Trial Outcomes & Findings for Evaluation of Safety of Repeated Doses of OP0201 Metered Dose Inhaler Compared to Placebo in Healthy Adult Volunteers (NCT NCT03748758)
NCT ID: NCT03748758
Last Updated: 2020-02-25
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE1
Target enrollment
30 participants
Primary outcome timeframe
21 Days
Results posted on
2020-02-25
Participant Flow
Participant milestones
| Measure |
Drug: OP0201 Cohort A 30 mg Per Day
Cohort A- 30 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day
Cohort B-60 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: Placebo
0 mg per day X 14 days
Drug: Placebo: Placebo
|
|---|---|---|---|
|
Overall Study
STARTED
|
12
|
12
|
6
|
|
Overall Study
COMPLETED
|
12
|
12
|
5
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Evaluation of Safety of Repeated Doses of OP0201 Metered Dose Inhaler Compared to Placebo in Healthy Adult Volunteers
Baseline characteristics by cohort
| Measure |
Drug: OP0201 Cohort A 30 mg Per Day
n=12 Participants
Cohort A- 30 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day
n=12 Participants
Cohort B-60 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: Placebo
n=6 Participants
0 mg per day X 14 days
Drug: Placebo: Placebo
|
Total
n=30 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
33.9 years
STANDARD_DEVIATION 7.2 • n=5 Participants
|
32.2 years
STANDARD_DEVIATION 6.2 • n=7 Participants
|
35.3 years
STANDARD_DEVIATION 10.4 • n=5 Participants
|
33.5 years
STANDARD_DEVIATION 7.4 • n=4 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
12 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
18 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 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
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
5 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
16 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
14 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
12 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
30 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 21 DaysOutcome measures
| Measure |
Drug: OP0201 Cohort A 30 mg Per Day
n=12 Participants
Cohort A- 30 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day
n=12 Participants
Cohort B-60 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: Placebo
n=6 Participants
0 mg per day X 14 days
Drug: Placebo: Placebo
|
Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 20 Mins
Cohort B-60 mg per day X 14 days Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 35 Mins
Cohort B-60 mg per day X 14 days Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 50 Mins
Cohort B-60 mg per day X 14 days Drug: OP0201: Drug OP0201
|
Drug: Placebo/ Day 1 Baseline
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 30 Mins Predose
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 5 Mins
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 20 Mins
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 35 Mins
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 50 Mins
0 mg per day X 14 days Drug: Placebo: Placebo
|
|---|---|---|---|---|---|---|---|---|---|---|---|---|
|
Number of Participants With of Adverse Events
|
5 Participants
|
9 Participants
|
4 Participants
|
—
|
—
|
—
|
—
|
—
|
—
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: Day 1 and Day 14Population: It is pre-specified to collect and report data for only "Cohort B" and "Placebo" Arms/Groups per protocol. The Number of participants analyzed for Cohort B at Day 14, 30 mins pre-dose and 5 mins post-dose is one less compared to other timepoints due to exclusion of serum samples that had missing collection times.
This will be evaluated by collecting blood on Day 1 baseline, Day 14 at 30 minutes pre-dose, and 5, 20, 35, and 50 minutes post-dose only in OP0201 Cohort B 60 mg per day
Outcome measures
| Measure |
Drug: OP0201 Cohort A 30 mg Per Day
n=12 Participants
Cohort A- 30 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day
n=11 Participants
Cohort B-60 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: Placebo
n=11 Participants
0 mg per day X 14 days
Drug: Placebo: Placebo
|
Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 20 Mins
n=12 Participants
Cohort B-60 mg per day X 14 days Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 35 Mins
n=12 Participants
Cohort B-60 mg per day X 14 days Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 50 Mins
n=12 Participants
Cohort B-60 mg per day X 14 days Drug: OP0201: Drug OP0201
|
Drug: Placebo/ Day 1 Baseline
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 30 Mins Predose
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 5 Mins
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 20 Mins
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 35 Mins
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 50 Mins
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
|---|---|---|---|---|---|---|---|---|---|---|---|---|
|
Evaluation of Systemic Exposure of DPPC: Observed DPPC Serum Concentration (µg/mL) by Nominal Timepoint
|
10.05 µg/mL
Standard Deviation 2.585
|
10.18 µg/mL
Standard Deviation 2.703
|
10.35 µg/mL
Standard Deviation 2.832
|
9.954 µg/mL
Standard Deviation 2.475
|
9.813 µg/mL
Standard Deviation 2.368
|
10.04 µg/mL
Standard Deviation 2.564
|
9.525 µg/mL
Standard Deviation 2.652
|
12.86 µg/mL
Standard Deviation 6.145
|
14.03 µg/mL
Standard Deviation 7.319
|
13.20 µg/mL
Standard Deviation 6.505
|
12.80 µg/mL
Standard Deviation 6.930
|
13.39 µg/mL
Standard Deviation 6.668
|
SECONDARY outcome
Timeframe: Day 1 and Day 14Population: It is pre-specified to collect and report data for only "Cohort B" and "Placebo" Arms/Groups per protocol. The Number of participants analyzed for Cohort B at Day 14, 30 mins pre-dose and 5 mins post-dose is one less compared to other timepoints due to exclusion of serum samples that had missing collection times.
This will be evaluated by collecting blood on Day 1 baseline, Day 14 at 30 minutes pre-dose, and 5, 20, 35, and 50 minutes post-dose only in OP0201 Cohort B 60 mg per day
Outcome measures
| Measure |
Drug: OP0201 Cohort A 30 mg Per Day
n=12 Participants
Cohort A- 30 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day
n=11 Participants
Cohort B-60 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: Placebo
n=11 Participants
0 mg per day X 14 days
Drug: Placebo: Placebo
|
Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 20 Mins
n=12 Participants
Cohort B-60 mg per day X 14 days Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 35 Mins
n=12 Participants
Cohort B-60 mg per day X 14 days Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 50 Mins
n=12 Participants
Cohort B-60 mg per day X 14 days Drug: OP0201: Drug OP0201
|
Drug: Placebo/ Day 1 Baseline
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 30 Mins Predose
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 5 Mins
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 20 Mins
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 35 Mins
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 50 Mins
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
|---|---|---|---|---|---|---|---|---|---|---|---|---|
|
Evaluation of Systemic Exposure of CP: Observed CP Serum Concentrations (µM) by Nominal Timepoint
|
299.503 µM
Standard Deviation 61.999
|
290.103 µM
Standard Deviation 35.978
|
293.473 µM
Standard Deviation 56.417
|
279.852 µM
Standard Deviation 44.170
|
279.289 µM
Standard Deviation 44.424
|
276.788 µM
Standard Deviation 45.493
|
298.519 µM
Standard Deviation 126.157
|
363.530 µM
Standard Deviation 178.783
|
387.945 µM
Standard Deviation 200.583
|
363.476 µM
Standard Deviation 165.774
|
366.353 µM
Standard Deviation 195.732
|
368.159 µM
Standard Deviation 178.270
|
SECONDARY outcome
Timeframe: Day 1 and Day 14Population: It is pre-specified to collect and report data for only "Cohort B" and "Placebo" Arms/Groups per protocol. The Number of participants analyzed for Cohort B at Day 14, 30 mins pre-dose and 5 mins post-dose is one less compared to other timepoints due to exclusion of serum samples that had missing collection times.
This will be evaluated by collecting blood on Day 1 baseline, Day 14 at 30 minutes pre-dose, and 5, 20, 35, and 50 minutes post-dose only in OP0201 Cohort B 60 mg per day
Outcome measures
| Measure |
Drug: OP0201 Cohort A 30 mg Per Day
n=11 Participants
Cohort A- 30 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day
n=11 Participants
Cohort B-60 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: Placebo
n=12 Participants
0 mg per day X 14 days
Drug: Placebo: Placebo
|
Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 20 Mins
n=12 Participants
Cohort B-60 mg per day X 14 days Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 35 Mins
n=12 Participants
Cohort B-60 mg per day X 14 days Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 50 Mins
n=2 Participants
Cohort B-60 mg per day X 14 days Drug: OP0201: Drug OP0201
|
Drug: Placebo/ Day 1 Baseline
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 30 Mins Predose
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 5 Mins
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 20 Mins
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 35 Mins
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 50 Mins
0 mg per day X 14 days Drug: Placebo: Placebo
|
|---|---|---|---|---|---|---|---|---|---|---|---|---|
|
Evaluation of Systemic Exposure of DPPC: Baseline-corrected DPPC Serum Concentration (µg/mL) by Nominal Timepoint
|
0.309 µg/mL
Standard Deviation 0.347
|
0.871 µg/mL
Standard Deviation 0.787
|
0.383 µg/mL
Standard Deviation 0.453
|
0.300 µg/mL
Standard Deviation 0.407
|
0.447 µg/mL
Standard Deviation 0.446
|
3.333 µg/mL
Standard Deviation 3.490
|
4.505 µg/mL
Standard Deviation 4.660
|
3.678 µg/mL
Standard Deviation 3.850
|
3.278 µg/mL
Standard Deviation 4.274
|
3.865 µg/mL
Standard Deviation 4.009
|
—
|
—
|
SECONDARY outcome
Timeframe: Day 1 and Day 14Population: It is pre-specified to collect and report data for only "Cohort B" and "Placebo" Arms/Groups per protocol. The Number of participants analyzed for Cohort B at Day 14, 30 mins pre-dose and 5 mins post-dose is one less compared to other timepoints due to exclusion of serum samples that had missing collection times.
This will be evaluated by collecting blood on Day 1 baseline, Day 14 at 30 minutes pre-dose, and 5, 20, 35, and 50 minutes post-dose only in OP0201 Cohort B 60 mg per day
Outcome measures
| Measure |
Drug: OP0201 Cohort A 30 mg Per Day
n=11 Participants
Cohort A- 30 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day
n=11 Participants
Cohort B-60 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: Placebo
n=12 Participants
0 mg per day X 14 days
Drug: Placebo: Placebo
|
Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 20 Mins
n=12 Participants
Cohort B-60 mg per day X 14 days Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 35 Mins
n=12 Participants
Cohort B-60 mg per day X 14 days Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 50 Mins
n=2 Participants
Cohort B-60 mg per day X 14 days Drug: OP0201: Drug OP0201
|
Drug: Placebo/ Day 1 Baseline
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 30 Mins Predose
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 5 Mins
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 20 Mins
n=2 Participants
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 35 Mins
0 mg per day X 14 days Drug: Placebo: Placebo
|
Drug: Placebo/ Day 14: 50 Mins
0 mg per day X 14 days Drug: Placebo: Placebo
|
|---|---|---|---|---|---|---|---|---|---|---|---|---|
|
Evaluation of Systemic Exposure of CP: Baseline-corrected CP Serum Concentrations (µM) by Nominal Timepoint
|
8.341 µM
Standard Deviation 15.478
|
15.625 µM
Standard Deviation 18.781
|
6.697 µM
Standard Deviation 14.869
|
6.818 µM
Standard Deviation 13.839
|
5.699 µM
Standard Deviation 10.410
|
65.011 µM
Standard Deviation 52.626
|
89.426 µM
Standard Deviation 74.425
|
64.957 µM
Standard Deviation 39.617
|
67.834 µM
Standard Deviation 69.575
|
69.641 µM
Standard Deviation 52.113
|
—
|
—
|
Adverse Events
Drug: OP0201 Cohort A 30 mg Per Day
Serious events: 0 serious events
Other events: 5 other events
Deaths: 0 deaths
Drug: OP0201 Cohort B 60 mg Per Day
Serious events: 0 serious events
Other events: 9 other events
Deaths: 0 deaths
Drug: Placebo
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Drug: OP0201 Cohort A 30 mg Per Day
n=12 participants at risk
Cohort A- 30 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: OP0201 Cohort B 60 mg Per Day
n=12 participants at risk
Cohort B-60 mg per day X 14 days
Drug: OP0201: Drug OP0201
|
Drug: Placebo
n=6 participants at risk
0 mg per day X 14 days
Drug: Placebo: Placebo
|
|---|---|---|---|
|
Ear and labyrinth disorders
Tinnitus
|
0.00%
0/12 • End of study (Day 21)
|
0.00%
0/12 • End of study (Day 21)
|
16.7%
1/6 • End of study (Day 21)
|
|
Gastrointestinal disorders
Toothache
|
8.3%
1/12 • End of study (Day 21)
|
0.00%
0/12 • End of study (Day 21)
|
0.00%
0/6 • End of study (Day 21)
|
|
General disorders
Feeling cold
|
0.00%
0/12 • End of study (Day 21)
|
8.3%
1/12 • End of study (Day 21)
|
0.00%
0/6 • End of study (Day 21)
|
|
Infections and infestations
Nasopharyngitis
|
8.3%
1/12 • End of study (Day 21)
|
0.00%
0/12 • End of study (Day 21)
|
0.00%
0/6 • End of study (Day 21)
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/12 • End of study (Day 21)
|
8.3%
1/12 • End of study (Day 21)
|
0.00%
0/6 • End of study (Day 21)
|
|
Investigations
Olfactory test abnormal
|
0.00%
0/12 • End of study (Day 21)
|
33.3%
4/12 • End of study (Day 21)
|
33.3%
2/6 • End of study (Day 21)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
8.3%
1/12 • End of study (Day 21)
|
0.00%
0/12 • End of study (Day 21)
|
0.00%
0/6 • End of study (Day 21)
|
|
Nervous system disorders
Headache
|
25.0%
3/12 • End of study (Day 21)
|
0.00%
0/12 • End of study (Day 21)
|
16.7%
1/6 • End of study (Day 21)
|
|
Nervous system disorders
Paraesthesia
|
0.00%
0/12 • End of study (Day 21)
|
8.3%
1/12 • End of study (Day 21)
|
0.00%
0/6 • End of study (Day 21)
|
|
Nervous system disorders
Somnolence
|
8.3%
1/12 • End of study (Day 21)
|
0.00%
0/12 • End of study (Day 21)
|
0.00%
0/6 • End of study (Day 21)
|
|
Respiratory, thoracic and mediastinal disorders
Nasal discomfort
|
8.3%
1/12 • End of study (Day 21)
|
16.7%
2/12 • End of study (Day 21)
|
16.7%
1/6 • End of study (Day 21)
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/12 • End of study (Day 21)
|
8.3%
1/12 • End of study (Day 21)
|
0.00%
0/6 • End of study (Day 21)
|
|
Respiratory, thoracic and mediastinal disorders
Nasal dryness
|
8.3%
1/12 • End of study (Day 21)
|
0.00%
0/12 • End of study (Day 21)
|
0.00%
0/6 • End of study (Day 21)
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
0.00%
0/12 • End of study (Day 21)
|
8.3%
1/12 • End of study (Day 21)
|
0.00%
0/6 • End of study (Day 21)
|
|
Respiratory, thoracic and mediastinal disorders
Throat irritation
|
0.00%
0/12 • End of study (Day 21)
|
0.00%
0/12 • End of study (Day 21)
|
16.7%
1/6 • End of study (Day 21)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee No information, data or results shall be used by the Institution, Investigator or any other individual or entity other than the Sponsor. The use of Study Results by any party other than Sponsor requires prior written approval by the Sponsor.
- Publication restrictions are in place
Restriction type: OTHER