Trial Outcomes & Findings for Study to Assess the Efficacy and Safety of Three Doses of PT001 in Japanese Subjects With Moderate to Severe COPD (NCT NCT03256552)
NCT ID: NCT03256552
Last Updated: 2018-01-23
Results Overview
Change from Baseline in Morning Pre-dose Trough FEV1
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
66 participants
Primary outcome timeframe
Baseline, Day 8
Results posted on
2018-01-23
Participant Flow
Conducted at 20 sites in Japan from January-September 2015. Entire period of study participation per subject was a maximum of 19 weeks. Planned target enrollment of 60 subjects.
This was a chronic dosing (7 days), 4-period, 4-treatment, placebo-controlled, crossover study. Each subject was randomized to 1 of 4 sequences. Each sequence included the four treatment groups. By-treatment sequence tabulations of the data were not pre-specified.
Unit of analysis: Participants
Participant milestones
| Measure |
Overall Study
All Subjects Randomized
|
|---|---|
|
Overall Study
STARTED
|
66 66
|
|
Overall Study
GP MDI 28.8 µg
|
61 61
|
|
Overall Study
GP MDI 14.4 µg
|
63 63
|
|
Overall Study
GP MDI 7.2 µg
|
62 62
|
|
Overall Study
Placebo MDI
|
65 65
|
|
Overall Study
COMPLETED
|
61 61
|
|
Overall Study
NOT COMPLETED
|
5 5
|
Reasons for withdrawal
| Measure |
Overall Study
All Subjects Randomized
|
|---|---|
|
Overall Study
Protocol Violation
|
1
|
|
Overall Study
Physician Decision
|
1
|
|
Overall Study
Adverse Event
|
1
|
|
Overall Study
Withdrawal by Subject
|
2
|
Baseline Characteristics
Study to Assess the Efficacy and Safety of Three Doses of PT001 in Japanese Subjects With Moderate to Severe COPD
Baseline characteristics by cohort
| Measure |
All Subjects
n=62 Participants
All Subjects in the MITT Population
|
|---|---|
|
Age, Continuous
|
67.5 Years
STANDARD_DEVIATION 7.0 • n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
59 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Day 8Population: MITT Population defined as all subjects who received treatment and had post-treatment efficacy data from at least two treatment periods.
Change from Baseline in Morning Pre-dose Trough FEV1
Outcome measures
| Measure |
GP MDI 28.8 µg
n=61 Participants
Glycopyrronium MDI 28.8 µg
|
GP MDI 14.4 µg
n=61 Participants
Glycopyrronium 14.4 µg
|
GP MDI 7.2 µg
n=62 Participants
Glycopyrronium 7.2 µg
|
Placebo MDI
n=61 Participants
Placebo MDI
|
|---|---|---|---|---|
|
Morning Pre-dose Trough FEV1
|
0.101 Liters
Interval 0.068 to 0.134
|
0.098 Liters
Interval 0.066 to 0.131
|
0.077 Liters
Interval 0.045 to 0.11
|
-0.031 Liters
Interval -0.064 to 0.002
|
SECONDARY outcome
Timeframe: Day 1 and Day 8Population: MITT Population defined as all subjects who received treatment and had post-treatment efficacy data from at least two treatment periods.
Change from Baseline in FEV1 AUC0-2 normalized for length of follow-up. FEV1 was measured at 15 min, 30 min, 1 hour, and 2 hours post dose.
Outcome measures
| Measure |
GP MDI 28.8 µg
n=61 Participants
Glycopyrronium MDI 28.8 µg
|
GP MDI 14.4 µg
n=61 Participants
Glycopyrronium 14.4 µg
|
GP MDI 7.2 µg
n=62 Participants
Glycopyrronium 7.2 µg
|
Placebo MDI
n=62 Participants
Placebo MDI
|
|---|---|---|---|---|
|
FEV1 AUC0-2
Day 1
|
0.176 Liters
Interval 0.146 to 0.205
|
0.140 Liters
Interval 0.111 to 0.17
|
0.127 Liters
Interval 0.098 to 0.157
|
0.043 Liters
Interval 0.014 to 0.073
|
|
FEV1 AUC0-2
Day 8
|
0.194 Liters
Interval 0.157 to 0.23
|
0.199 Liters
Interval 0.162 to 0.236
|
0.170 Liters
Interval 0.133 to 0.206
|
0.019 Liters
Interval -0.018 to 0.055
|
SECONDARY outcome
Timeframe: Day 1 and Day 8Population: MITT Population defined as all subjects who received treatment and had post-treatment efficacy data from at least two treatment periods.
Peak Change from Baseline in FEV1
Outcome measures
| Measure |
GP MDI 28.8 µg
n=61 Participants
Glycopyrronium MDI 28.8 µg
|
GP MDI 14.4 µg
n=61 Participants
Glycopyrronium 14.4 µg
|
GP MDI 7.2 µg
n=62 Participants
Glycopyrronium 7.2 µg
|
Placebo MDI
n=62 Participants
Placebo MDI
|
|---|---|---|---|---|
|
Peak Change in FEV1
Day 1
|
0.254 Liters
Interval 0.219 to 0.289
|
0.212 Liters
Interval 0.177 to 0.247
|
0.192 Liters
Interval 0.157 to 0.227
|
0.104 Liters
Interval 0.069 to 0.139
|
|
Peak Change in FEV1
Day 8
|
0.260 Liters
Interval 0.219 to 0.301
|
0.265 Liters
Interval 0.224 to 0.306
|
0.238 Liters
Interval 0.197 to 0.279
|
0.082 Liters
Interval 0.041 to 0.124
|
SECONDARY outcome
Timeframe: Baseline, Day 8Population: MITT Population defined as all subjects who received treatment and had post-treatment efficacy data from at least two treatment periods.
Change from Baseline in FVC AUC0-2 on Day 8 normalized for length of follow-up. FVC was measured at 15 min, 30 min, 1 hour, and 2 hours post dose.
Outcome measures
| Measure |
GP MDI 28.8 µg
n=61 Participants
Glycopyrronium MDI 28.8 µg
|
GP MDI 14.4 µg
n=61 Participants
Glycopyrronium 14.4 µg
|
GP MDI 7.2 µg
n=62 Participants
Glycopyrronium 7.2 µg
|
Placebo MDI
n=61 Participants
Placebo MDI
|
|---|---|---|---|---|
|
FVC AUC0-2
|
0.273 Liters
Interval 0.207 to 0.339
|
0.265 Liters
Interval 0.199 to 0.331
|
0.223 Liters
Interval 0.157 to 0.288
|
0.047 Liters
Interval -0.019 to 0.113
|
Adverse Events
GP MDI 28.8 µg
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
GP MDI 14.4 µg
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
GP MDI 7.2 µg
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Placebo MDI
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
GP MDI 28.8 µg
n=61 participants at risk
Glycopyrronium MDI 28.8 µg
|
GP MDI 14.4 µg
n=63 participants at risk
Glycopyrronium 14.4 µg
|
GP MDI 7.2 µg
n=62 participants at risk
Glycopyrronium 7.2 µg
|
Placebo MDI
n=65 participants at risk
Placebo MDI
|
|---|---|---|---|---|
|
Infections and infestations
Pneumonia
|
0.00%
0/61 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
0.00%
0/63 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
0.00%
0/62 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
1.5%
1/65 • Number of events 1 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
|
Respiratory, thoracic and mediastinal disorders
Chronic Obstructive Pulmonary Disease
|
0.00%
0/61 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
0.00%
0/63 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
0.00%
0/62 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
1.5%
1/65 • Number of events 1 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
Other adverse events
| Measure |
GP MDI 28.8 µg
n=61 participants at risk
Glycopyrronium MDI 28.8 µg
|
GP MDI 14.4 µg
n=63 participants at risk
Glycopyrronium 14.4 µg
|
GP MDI 7.2 µg
n=62 participants at risk
Glycopyrronium 7.2 µg
|
Placebo MDI
n=65 participants at risk
Placebo MDI
|
|---|---|---|---|---|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/61 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
3.2%
2/63 • Number of events 2 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
1.6%
1/62 • Number of events 1 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
1.5%
1/65 • Number of events 1 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
|
Skin and subcutaneous tissue disorders
Rash
|
3.3%
2/61 • Number of events 2 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
0.00%
0/63 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
0.00%
0/62 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
0.00%
0/65 • Adverse events were collected from the time the subject signed consent up to the follow-up visit 7-14 days after last dose of study drug.
Safety population included all participants who received at least one dose of investigational drug and had safety data available; participants were included in safety population according to the investigational drug received
|
Additional Information
Colin Reisner, MD, FCCP, FAAAAI
Pearl Therapeutics, Inc
Phone: 650-305-2600
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Drafts of any and all publications or presentations of this study must be submitted at least 30 days prior to submission for publication or presentation to Pearl Therapeutics for review, approval, and to ensure consistency. Pearl Therapeutics has the right to request appropriate modification to correct facts and to represent its opinions, or the opinions of the publication committee, if these differ with the proposed publication.
- Publication restrictions are in place
Restriction type: OTHER