil will be given 20mg po tid for 1 week, and then will be give 40mg po tid for 5 weeks.
Nervous system disorders headache	22.2% 6/27
Respiratory, thoracic and mediastinal disorders rhinorhhea/congestion	33.3% 9/27
General disorders Sore throat/throat clearing	22.2% 6/27
Respiratory, thoracic and mediastinal disorders Increased cough	22.2% 6/27
General disorders Flushing	18.5% 5/27
Gastrointestinal disorders dyspepsia	14.8% 4/27
Musculoskeletal and connective tissue disorders myalgia	14.8% 4/27
Respiratory, thoracic and mediastinal disorders Increased/thicker sputum	14.8% 4/27
General disorders Insomnia	11.1% 3/27
Respiratory, thoracic and mediastinal disorders Chest tigntness/congestion	7.4% 2/27

Additional Information

Jennifer L. Taylor-Cousar, MD

National Jewish Health

Phone: (303) 270-2764

Email: Taylor-CousarJ@NJHealth.org

Results disclosure agreements

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