Trial Outcomes & Findings for Patient Global Impression Questions for Activity-Induced Symptoms in Participants With PAH (NCT NCT03888365)
NCT ID: NCT03888365
Last Updated: 2021-03-16
Results Overview
A global index that consists of 3 questions to which participants will rate the severity of 1) their PAH symptoms, 2) shortness of breath (SOB), and 3) fatigue. The minimum and maximum range of scores for each of the 3 individual questions was 1-5, with severity scale choices of 1=Not present, 2=Mild, 3=Moderate, 4=Severe, and 5=Very Severe. A higher score indicated worse outcome. The Baseline is defined as the average respective severity rating measured at 15 minutes and 0 minute prior to the ISWT. In the case of a single, missing severity rating measured pre-ISWT, the other non-missing single severity rating would be used as baseline value. Only participants with both a measurement at baseline and at the given post-baseline visit are summarized. Change from Baseline = Post-Baseline value - Baseline value. Peak levels are the highest concentrations of a drug in plasma.
COMPLETED
43 participants
Baseline, Approximately 30 m of previous dose of inhaled treprostinil (the expected peak level) on Day 1
2021-03-16
Participant Flow
The study included a 14-day Screening Period and Day 1 clinic visit. Participants performed a Incremental Shuttle Walk Test (ISWT) to induce symptoms of Pulmonary Arterial Hypertension (PAH). The ISWT required participants to walk back and forth on a 10-meter course. Total number of shuttles completed by a participant during the Screening ISWT was the maximum targeted for the participant for remaining study ISWTs. Study also included 2 periods/cohort. There was ≥1-h rest period between ISWTs.
The 2 periods for Cohort A: an ISWT initiated at 30 minutes (m) of previous dose (the expected peak level period) and an ISWT initiated at 3-4 hours (h) of previous dose of inhaled treprostinil (the expected trough level period). The 2 periods for Cohort B: an ISWT initiated at \~4h after morning dose of PAH medication (Period 1) and an ISWT initiated at ≥1h following completion of previous ISWT (Period 2). Peak and trough levels are the highest and lowest concentrations of a drug in plasma.
Participant milestones
| Measure |
Cohort A: Treprostinil
Participants who were currently prescribed and using inhaled treprostinil for the treatment of PAH.
|
Cohort B: Non-Treprostinil PAH Medications
Participants who were taking other PAH medications (instead of inhaled treprostinil).
|
|---|---|---|
|
Cohort A Peak Level/Cohort B Period 1
STARTED
|
20
|
23
|
|
Cohort A Peak Level/Cohort B Period 1
Safety Population
|
20
|
23
|
|
Cohort A Peak Level/Cohort B Period 1
COMPLETED
|
20
|
23
|
|
Cohort A Peak Level/Cohort B Period 1
NOT COMPLETED
|
0
|
0
|
|
Rest - At Least 1 h
STARTED
|
20
|
23
|
|
Rest - At Least 1 h
COMPLETED
|
20
|
23
|
|
Rest - At Least 1 h
NOT COMPLETED
|
0
|
0
|
|
Cohort A Trough Level/Cohort B Period 2
STARTED
|
20
|
23
|
|
Cohort A Trough Level/Cohort B Period 2
COMPLETED
|
20
|
23
|
|
Cohort A Trough Level/Cohort B Period 2
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Patient Global Impression Questions for Activity-Induced Symptoms in Participants With PAH
Baseline characteristics by cohort
| Measure |
Cohort A: Treprostinil
n=20 Participants
Participants who were currently prescribed and using inhaled treprostinil for the treatment of PAH.
|
Cohort B: Non-Treprostinil PAH Medications
n=23 Participants
Participants who were taking other PAH medications (instead of inhaled treprostinil).
|
Total
n=43 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
61.2 years
STANDARD_DEVIATION 8.69 • n=5 Participants
|
55.9 years
STANDARD_DEVIATION 13.14 • n=7 Participants
|
58.3 years
STANDARD_DEVIATION 11.48 • n=5 Participants
|
|
Sex: Female, Male
Female
|
16 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
33 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
19 Participants
n=5 Participants
|
21 Participants
n=7 Participants
|
40 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 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
|
5 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
14 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
32 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
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Approximately 30 m of previous dose of inhaled treprostinil (the expected peak level) on Day 1Population: All Participants: Participants who consented to the study protocol. Data for participants in Cohort A who included an ISWT initiated at 30 m of previous dose (expected peak level) are presented.
A global index that consists of 3 questions to which participants will rate the severity of 1) their PAH symptoms, 2) shortness of breath (SOB), and 3) fatigue. The minimum and maximum range of scores for each of the 3 individual questions was 1-5, with severity scale choices of 1=Not present, 2=Mild, 3=Moderate, 4=Severe, and 5=Very Severe. A higher score indicated worse outcome. The Baseline is defined as the average respective severity rating measured at 15 minutes and 0 minute prior to the ISWT. In the case of a single, missing severity rating measured pre-ISWT, the other non-missing single severity rating would be used as baseline value. Only participants with both a measurement at baseline and at the given post-baseline visit are summarized. Change from Baseline = Post-Baseline value - Baseline value. Peak levels are the highest concentrations of a drug in plasma.
Outcome measures
| Measure |
Cohort A: Treprostinil
n=20 Participants
Participants who were currently prescribed and using inhaled treprostinil for the treatment of PAH.
|
Cohort B: Non-Treprostinil PAH Medications
Participants who were taking other PAH medications (instead of inhaled treprostinil).
|
|---|---|---|
|
Change From Baseline in Patient Global Impression of Severity (PGI-S) Score in Cohort A Participants at Approximately 30 Minutes of Previous Dose of Inhaled Treprostinil (the Expected Peak Level) on Day 1
PAH, Baseline
|
1.25 scores on a scale
Standard Deviation 0.47
|
—
|
|
Change From Baseline in Patient Global Impression of Severity (PGI-S) Score in Cohort A Participants at Approximately 30 Minutes of Previous Dose of Inhaled Treprostinil (the Expected Peak Level) on Day 1
PAH, Change at 30 m of previous dose of inhaled treprostinil (expected peak level) on Day 1
|
1.65 scores on a scale
Standard Deviation 0.80
|
—
|
|
Change From Baseline in Patient Global Impression of Severity (PGI-S) Score in Cohort A Participants at Approximately 30 Minutes of Previous Dose of Inhaled Treprostinil (the Expected Peak Level) on Day 1
SOB, Baseline
|
1.15 scores on a scale
Standard Deviation 0.33
|
—
|
|
Change From Baseline in Patient Global Impression of Severity (PGI-S) Score in Cohort A Participants at Approximately 30 Minutes of Previous Dose of Inhaled Treprostinil (the Expected Peak Level) on Day 1
SOB, Change at 30 m of previous dose of inhaled treprostinil (expected peak level) on Day 1
|
1.90 scores on a scale
Standard Deviation 0.80
|
—
|
|
Change From Baseline in Patient Global Impression of Severity (PGI-S) Score in Cohort A Participants at Approximately 30 Minutes of Previous Dose of Inhaled Treprostinil (the Expected Peak Level) on Day 1
Fatigue, Baseline
|
1.30 scores on a scale
Standard Deviation 0.55
|
—
|
|
Change From Baseline in Patient Global Impression of Severity (PGI-S) Score in Cohort A Participants at Approximately 30 Minutes of Previous Dose of Inhaled Treprostinil (the Expected Peak Level) on Day 1
Fatigue, Change at 30 m of previous dose of inhaled treprostinil (expected peak level) on Day 1
|
1.40 scores on a scale
Standard Deviation 0.95
|
—
|
PRIMARY outcome
Timeframe: Baseline, Approximately 3-4 h of previous dose of inhaled treprostinil (the expected trough level) on Day 1Population: All Participants: Participants who consented to the study protocol. Data for participants in Cohort A who included an ISWT initiated at 3-4 h of previous dose of inhaled treprostinil (expected trough level) are presented.
A global index that consists of 3 questions to which participants will rate the severity of 1) their PAH symptoms, 2) SOB, and 3) fatigue. The minimum and maximum range of scores for each of the 3 individual questions was 1-5, with severity scale choices of 1=Not present, 2=Mild, 3=Moderate, 4=Severe, and 5=Very Severe. A higher score indicated worse outcome. The Baseline is defined as the average respective severity rating measured at 15 minutes and 0 minute prior to the ISWT. In the case of a single, missing severity rating measured pre-ISWT, the other non-missing single severity rating would be used as baseline value. Only participants with both a measurement at baseline and at the given post-baseline visit are summarized. Change from Baseline = Post-Baseline value - Baseline value. Trough levels are the lowest concentrations of a drug in plasma.
Outcome measures
| Measure |
Cohort A: Treprostinil
n=20 Participants
Participants who were currently prescribed and using inhaled treprostinil for the treatment of PAH.
|
Cohort B: Non-Treprostinil PAH Medications
Participants who were taking other PAH medications (instead of inhaled treprostinil).
|
|---|---|---|
|
Change From Baseline in PGI-S Score in Cohort A Participants at Approximately 3-4 Hours of Previous Dose of Inhaled Treprostinil (the Expected Trough Level) on Day 1
PAH, Baseline
|
1.10 scores on a scale
Standard Deviation 0.31
|
—
|
|
Change From Baseline in PGI-S Score in Cohort A Participants at Approximately 3-4 Hours of Previous Dose of Inhaled Treprostinil (the Expected Trough Level) on Day 1
PAH, Change at 3-4 h of previous dose of inhaled treprostinil (expected trough level) on Day 1
|
1.75 scores on a scale
Standard Deviation 0.79
|
—
|
|
Change From Baseline in PGI-S Score in Cohort A Participants at Approximately 3-4 Hours of Previous Dose of Inhaled Treprostinil (the Expected Trough Level) on Day 1
SOB, Baseline
|
1.10 scores on a scale
Standard Deviation 0.31
|
—
|
|
Change From Baseline in PGI-S Score in Cohort A Participants at Approximately 3-4 Hours of Previous Dose of Inhaled Treprostinil (the Expected Trough Level) on Day 1
SOB, Change at 3-4 h of previous dose of inhaled treprostinil (expected trough level) on Day 1
|
1.85 scores on a scale
Standard Deviation 0.93
|
—
|
|
Change From Baseline in PGI-S Score in Cohort A Participants at Approximately 3-4 Hours of Previous Dose of Inhaled Treprostinil (the Expected Trough Level) on Day 1
Fatigue, Baseline
|
1.25 scores on a scale
Standard Deviation 0.44
|
—
|
|
Change From Baseline in PGI-S Score in Cohort A Participants at Approximately 3-4 Hours of Previous Dose of Inhaled Treprostinil (the Expected Trough Level) on Day 1
Fatigue, Change at 3-4 h of previous dose of inhaled treprostinil (expected trough level) on Day 1
|
1.50 scores on a scale
Standard Deviation 1.15
|
—
|
PRIMARY outcome
Timeframe: Baseline, Approximately 4 h after morning dose of non-treprostinil PAH medication (Period 1) on Day 1Population: All Participants: Participants who consented to the study protocol. Data for participants in Cohort B who included an ISWT initiated at 4 h after morning dose of non-treprostinil PAH medication (Period 1) on Day 1 are presented.
A global index that consists of 3 questions to which participants will rate the severity of 1) their PAH symptoms, 2) SOB, and 3) fatigue. The minimum and maximum range of scores for each of the 3 individual questions was 1-5, with severity scale choices of 1=Not present, 2=Mild, 3=Moderate, 4=Severe, and 5=Very Severe. A higher score indicated worse outcome. The Baseline is defined as the average respective severity rating measured at 15 minutes and 0 minute prior to the ISWT. In the case of a single, missing severity rating measured pre-ISWT, the other non-missing single severity rating would be used as baseline value. Only participants with both a measurement at baseline and at the given post-baseline visit are summarized. Change from Baseline = Post-Baseline value - Baseline value.
Outcome measures
| Measure |
Cohort A: Treprostinil
n=23 Participants
Participants who were currently prescribed and using inhaled treprostinil for the treatment of PAH.
|
Cohort B: Non-Treprostinil PAH Medications
Participants who were taking other PAH medications (instead of inhaled treprostinil).
|
|---|---|---|
|
Change From Baseline in PGI-S Score in Cohort B Participants at Approximately 4 Hours After Morning Dose of Non-Treprostinil PAH Medication (Period 1) on Day 1
PAH, Baseline
|
1.22 scores on a scale
Standard Deviation 0.39
|
—
|
|
Change From Baseline in PGI-S Score in Cohort B Participants at Approximately 4 Hours After Morning Dose of Non-Treprostinil PAH Medication (Period 1) on Day 1
PAH, Change at 4 h after morning dose of non-treprostinil PAH medication (Period 1) on Day 1
|
1.74 scores on a scale
Standard Deviation 0.90
|
—
|
|
Change From Baseline in PGI-S Score in Cohort B Participants at Approximately 4 Hours After Morning Dose of Non-Treprostinil PAH Medication (Period 1) on Day 1
SOB, Baseline
|
1.17 scores on a scale
Standard Deviation 0.36
|
—
|
|
Change From Baseline in PGI-S Score in Cohort B Participants at Approximately 4 Hours After Morning Dose of Non-Treprostinil PAH Medication (Period 1) on Day 1
SOB, Change at 4 h after morning dose of non-treprostinil PAH medication (Period 1) on Day 1
|
1.78 scores on a scale
Standard Deviation 0.78
|
—
|
|
Change From Baseline in PGI-S Score in Cohort B Participants at Approximately 4 Hours After Morning Dose of Non-Treprostinil PAH Medication (Period 1) on Day 1
Fatigue, Baseline
|
1.17 scores on a scale
Standard Deviation 0.36
|
—
|
|
Change From Baseline in PGI-S Score in Cohort B Participants at Approximately 4 Hours After Morning Dose of Non-Treprostinil PAH Medication (Period 1) on Day 1
Fatigue, Change at 4 h after morning dose of non-treprostinil PAH medication (Period 1) on Day 1
|
1.61 scores on a scale
Standard Deviation 1.07
|
—
|
PRIMARY outcome
Timeframe: Baseline, At least 1 h following completion of previous ISWT (Period 2) on Day 1Population: All Participants: Participants who consented to the study protocol. Data for participants in Cohort B with an ISWT initiated at least 1 h following completion of previous ISWT (Period 2) are presented.
A global index that consists of 3 questions to which participants will rate the severity of 1) their PAH symptoms, 2) SOB, and 3) fatigue. The minimum and maximum range of scores for each of the 3 individual questions was 1-5, with severity scale choices of 1=Not present, 2=Mild, 3=Moderate, 4=Severe, and 5=Very Severe. A higher score indicated worse outcome. The Baseline is defined as the average respective severity rating measured at 15 minutes and 0 minute prior to the ISWT. In the case of a single, missing severity rating measured pre-ISWT, the other non-missing single severity rating would be used as baseline value. Only participants with both a measurement at baseline and at the given post-baseline visit are summarized. Change from Baseline = Post-Baseline value - Baseline value.
Outcome measures
| Measure |
Cohort A: Treprostinil
n=23 Participants
Participants who were currently prescribed and using inhaled treprostinil for the treatment of PAH.
|
Cohort B: Non-Treprostinil PAH Medications
Participants who were taking other PAH medications (instead of inhaled treprostinil).
|
|---|---|---|
|
Change From Baseline in PGI-S Scores in Cohort B Participants With an ISWT at Least 1 h Following Completion of Previous ISWT (Period 2) on Day 1
PAH, Baseline
|
1.13 scores on a scale
Standard Deviation 0.34
|
—
|
|
Change From Baseline in PGI-S Scores in Cohort B Participants With an ISWT at Least 1 h Following Completion of Previous ISWT (Period 2) on Day 1
PAH, Change at least 1 h following completion of previous ISWT (Period 2) on Day 1
|
1.87 scores on a scale
Standard Deviation 0.97
|
—
|
|
Change From Baseline in PGI-S Scores in Cohort B Participants With an ISWT at Least 1 h Following Completion of Previous ISWT (Period 2) on Day 1
SOB, Baseline
|
1.04 scores on a scale
Standard Deviation 0.21
|
—
|
|
Change From Baseline in PGI-S Scores in Cohort B Participants With an ISWT at Least 1 h Following Completion of Previous ISWT (Period 2) on Day 1
SOB, Change at least 1 h following completion of previous ISWT (Period 2) on Day 1
|
2.13 scores on a scale
Standard Deviation 0.92
|
—
|
|
Change From Baseline in PGI-S Scores in Cohort B Participants With an ISWT at Least 1 h Following Completion of Previous ISWT (Period 2) on Day 1
Fatigue, Baseline
|
1.13 scores on a scale
Standard Deviation 0.34
|
—
|
|
Change From Baseline in PGI-S Scores in Cohort B Participants With an ISWT at Least 1 h Following Completion of Previous ISWT (Period 2) on Day 1
Fatigue, Change at least 1 h following completion of previous ISWT (Period 2) on Day 1
|
1.91 scores on a scale
Standard Deviation 1.04
|
—
|
SECONDARY outcome
Timeframe: Baseline, ~30 m of previous dose of inhaled treprostinil, ~3-4 h of previous dose of inhaled treprostinil, ~4 h after morning dose of non-treprostinil PAH medication, and ≥1 h following completion of previous ISWT on Day 1Population: All Participants: Participants who consented to the study protocol. Here, 'Number Analyzed' signifies participants evaluable at the specified timepoint.
The modified Borg scale allows participants to rate maximum level of dyspnea experienced during 6-Minute Walk Test (6MWT). Scores ranged from 0 (best condition) to 10 (worst condition). Baseline defined as average from Borg Dyspnea Scores measured at 15 and 0 m prior to ISWT. If a single pre-ISWT Borg Dyspnea Score was missing, the other nonmissing single score was used as baseline value. Change from Baseline=Post-Baseline value - Baseline value. Data for participants in Cohort A with an ISWT initiated at 30 m of previous dose (expected peak level) and an ISWT initiated at 3-4 h of previous dose of inhaled treprostinil (expected trough level) on Day 1 are presented. Data for participants in Cohort B with an ISWT initiated at 4 h after morning dose of non-treprostinil PAH medication (Period 1) and with an ISWT initiated at least 1 h following completion of previous ISWT (Period 2) on Day 1 are presented. Peak and trough levels are highest and lowest concentrations of a drug in plasma.
Outcome measures
| Measure |
Cohort A: Treprostinil
n=20 Participants
Participants who were currently prescribed and using inhaled treprostinil for the treatment of PAH.
|
Cohort B: Non-Treprostinil PAH Medications
n=23 Participants
Participants who were taking other PAH medications (instead of inhaled treprostinil).
|
|---|---|---|
|
Change From Baseline in Modified Borg Dyspnea Scores at Day 1
Expected peak level, Baseline
|
0.28 scores on a scale
Standard Deviation 0.54
|
—
|
|
Change From Baseline in Modified Borg Dyspnea Scores at Day 1
Change at 30 m of previous dose of inhaled treprostinil (expected peak level) on Day 1
|
3.00 scores on a scale
Standard Deviation 1.78
|
—
|
|
Change From Baseline in Modified Borg Dyspnea Scores at Day 1
Expected trough level, Baseline
|
0.23 scores on a scale
Standard Deviation 0.62
|
—
|
|
Change From Baseline in Modified Borg Dyspnea Scores at Day 1
Change at 3-4 h of previous dose of inhaled treprostinil (expected trough level) on Day 1
|
3.13 scores on a scale
Standard Deviation 2.16
|
—
|
|
Change From Baseline in Modified Borg Dyspnea Scores at Day 1
Period 1, Baseline
|
—
|
0.24 scores on a scale
Standard Deviation 0.50
|
|
Change From Baseline in Modified Borg Dyspnea Scores at Day 1
Change at 4 h after morning dose of non-treprostinil PAH medication (Period 1) on Day 1
|
—
|
2.85 scores on a scale
Standard Deviation 1.81
|
|
Change From Baseline in Modified Borg Dyspnea Scores at Day 1
Period 2, Baseline
|
—
|
0.23 scores on a scale
Standard Deviation 0.64
|
|
Change From Baseline in Modified Borg Dyspnea Scores at Day 1
Change at least 1 h following completion of previous ISWT (Period 2) on Day 1
|
—
|
3.03 scores on a scale
Standard Deviation 1.90
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, ~30 m of previous dose of inhaled treprostinil, ~3-4 h of previous dose of inhaled treprostinil, ~4 h after morning dose of non-treprostinil PAH medication, and ≥1 h following completion of previous ISWT on Day 1Population: All Participants: Participants who consented to the study protocol. Here, 'Number Analyzed' signifies participants evaluable at the specified timepoint
Pulse Oximetry includes the collection of saturation peripheral capillary oxygenation (SpO\_2). Baseline was defined as the average from pulse oximetry measured at 15 minutes and 0 minute prior to the ISWT. In the case of a single, missing pre-ISWT pulse oximetry, the other non-missing single measurement would be used as baseline value. Change from Baseline = Post-Baseline value - Baseline value. Data for participants in Cohort A who had an ISWT initiated at 30 m of previous dose (expected peak level) and an ISWT initiated at 3-4 h of previous dose of inhaled treprostinil (expected trough level) on Day 1 are presented. Additionally, data for participants in Cohort B who included an ISWT initiated at 4 h after morning dose of non-treprostinil PAH medication (Period 1) and with an ISWT initiated at least 1 h following completion of previous ISWT (Period 2) on Day 1 are presented. Peak and trough levels are the highest and lowest concentrations of a drug in plasma.
Outcome measures
| Measure |
Cohort A: Treprostinil
n=20 Participants
Participants who were currently prescribed and using inhaled treprostinil for the treatment of PAH.
|
Cohort B: Non-Treprostinil PAH Medications
n=23 Participants
Participants who were taking other PAH medications (instead of inhaled treprostinil).
|
|---|---|---|
|
Change From Baseline in Pulse Oximetry at Day 1
Expected peak level, Baseline
|
93.73 percent saturation (SpO_2)
Standard Deviation 2.94
|
—
|
|
Change From Baseline in Pulse Oximetry at Day 1
Change at 30 m of previous dose of inhaled treprostinil (expected peak level) on Day 1
|
-8.13 percent saturation (SpO_2)
Standard Deviation 6.68
|
—
|
|
Change From Baseline in Pulse Oximetry at Day 1
Expected trough level, Baseline
|
95.15 percent saturation (SpO_2)
Standard Deviation 3.12
|
—
|
|
Change From Baseline in Pulse Oximetry at Day 1
Change at 3-4 h of previous dose of inhaled treprostinil (expected trough level) on Day 1
|
-10.35 percent saturation (SpO_2)
Standard Deviation 9.35
|
—
|
|
Change From Baseline in Pulse Oximetry at Day 1
Period 1, Baseline
|
—
|
94.98 percent saturation (SpO_2)
Standard Deviation 3.24
|
|
Change From Baseline in Pulse Oximetry at Day 1
Change at 4 h after morning dose of non-treprostinil PAH medication (Period 1) on Day 1
|
—
|
-4.85 percent saturation (SpO_2)
Standard Deviation 5.13
|
|
Change From Baseline in Pulse Oximetry at Day 1
Period 2, Baseline
|
—
|
94.04 percent saturation (SpO_2)
Standard Deviation 3.14
|
|
Change From Baseline in Pulse Oximetry at Day 1
Change at least 1 h following completion of previous ISWT (Period 2) on Day 1
|
—
|
-2.91 percent saturation (SpO_2)
Standard Deviation 3.79
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline, ~30 m of previous dose of inhaled treprostinil, ~3-4 h of previous dose of inhaled treprostinil, ~4 h after morning dose of non-treprostinil PAH medication, and ≥1 h following completion of previous ISWT on Day 1Population: All Participants: Participants who consented to the study protocol. Here, 'Number Analyzed' signifies participants evaluable at the specified timepoint
Heart rate was captured as beats per minute (bpm). Baseline was defined as the average from pulse oximetry measured at 15 minutes and 0 minute prior to the Incremental Shuttle Walk Test (ISWT). In the case of a single, missing pre-ISWT pulse oximetry, the other non-missing single measurement would be used as baseline value. Change from Baseline = Post-Baseline value - Baseline value. Data for participants in Cohort A who had an ISWT initiated at 30 m of previous dose (expected peak level) and an ISWT initiated at 3-4 h of previous dose of inhaled treprostinil (expected trough level) on Day 1 are presented. Additionally, data for participants in Cohort B who included an ISWT initiated at 4 h after morning dose of non-treprostinil PAH medication (Period 1) and with an ISWT initiated at least 1 h following completion of previous ISWT (Period 2) on Day 1 are presented. Peak and trough levels are the highest and lowest concentrations of a drug in plasma.
Outcome measures
| Measure |
Cohort A: Treprostinil
n=20 Participants
Participants who were currently prescribed and using inhaled treprostinil for the treatment of PAH.
|
Cohort B: Non-Treprostinil PAH Medications
n=23 Participants
Participants who were taking other PAH medications (instead of inhaled treprostinil).
|
|---|---|---|
|
Change From Baseline in Heart Rate at Day 1
Period 1, Baseline
|
—
|
74.59 bpm
Standard Deviation 11.67
|
|
Change From Baseline in Heart Rate at Day 1
Expected peak level, Baseline
|
80.08 bpm
Standard Deviation 11.93
|
—
|
|
Change From Baseline in Heart Rate at Day 1
Change at 30 m of previous dose of inhaled treprostinil (expected peak level) on Day 1
|
24.48 bpm
Standard Deviation 18.10
|
—
|
|
Change From Baseline in Heart Rate at Day 1
Expected trough level, Baseline
|
75.58 bpm
Standard Deviation 10.78
|
—
|
|
Change From Baseline in Heart Rate at Day 1
Change at 3-4 h of previous dose of inhaled treprostinil (expected trough level) on Day 1
|
23.08 bpm
Standard Deviation 19.10
|
—
|
|
Change From Baseline in Heart Rate at Day 1
Change at 4 h after morning dose of non-treprostinil PAH medication (Period 1) on Day 1
|
—
|
31.28 bpm
Standard Deviation 13.67
|
|
Change From Baseline in Heart Rate at Day 1
Period 2, Baseline
|
—
|
75.26 bpm
Standard Deviation 12.04
|
|
Change From Baseline in Heart Rate at Day 1
Change at least 1 h following completion of previous ISWT (Period 2) on Day 1
|
—
|
33.39 bpm
Standard Deviation 18.08
|
Adverse Events
Cohort A: Treprostinil
Cohort B: Non-Treprostinil PAH Medications
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Cohort A: Treprostinil
n=20 participants at risk
Participants who were currently prescribed and using inhaled treprostinil for the treatment of PAH.
|
Cohort B: Non-Treprostinil PAH Medications
n=23 participants at risk
Participants who were taking other PAH medications (instead of inhaled treprostinil).
|
|---|---|---|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/20 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
4.3%
1/23 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/20 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
4.3%
1/23 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
|
General disorders
Chills
|
0.00%
0/20 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
4.3%
1/23 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
|
General disorders
Fatigue
|
0.00%
0/20 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
4.3%
1/23 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
|
General disorders
Non-cardiac chest pain
|
0.00%
0/20 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
4.3%
1/23 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
|
Infections and infestations
Bronchitis
|
0.00%
0/20 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
4.3%
1/23 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/20 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
8.7%
2/23 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/20 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
4.3%
1/23 • Baseline through Day 1 (up to at least 5 hours or at the Investigator's discretion)
Safety Population: All participants who initiated an ISWT at Screening.
|
Additional Information
Lung Biotechnology PBC Study Director
Lung Biotechnology PBC
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60