Trial Outcomes & Findings for A Clinical Study to Measure the Effect of OP-101 After Being Administered Subcutaneous in Healthy Volunteers (NCT NCT04321980)

Results Overview

An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. A TEAE (treatment-emergent adverse event) was defined as an AE that emerges, having been absent prior to the study, or an AE that worsens in severity after the first dose of the study drug. Serious AE (SAE) was an AE resulting in any of the following outcomes: death; life-threatening adverse event, required hospitalization or prolongation of existing hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect, or a medically important event.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

8 participants

Primary outcome timeframe

Up to Day 15

Results posted on

2021-06-14

Participant Flow

A total of 8 participants were enrolled and treated in the study.

Participant milestones

Participant milestones
Measure	Cohort 1: 4 mg/kg Participants in Cohort 1 received a single dose of 4 milligram per kg (mg/kg) OP-101 as subcutaneous (SC) injection on Day 1.	Cohort 2: 8 mg/kg Participants in Cohort 2 received a single dose of 8 mg/kg OP-101 as SC injection on Day 1.
Overall Study STARTED	4	4
Overall Study COMPLETED	4	4
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Clinical Study to Measure the Effect of OP-101 After Being Administered Subcutaneous in Healthy Volunteers

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort 1: 4 mg/kg n=4 Participants Participants in Cohort 1 received a single dose of 4 milligram per kg (mg/kg) OP-101 as subcutaneous (SC) injection on Day 1.	Cohort 2: 8 mg/kg n=4 Participants Participants in Cohort 2 received a single dose of 8 mg/kg OP-101 as SC injection on Day 1.	Total n=8 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=93 Participants	4 Participants n=4 Participants	8 Participants n=27 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Sex: Female, Male Female	3 Participants n=93 Participants	4 Participants n=4 Participants	7 Participants n=27 Participants
Sex: Female, Male Male	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=93 Participants	4 Participants n=4 Participants	7 Participants n=27 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race/Ethnicity, Customized Asian	0 Participants n=93 Participants	1 Participants n=4 Participants	1 Participants n=27 Participants
Race/Ethnicity, Customized White	3 Participants n=93 Participants	3 Participants n=4 Participants	6 Participants n=27 Participants
Race/Ethnicity, Customized Filipino	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants

PRIMARY outcome

Timeframe: Up to Day 15

Population: Analysis was performed on safety population.

An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. A TEAE (treatment-emergent adverse event) was defined as an AE that emerges, having been absent prior to the study, or an AE that worsens in severity after the first dose of the study drug. Serious AE (SAE) was an AE resulting in any of the following outcomes: death; life-threatening adverse event, required hospitalization or prolongation of existing hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect, or a medically important event.

Outcome measures

Outcome measures
Measure	Cohort 1: 4 mg/kg n=4 Participants Participants in Cohort 1 received a single dose of 4 mg/kg OP-101 as SC injection on Day 1.	Cohort 2: 8 mg/kg n=4 Participants Participants in Cohort 2 received a single dose of 8 mg/kg OP-101 as SC injection on Day 1.
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events Any TEAE	2 Participants	3 Participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events Any SAE	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Pre-dose, 0.5 hours and 1, 2, 4, 6, 8, 10, 12, 16, 24, 30-36, 48 hours post-dose

Population: Analysis was performed on pharmacokinetic (PK) parameter population which was defined as all participants who received a dose of study drug and had at least 1 PK parameter.

Cmax: maximum observed plasma concentration.

Outcome measures

Outcome measures
Measure	Cohort 1: 4 mg/kg n=4 Participants Participants in Cohort 1 received a single dose of 4 mg/kg OP-101 as SC injection on Day 1.	Cohort 2: 8 mg/kg n=4 Participants Participants in Cohort 2 received a single dose of 8 mg/kg OP-101 as SC injection on Day 1.
Pharmacokinetics: Maximum Observed Plasma Concentration (Cmax) of OP-101	3.473 microgram per milliliter(mcg/mL) Standard Deviation 0.8183	6.640 microgram per milliliter(mcg/mL) Standard Deviation 0.6073

SECONDARY outcome

Timeframe: Pre-dose, 0.5 hours and at 1, 2, 4, 6, 8, 10, 12, 16, 24, 30-36, 48 hours post-dose

Population: Analysis was performed on PK parameter population.

Tmax: time to reach maximum observed plasma concentration.

Outcome measures

Outcome measures
Measure	Cohort 1: 4 mg/kg n=4 Participants Participants in Cohort 1 received a single dose of 4 mg/kg OP-101 as SC injection on Day 1.	Cohort 2: 8 mg/kg n=4 Participants Participants in Cohort 2 received a single dose of 8 mg/kg OP-101 as SC injection on Day 1.
Pharmacokinetics: Time to Reach Maximum Plasma Concentration (Tmax) of OP-101	14.015 hours Interval 10.0 to 16.05	16.000 hours Interval 12.0 to 24.05

SECONDARY outcome

Timeframe: Pre-dose, 0.5 hours and 1, 2, 4, 6, 8, 10, 12, 16, 24, 30-36, 48 hours post-dose

Population: Analysis was performed on PK parameter population.

AUC0-last: Area under the concentration versus time curve from time zero to the last quantifiable concentration (Clast).

Outcome measures

Outcome measures
Measure	Cohort 1: 4 mg/kg n=4 Participants Participants in Cohort 1 received a single dose of 4 mg/kg OP-101 as SC injection on Day 1.	Cohort 2: 8 mg/kg n=4 Participants Participants in Cohort 2 received a single dose of 8 mg/kg OP-101 as SC injection on Day 1.
Pharmacokinetics: Area Under the Concentration Versus Time Curve From Time Zero to the Last Quantifiable Concentration (AUC0-last) of OP-101	80.940 hr*mcg/mL Standard Deviation 51.8190	212.325 hr*mcg/mL Standard Deviation 30.6459

SECONDARY outcome

Timeframe: Pre-dose, 0.5 hours and 1, 2, 4, 6, 8, 10, 12, 16, 24, 30-36, 48 hours post-dose

Population: Analysis was performed on PK parameter population.

Area under the concentration versus time curve from time zero to 48 hour post dose time point.

Outcome measures

Outcome measures
Measure	Cohort 1: 4 mg/kg n=4 Participants Participants in Cohort 1 received a single dose of 4 mg/kg OP-101 as SC injection on Day 1.	Cohort 2: 8 mg/kg n=4 Participants Participants in Cohort 2 received a single dose of 8 mg/kg OP-101 as SC injection on Day 1.
Pharmacokinetics: Area Under the Concentration Versus Time Curve From Time Zero to 48 Hour Post Dose Time Point (AUC0-48) of OP-101	121.705 hr*mcg/mL Standard Deviation 16.9069	222.173 hr*mcg/mL Standard Deviation 12.4690

SECONDARY outcome

Timeframe: Pre-dose, 0 to 4, 4 to 8, 8 to 12, 12 to 18, 18 to 24, and 24 to 48 hours post dose

Population: Analysis was performed on PK parameter population. Here, 'overall number of participants analyzed' = participants evaluable for this outcome measure.

CLR was defined as renal clearance of the drug from plasma utilizing the AUC and cumulative amount of unchanged study drug excreted into the urine (Ae) to the same duration (as Amount recovered/AUC at 0-48 hours).

Outcome measures

Outcome measures
Measure	Cohort 1: 4 mg/kg n=2 Participants Participants in Cohort 1 received a single dose of 4 mg/kg OP-101 as SC injection on Day 1.	Cohort 2: 8 mg/kg n=4 Participants Participants in Cohort 2 received a single dose of 8 mg/kg OP-101 as SC injection on Day 1.
Pharmacokinetics: Renal Clearance (CLR) for OP-101	0.175 liter per hour (L/hr) Standard Deviation 0.0354	0.150 liter per hour (L/hr) Standard Deviation 0.0606

Adverse Events

Cohort 1: 4 mg/kg

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Cohort 2: 8 mg/kg

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Cohort 1: 4 mg/kg n=4 participants at risk Participants in Cohort 1 received a single dose of 4 mg/kg OP-101 as SC injection on Day 1.	Cohort 2: 8 mg/kg n=4 participants at risk Participants in Cohort 2 received a single dose of 8 mg/kg OP-101 as SC injection on Day 1.
General disorders Injection site erythema	0.00% 0/4 • Up to Day 15 Analysis was performed on safety population.	75.0% 3/4 • Up to Day 15 Analysis was performed on safety population.
General disorders Injection site mass	0.00% 0/4 • Up to Day 15 Analysis was performed on safety population.	25.0% 1/4 • Up to Day 15 Analysis was performed on safety population.
General disorders Injection site pain	25.0% 1/4 • Up to Day 15 Analysis was performed on safety population.	0.00% 0/4 • Up to Day 15 Analysis was performed on safety population.
General disorders Injection site pallor	25.0% 1/4 • Up to Day 15 Analysis was performed on safety population.	0.00% 0/4 • Up to Day 15 Analysis was performed on safety population.
Reproductive system and breast disorders Dysmenorrhoea	0.00% 0/4 • Up to Day 15 Analysis was performed on safety population.	25.0% 1/4 • Up to Day 15 Analysis was performed on safety population.

Additional Information

Jeffrey L Cleland, PhD, Executive Chair

Orpheris, Inc.

Phone: 650-505-5048

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place