Trial Outcomes & Findings for Tadalafil for Sarcoidosis Associated Pulmonary Hypertension (NCT NCT01324999)

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE2/PHASE3

Target enrollment

12 participants

Primary outcome timeframe

Baseline, Week 8, Week 16, Week 24

Results posted on

2017-07-24

Participant Flow

Participant milestones

Participant milestones
Measure	Tadalafil 40 mg daily
Overall Study STARTED	12
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Reasons for withdrawal
Measure	Tadalafil 40 mg daily
Overall Study Adverse Event	3
Overall Study Withdrawal by Subject	2

Baseline Characteristics

Tadalafil for Sarcoidosis Associated Pulmonary Hypertension

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Tadalafil n=12 Participants 40 mg daily
Age, Continuous	54.5 years STANDARD_DEVIATION 9.48 • n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	9 Participants n=5 Participants
Race/Ethnicity, Customized White	3 Participants n=5 Participants
Region of Enrollment United States	12 participants n=5 Participants
FEV1	58.0 percent predicted STANDARD_DEVIATION 14.7 • n=5 Participants
FVC	65.9 percent predicted STANDARD_DEVIATION 13.4 • n=5 Participants
DLCO	60.3 percent predicted STANDARD_DEVIATION 25.0 • n=5 Participants
Right Atrial Pressure	6.2 mm Hg STANDARD_DEVIATION 4.0 • n=5 Participants
Mean Pulmonary Artery Pressure	34.8 mm Hg STANDARD_DEVIATION 7.1 • n=5 Participants
Pulmonary Artery Wedge Pressure	10.3 mm Hg STANDARD_DEVIATION 3.6 • n=5 Participants
Cardiac Index	2.4 L/min/m² STANDARD_DEVIATION 0.6 • n=5 Participants
Pulmonary Vascular Resistance	6.2 Wood Units STANDARD_DEVIATION 3.6 • n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 8, Week 16, Week 24

Population: Only 6 of the 7 study completers performed 6 minute walk test through week 24

Outcome measures

Outcome measures
Measure	Tadalafil n=6 Participants 40 mg daily
6 Minute Walk Distance Baseline	419.1 meters Standard Deviation 170.1
6 Minute Walk Distance Week 8	418.1 meters Standard Deviation 143.5
6 Minute Walk Distance Week 16	436.5 meters Standard Deviation 178.1
6 Minute Walk Distance Week 24	377.1 meters Standard Deviation 140.9

SECONDARY outcome

Timeframe: Baseline, Week 24

Population: only 6 of the 7 study completers performed the 6 minute walk test through week 24.

Percent oxygen saturation of hemoglobin as measured by pulse oximetry in the resting state.

Outcome measures

Outcome measures
Measure	Tadalafil n=6 Participants 40 mg daily
Resting Oxygen Saturation Week 24	95.2 percent Standard Deviation 2.6
Resting Oxygen Saturation Baseline	96.5 percent Standard Deviation 1.8

SECONDARY outcome

Timeframe: Baseline, Week 24

Population: Only 6 of the 7 study completers performed the 6 minute walk test through week 24

Change (decrease) in oxygen saturation from start of 6 minute walk to nadir during the 6 minute walk test

Outcome measures

Outcome measures
Measure	Tadalafil n=6 Participants 40 mg daily
Oxygen Desaturation During 6 Minute Walk Test Baseline	9 oxygen saturation percentage points Standard Deviation 6
Oxygen Desaturation During 6 Minute Walk Test Week 24	9 oxygen saturation percentage points Standard Deviation 9

SECONDARY outcome

Timeframe: Baseline, Week 8, Week 16, Week 24

Population: Only 6 of the 7 study completers performed 6 minute walk test through the end of the study.

The modified Borg scale consists of a vertical scale labelled 0 to10 with corresponding verbal expressions of progressively increasing sensation (shortness of breath) intensity. 0 represents no dyspnea, and 10 is the highest level of perceived dyspnea.

Outcome measures

Outcome measures
Measure	Tadalafil n=6 Participants 40 mg daily
Maximum Borg Dyspnea Score During 6 Minute Walk Test Baseline	4.8 units on a scale Standard Deviation 1.2
Maximum Borg Dyspnea Score During 6 Minute Walk Test Week 24	6.3 units on a scale Standard Deviation 2.3
Maximum Borg Dyspnea Score During 6 Minute Walk Test Week 8	5.0 units on a scale Standard Deviation 2.4
Maximum Borg Dyspnea Score During 6 Minute Walk Test Week 16	5.3 units on a scale Standard Deviation 2.1

SECONDARY outcome

Timeframe: Baseline, Week 8, Week 16, Week 24

Outcome measures

Outcome measures
Measure	Tadalafil n=7 Participants 40 mg daily
Brain Natriuretic Peptide Level Baseline	58.8 pg/mL Standard Deviation 89.6
Brain Natriuretic Peptide Level Week 8	32.3 pg/mL Standard Deviation 46.6
Brain Natriuretic Peptide Level Week 16	37.5 pg/mL Standard Deviation 51.4
Brain Natriuretic Peptide Level Week 24	42.6 pg/mL Standard Deviation 49.7

SECONDARY outcome

Timeframe: Baseline, Week 8, Week 16, Week 24

SF- 36 investigates the standard of quality of life through a general health assessment and not specific to a particular disease, age or treatment group. It is a 36-item questionnaire measuring 8 domains (physical functioning, role physical, bodily pain, general health, vitality, social functioning, role emotional, and mental health). Each domain score ranges from 0 (worst) to 100 (best), with higher scores reflecting better health-related functional status. The global score is determined by taking an average of the individual domain scores. The global score range is from 0 (worst) to 100 (best).

Outcome measures

Outcome measures
Measure	Tadalafil n=7 Participants 40 mg daily
Short Form-36 Global Score Week 8	37.7 units on a scale Standard Deviation 6.7
Short Form-36 Global Score Week 16	39.2 units on a scale Standard Deviation 6.5
Short Form-36 Global Score Week 24	38.9 units on a scale Standard Deviation 8.5
Short Form-36 Global Score Baseline	38.5 units on a scale Standard Deviation 5.3

SECONDARY outcome

Timeframe: Baseline, Week 8, Week 16, Week 24

The SGRQ ranges from 0 (no impairment of quality of life) to 100 (highest impairment of quality of life)

Outcome measures

Outcome measures
Measure	Tadalafil n=7 Participants 40 mg daily
St. George's Respiratory Questionnaire (SGRQ) Score Baseline	64.5 units on a scale Standard Deviation 15.5
St. George's Respiratory Questionnaire (SGRQ) Score Week 8	65.1 units on a scale Standard Deviation 18.9
St. George's Respiratory Questionnaire (SGRQ) Score Week 16	64.3 units on a scale Standard Deviation 15.2
St. George's Respiratory Questionnaire (SGRQ) Score Week 24	71.1 units on a scale Standard Deviation 15.2

SECONDARY outcome

Timeframe: Baseline, Week 24

The WHO functional classification ranges from I (patient's disease does not affect daily activities) to IV (patient's disease causes severe impairment). Higher scores indicate more severe impairment.

Outcome measures

Outcome measures
Measure	Tadalafil n=7 Participants 40 mg daily
Number of Participants With Change in WHO Functional Class (WHO FC) Number of participants that improved to WHO FC I	1 participants
Number of Participants With Change in WHO Functional Class (WHO FC) Number of participants that improved to WHO FC II	0 participants
Number of Participants With Change in WHO Functional Class (WHO FC) Number of participants that improved to WHO FC III	0 participants

Adverse Events

Tadalafil

Serious events: 0 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Tadalafil n=12 participants at risk 40 mg daily
Nervous system disorders Headache	16.7% 2/12 • Number of events 2 • 24 weeks
Musculoskeletal and connective tissue disorders Joint pain	8.3% 1/12 • Number of events 1 • 24 weeks
Skin and subcutaneous tissue disorders Herpes zoster	8.3% 1/12 • Number of events 1 • 24 weeks
Respiratory, thoracic and mediastinal disorders Influenza/pneumonia	8.3% 1/12 • Number of events 1 • 24 weeks

Additional Information

H. James Ford, MD

University of North Carolina at Chapel Hill

Phone: (919) 966-2531

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place