Healthy Adult Volunteers
Conditions
Brief summary
The purpose of this study is to evaluate the safety and tolerability of two dose levels of OP0201 and placebo, when administered intranasally in healthy adults subjects.
Interventions
COMBINATION_PRODUCTDrug: OP0201
Drug OP0201
COMBINATION_PRODUCTDrug: Placebo
Placebo
Sponsors
Novus Therapeutics, Inc
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 50 Years
Healthy volunteers
Yes
Inclusion criteria
includes but is not limited to: 1. Body mass index of 18 to 30 kg/m2 (inclusive) and a minimum body weight of 50 kg at Screening. 2. Able and willing to follow study instructions (including compliance with daily study treatment administration) and likely to complete all required study visits as assessed by the Investigator's judgement. 3. Female subjects must agree to use an acceptable method of contraception (for pregnancy considerations and contraceptive requirements). 4. Female subjects who are of childbearing potential must have a negative urine pregnancy test result at Screening and Day -1 prior to randomization. 5. Male subjects must agree to use contraception 6. Subjects must agree to refrain from immersing their head fully under water (eg, swimming, diving) from the time of signed informed consent until after the study exit visit. 7. Physiologic tympanogram classified as Type A (normal) by the Investigator or designee.
Exclusion criteria
includes but is not limited to: 1. History or presence of significant medical condition or a clinically significant abnormal finding, as determined by the Investigator. 2. Presence of a clinically significant abnormal olfactory test finding at Screening defined as a total UPSIT score \<35 (for females) and \<34 (for males). 3. Clinically significant ear disorder/disease currently or within 6 weeks prior to Screening. 4. History of tympanostomy tubes in one or both ears within 1 year prior to Screening. 5. Upper respiratory tract infection or pharyngitis currently or within 6 weeks prior to Screening. 6. Allergy or sinus conditions (eg, sinusitis, non-specific nasal inflammation) currently or within 6 weeks prior to Screening. 7. Clinically relevant blockage of one or both nasal passages, in the Investigator's opinion. 8. Gastroesophageal reflux disease currently or within 6 weeks prior to Screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants With of Adverse Events | 21 Days |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Evaluation of Systemic Exposure of DPPC: Observed DPPC Serum Concentration (µg/mL) by Nominal Timepoint | Day 1 and Day 14 | 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 |
| Evaluation of Systemic Exposure of CP: Observed CP Serum Concentrations (µM) by Nominal Timepoint | Day 1 and Day 14 | 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 |
| Evaluation of Systemic Exposure of DPPC: Baseline-corrected DPPC Serum Concentration (µg/mL) by Nominal Timepoint | Day 1 and Day 14 | 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 |
| Evaluation of Systemic Exposure of CP: Baseline-corrected CP Serum Concentrations (µM) by Nominal Timepoint | Day 1 and Day 14 | 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 |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Drug: OP0201 Cohort A 30 mg Per Day Cohort A- 30 mg per day X 14 days
Drug: OP0201: Drug OP0201 | 12 |
| Drug: OP0201 Cohort B 60 mg Per Day Cohort B-60 mg per day X 14 days
Drug: OP0201: Drug OP0201 | 12 |
| Drug: Placebo 0 mg per day X 14 days
Drug: Placebo: Placebo | 6 |
| Total | 30 |
Baseline characteristics
| Characteristic | Drug: Placebo | Total | Drug: OP0201 Cohort A 30 mg Per Day | Drug: OP0201 Cohort B 60 mg Per Day |
|---|---|---|---|---|
| Age, Continuous | 35.3 years STANDARD_DEVIATION 10.4 | 33.5 years STANDARD_DEVIATION 7.4 | 33.9 years STANDARD_DEVIATION 7.2 | 32.2 years STANDARD_DEVIATION 6.2 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 3 Participants | 16 Participants | 5 Participants | 8 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 3 Participants | 14 Participants | 7 Participants | 4 Participants |
| Region of Enrollment United States | 6 Participants | 30 Participants | 12 Participants | 12 Participants |
| Sex: Female, Male Female | 4 Participants | 12 Participants | 6 Participants | 2 Participants |
| Sex: Female, Male Male | 2 Participants | 18 Participants | 6 Participants | 10 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 12 | 0 / 12 | 0 / 6 |
| other Total, other adverse events | 5 / 12 | 9 / 12 | 4 / 6 |
| serious Total, serious adverse events | 0 / 12 | 0 / 12 | 0 / 6 |
Outcome results
Primary
Number of Participants With of Adverse Events
Time frame: 21 Days
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Drug: OP0201 Cohort A 30 mg Per Day | Number of Participants With of Adverse Events | 5 Participants |
| Drug: OP0201 Cohort B 60 mg Per Day | Number of Participants With of Adverse Events | 9 Participants |
| Drug: Placebo | Number of Participants With of Adverse Events | 4 Participants |
Secondary
Evaluation of Systemic Exposure of CP: Baseline-corrected CP Serum Concentrations (µM) by Nominal Timepoint
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
Time frame: Day 1 and Day 14
Population: 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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Drug: OP0201 Cohort A 30 mg Per Day | Evaluation of Systemic Exposure of CP: Baseline-corrected CP Serum Concentrations (µM) by Nominal Timepoint | 8.341 µM | Standard Deviation 15.478 |
| Drug: OP0201 Cohort B 60 mg Per Day | Evaluation of Systemic Exposure of CP: Baseline-corrected CP Serum Concentrations (µM) by Nominal Timepoint | 15.625 µM | Standard Deviation 18.781 |
| Drug: Placebo | Evaluation of Systemic Exposure of CP: Baseline-corrected CP Serum Concentrations (µM) by Nominal Timepoint | 6.697 µM | Standard Deviation 14.869 |
| Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 20 Mins | Evaluation of Systemic Exposure of CP: Baseline-corrected CP Serum Concentrations (µM) by Nominal Timepoint | 6.818 µM | Standard Deviation 13.839 |
| Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 35 Mins | Evaluation of Systemic Exposure of CP: Baseline-corrected CP Serum Concentrations (µM) by Nominal Timepoint | 5.699 µM | Standard Deviation 10.41 |
| Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 50 Mins | Evaluation of Systemic Exposure of CP: Baseline-corrected CP Serum Concentrations (µM) by Nominal Timepoint | 65.011 µM | Standard Deviation 52.626 |
| Drug: Placebo/ Day 1 Baseline | Evaluation of Systemic Exposure of CP: Baseline-corrected CP Serum Concentrations (µM) by Nominal Timepoint | 89.426 µM | Standard Deviation 74.425 |
| Drug: Placebo/ Day 14: 30 Mins Predose | Evaluation of Systemic Exposure of CP: Baseline-corrected CP Serum Concentrations (µM) by Nominal Timepoint | 64.957 µM | Standard Deviation 39.617 |
| Drug: Placebo/ Day 14: 5 Mins | Evaluation of Systemic Exposure of CP: Baseline-corrected CP Serum Concentrations (µM) by Nominal Timepoint | 67.834 µM | Standard Deviation 69.575 |
| Drug: Placebo/ Day 14: 20 Mins | Evaluation of Systemic Exposure of CP: Baseline-corrected CP Serum Concentrations (µM) by Nominal Timepoint | 69.641 µM | Standard Deviation 52.113 |
Secondary
Evaluation of Systemic Exposure of CP: Observed CP Serum Concentrations (µM) by Nominal Timepoint
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
Time frame: Day 1 and Day 14
Population: 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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Drug: OP0201 Cohort A 30 mg Per Day | Evaluation of Systemic Exposure of CP: Observed CP Serum Concentrations (µM) by Nominal Timepoint | 299.503 µM | Standard Deviation 61.999 |
| Drug: OP0201 Cohort B 60 mg Per Day | Evaluation of Systemic Exposure of CP: Observed CP Serum Concentrations (µM) by Nominal Timepoint | 290.103 µM | Standard Deviation 35.978 |
| Drug: Placebo | Evaluation of Systemic Exposure of CP: Observed CP Serum Concentrations (µM) by Nominal Timepoint | 293.473 µM | Standard Deviation 56.417 |
| Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 20 Mins | Evaluation of Systemic Exposure of CP: Observed CP Serum Concentrations (µM) by Nominal Timepoint | 279.852 µM | Standard Deviation 44.17 |
| Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 35 Mins | Evaluation of Systemic Exposure of CP: Observed CP Serum Concentrations (µM) by Nominal Timepoint | 279.289 µM | Standard Deviation 44.424 |
| Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 50 Mins | Evaluation of Systemic Exposure of CP: Observed CP Serum Concentrations (µM) by Nominal Timepoint | 276.788 µM | Standard Deviation 45.493 |
| Drug: Placebo/ Day 1 Baseline | Evaluation of Systemic Exposure of CP: Observed CP Serum Concentrations (µM) by Nominal Timepoint | 298.519 µM | Standard Deviation 126.157 |
| Drug: Placebo/ Day 14: 30 Mins Predose | Evaluation of Systemic Exposure of CP: Observed CP Serum Concentrations (µM) by Nominal Timepoint | 363.530 µM | Standard Deviation 178.783 |
| Drug: Placebo/ Day 14: 5 Mins | Evaluation of Systemic Exposure of CP: Observed CP Serum Concentrations (µM) by Nominal Timepoint | 387.945 µM | Standard Deviation 200.583 |
| Drug: Placebo/ Day 14: 20 Mins | Evaluation of Systemic Exposure of CP: Observed CP Serum Concentrations (µM) by Nominal Timepoint | 363.476 µM | Standard Deviation 165.774 |
| Drug: Placebo/ Day 14: 35 Mins | Evaluation of Systemic Exposure of CP: Observed CP Serum Concentrations (µM) by Nominal Timepoint | 366.353 µM | Standard Deviation 195.732 |
| Drug: Placebo/ Day 14: 50 Mins | Evaluation of Systemic Exposure of CP: Observed CP Serum Concentrations (µM) by Nominal Timepoint | 368.159 µM | Standard Deviation 178.27 |
Secondary
Evaluation of Systemic Exposure of DPPC: Baseline-corrected DPPC Serum Concentration (µg/mL) by Nominal Timepoint
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
Time frame: Day 1 and Day 14
Population: 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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Drug: OP0201 Cohort A 30 mg Per Day | Evaluation of Systemic Exposure of DPPC: Baseline-corrected DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 0.309 µg/mL | Standard Deviation 0.347 |
| Drug: OP0201 Cohort B 60 mg Per Day | Evaluation of Systemic Exposure of DPPC: Baseline-corrected DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 0.871 µg/mL | Standard Deviation 0.787 |
| Drug: Placebo | Evaluation of Systemic Exposure of DPPC: Baseline-corrected DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 0.383 µg/mL | Standard Deviation 0.453 |
| Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 20 Mins | Evaluation of Systemic Exposure of DPPC: Baseline-corrected DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 0.300 µg/mL | Standard Deviation 0.407 |
| Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 35 Mins | Evaluation of Systemic Exposure of DPPC: Baseline-corrected DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 0.447 µg/mL | Standard Deviation 0.446 |
| Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 50 Mins | Evaluation of Systemic Exposure of DPPC: Baseline-corrected DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 3.333 µg/mL | Standard Deviation 3.49 |
| Drug: Placebo/ Day 1 Baseline | Evaluation of Systemic Exposure of DPPC: Baseline-corrected DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 4.505 µg/mL | Standard Deviation 4.66 |
| Drug: Placebo/ Day 14: 30 Mins Predose | Evaluation of Systemic Exposure of DPPC: Baseline-corrected DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 3.678 µg/mL | Standard Deviation 3.85 |
| Drug: Placebo/ Day 14: 5 Mins | Evaluation of Systemic Exposure of DPPC: Baseline-corrected DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 3.278 µg/mL | Standard Deviation 4.274 |
| Drug: Placebo/ Day 14: 20 Mins | Evaluation of Systemic Exposure of DPPC: Baseline-corrected DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 3.865 µg/mL | Standard Deviation 4.009 |
Secondary
Evaluation of Systemic Exposure of DPPC: Observed DPPC Serum Concentration (µg/mL) by Nominal Timepoint
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
Time frame: Day 1 and Day 14
Population: 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.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Drug: OP0201 Cohort A 30 mg Per Day | Evaluation of Systemic Exposure of DPPC: Observed DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 10.05 µg/mL | Standard Deviation 2.585 |
| Drug: OP0201 Cohort B 60 mg Per Day | Evaluation of Systemic Exposure of DPPC: Observed DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 10.18 µg/mL | Standard Deviation 2.703 |
| Drug: Placebo | Evaluation of Systemic Exposure of DPPC: Observed DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 10.35 µg/mL | Standard Deviation 2.832 |
| Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 20 Mins | Evaluation of Systemic Exposure of DPPC: Observed DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 9.954 µg/mL | Standard Deviation 2.475 |
| Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 35 Mins | Evaluation of Systemic Exposure of DPPC: Observed DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 9.813 µg/mL | Standard Deviation 2.368 |
| Drug: OP0201 Cohort B 60 mg Per Day/ Day 14: 50 Mins | Evaluation of Systemic Exposure of DPPC: Observed DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 10.04 µg/mL | Standard Deviation 2.564 |
| Drug: Placebo/ Day 1 Baseline | Evaluation of Systemic Exposure of DPPC: Observed DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 9.525 µg/mL | Standard Deviation 2.652 |
| Drug: Placebo/ Day 14: 30 Mins Predose | Evaluation of Systemic Exposure of DPPC: Observed DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 12.86 µg/mL | Standard Deviation 6.145 |
| Drug: Placebo/ Day 14: 5 Mins | Evaluation of Systemic Exposure of DPPC: Observed DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 14.03 µg/mL | Standard Deviation 7.319 |
| Drug: Placebo/ Day 14: 20 Mins | Evaluation of Systemic Exposure of DPPC: Observed DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 13.20 µg/mL | Standard Deviation 6.505 |
| Drug: Placebo/ Day 14: 35 Mins | Evaluation of Systemic Exposure of DPPC: Observed DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 12.80 µg/mL | Standard Deviation 6.93 |
| Drug: Placebo/ Day 14: 50 Mins | Evaluation of Systemic Exposure of DPPC: Observed DPPC Serum Concentration (µg/mL) by Nominal Timepoint | 13.39 µg/mL | Standard Deviation 6.668 |