Trial Outcomes & Findings for A 48-week Study of the Effect of Dual Therapy (Inhaled Treprostinil and Tadafafil) Versus Monotherapy (Tadalafil). (NCT NCT01305252)
NCT ID: NCT01305252
Last Updated: 2017-06-02
Results Overview
Effect of dual-upfront therapies versus mono-therapy on percent change of right ventricular function assesed by cardiac MRI (cMRI) at 24 weeks compared with the baseline.
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
21 participants
Primary outcome timeframe
Basline and 24 weeks
Results posted on
2017-06-02
Participant Flow
Participant milestones
| Measure |
Tadalafil Alone
tadalafil 40mg QD
tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
Tadalafil and Treprostinil Inhalations
inhaled treprostinil: Treprostinil inhalation QID starting at 3 breaths per inhalation \& gradually increasing to 9 breaths. Each breath provides approximately 6mcg of treprostinil.
tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
|---|---|---|
|
Overall Study
STARTED
|
10
|
11
|
|
Overall Study
COMPLETED
|
9
|
11
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A 48-week Study of the Effect of Dual Therapy (Inhaled Treprostinil and Tadafafil) Versus Monotherapy (Tadalafil).
Baseline characteristics by cohort
| Measure |
Tadalafil Alone
n=10 Participants
tadalafil 40mg QD
tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
Tadalafil and Treprostinil Inhalations
n=11 Participants
inhaled treprostinil: Treprostinil inhalation QID starting at 3 breaths per inhalation \& gradually increasing to 9 breaths. Each breath provides approximately 6mcg of treprostinil.
tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
Total
n=21 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
10 Participants
n=99 Participants
|
11 Participants
n=107 Participants
|
21 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Continuous
|
51.6 years
STANDARD_DEVIATION 11.4 • n=99 Participants
|
52.1 years
STANDARD_DEVIATION 9.7 • n=107 Participants
|
51.9 years
STANDARD_DEVIATION 10.3 • n=206 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=99 Participants
|
9 Participants
n=107 Participants
|
16 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Basline and 24 weeksEffect of dual-upfront therapies versus mono-therapy on percent change of right ventricular function assesed by cardiac MRI (cMRI) at 24 weeks compared with the baseline.
Outcome measures
| Measure |
Tadalafil and Treprostinil Inhalations
n=11 Participants
Inhaled treprostinil: Treprostinil inhalation QID starting at 3 breaths per inhalation \& gradually increasing to 9 breaths. Each breath provides approximately 6mcg of treprostinil.
Tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
Tadalafil Alone
n=10 Participants
Tadalafil 40mg QD
Tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
|---|---|---|
|
Change in Right Ventricular Ejection Fraction
|
7.45 percent change
Interval 0.95 to 13.96
|
2.8 percent change
Interval -1.49 to 7.09
|
SECONDARY outcome
Timeframe: Baseline and 24 weeksChange in 6MWD during 24 week period compared between Tada and Tada+iTre.
Outcome measures
| Measure |
Tadalafil and Treprostinil Inhalations
n=10 Participants
Inhaled treprostinil: Treprostinil inhalation QID starting at 3 breaths per inhalation \& gradually increasing to 9 breaths. Each breath provides approximately 6mcg of treprostinil.
Tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
Tadalafil Alone
n=11 Participants
Tadalafil 40mg QD
Tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
|---|---|---|
|
6 Minute Walk Distance
|
82.63 meters
Interval 23.4 to 141.86
|
87.69 meters
Interval 6.98 to 168.41
|
SECONDARY outcome
Timeframe: Baseline and 24 weeksChange from baseline in N-terminal Pro B-type Natriuretic Peptide (NT-proBNP)
Outcome measures
| Measure |
Tadalafil and Treprostinil Inhalations
n=11 Participants
Inhaled treprostinil: Treprostinil inhalation QID starting at 3 breaths per inhalation \& gradually increasing to 9 breaths. Each breath provides approximately 6mcg of treprostinil.
Tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
Tadalafil Alone
n=10 Participants
Tadalafil 40mg QD
Tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
|---|---|---|
|
N-terminal Pro B-type Natriuretic Peptide (NT-proBNP)
|
1239 pg/mL
Standard Deviation 1393
|
988.4 pg/mL
Standard Deviation 827.6
|
SECONDARY outcome
Timeframe: Baseline and 48 weekAt 48 week,WHO/NYHA functional class was assessed for change in WHO/NYHA functional class.Change NYHA is measured as decrease or increase in NYHA class in the subjects compared with baseline. NYHA /WHO functional class is described below: NYHA functional class I:no symptoms and no limitation in ordinary physical activity NYHA functional class II:Mild symptoms (mild shortness of breath and/or angina) and slight limitation during ordinary activity NYHA functional class III:Marked limitation in activity due to symptoms, even during less-than-ordinary activity NYHA functional class IV:Severe limitations. Experiences symptoms even while at rest A higher functional class represent worse symptoms.
Outcome measures
| Measure |
Tadalafil and Treprostinil Inhalations
n=10 Participants
Inhaled treprostinil: Treprostinil inhalation QID starting at 3 breaths per inhalation \& gradually increasing to 9 breaths. Each breath provides approximately 6mcg of treprostinil.
Tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
Tadalafil Alone
n=11 Participants
Tadalafil 40mg QD
Tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
|---|---|---|
|
Change in NYHA/WHO Class
|
6 participants
|
11 participants
|
SECONDARY outcome
Timeframe: Baseline and 24 weeksB-type Natriuretic peptide measures the percent change from baseline.
Outcome measures
| Measure |
Tadalafil and Treprostinil Inhalations
n=10 Participants
Inhaled treprostinil: Treprostinil inhalation QID starting at 3 breaths per inhalation \& gradually increasing to 9 breaths. Each breath provides approximately 6mcg of treprostinil.
Tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
Tadalafil Alone
n=11 Participants
Tadalafil 40mg QD
Tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
|---|---|---|
|
B-type Natriuretic Peptide (BNP)
|
374 percent change
Standard Deviation 133.8
|
295.6 percent change
Standard Deviation 133.2
|
Adverse Events
Tadalafil Alone
Serious events: 1 serious events
Other events: 9 other events
Deaths: 0 deaths
Tadalafil and Treprostinil Inhalations
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Tadalafil Alone
n=10 participants at risk
tadalafil 40mg QD
tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
Tadalafil and Treprostinil Inhalations
n=11 participants at risk
inhaled treprostinil: Treprostinil inhalation QID starting at 3 breaths per inhalation \& gradually increasing to 9 breaths. Each breath provides approximately 6mcg of treprostinil.
tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
|---|---|---|
|
Cardiac disorders
Death
|
10.0%
1/10 • Number of events 1 • initial 24 weeks
|
0.00%
0/11 • initial 24 weeks
|
Other adverse events
| Measure |
Tadalafil Alone
n=10 participants at risk
tadalafil 40mg QD
tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
Tadalafil and Treprostinil Inhalations
n=11 participants at risk
inhaled treprostinil: Treprostinil inhalation QID starting at 3 breaths per inhalation \& gradually increasing to 9 breaths. Each breath provides approximately 6mcg of treprostinil.
tadalafil: tadalafil 20mg QD PO increasing to 40mg QD as tolerated
|
|---|---|---|
|
General disorders
Nasal congestion/allergic Rhinitis
|
20.0%
2/10 • initial 24 weeks
|
45.5%
5/11 • initial 24 weeks
|
|
Infections and infestations
Upper respiratory tract infection
|
20.0%
2/10 • initial 24 weeks
|
27.3%
3/11 • initial 24 weeks
|
|
Musculoskeletal and connective tissue disorders
musculoskeletal pain
|
10.0%
1/10 • initial 24 weeks
|
27.3%
3/11 • initial 24 weeks
|
|
Infections and infestations
Dental infection
|
30.0%
3/10 • initial 24 weeks
|
0.00%
0/11 • initial 24 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
10.0%
1/10 • initial 24 weeks
|
0.00%
0/11 • initial 24 weeks
|
|
Gastrointestinal disorders
Diarrhea
|
10.0%
1/10 • initial 24 weeks
|
0.00%
0/11 • initial 24 weeks
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
0.00%
0/10 • initial 24 weeks
|
9.1%
1/11 • initial 24 weeks
|
|
General disorders
Edema
|
0.00%
0/10 • initial 24 weeks
|
9.1%
1/11 • initial 24 weeks
|
Additional Information
Roham Zamanian, MD, FCCP
Stanford University School of Medicine
Phone: 650- 725-5495
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place