SAD Study of APR002 Investigating the Safety/Tolerability, Pharmacokinetics/Pharmacodynamics in Healthy Subjects

A Phase 1, Randomized, Blinded, Placebo-Controlled, First-in-Human, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of APR002 Administered by Inhalation in Healthy Volunteers

This study will investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of single ascending doses of APR002 in healthy subjects.

Study Overview

• Status: Completed
• Conditions: COVID-19
• Intervention / Treatment: Drug: Placebo; Drug: APR002

Detailed Description

A Phase 1, Randomized, Blinded, Placebo-Controlled, First-in-Human, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of APR002 Administered by Inhalation in Healthy Volunteers

• Study Type: Interventional
• Enrollment (Actual): 42
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Clinical Trials Support; Phone Number: 650-273-2627; Email: clinicaltrials@ghddi.org

Study Locations

• Australia
  • South Australia
    • Adelaide, South Australia, Australia, 5000
      • CMAX Clinical Research

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria:

• Healthy men or women aged 18 to 55 years (inclusive) with suitable veins for cannulation or repeated venipuncture.
• Women can be of childbearing potential and must have been stable on a highly effective contraceptive method for at least 3 months prior to Visit 1 (screening) and be willing to continue on the chosen contraceptive method
• Male subjects should be willing to use a condom (with spermicide) to prevent pregnancy and drug exposure of a female partner and refrain from donating sperm or fathering a child from the day of the investigational product administration until 3 months
• Have a body mass index (BMI) between 18 and 32 kg/m2 and weigh at least 45 kg.

Exclusion Criteria:

• History or presence of clinically significant medical (e.g., chronic obstructive pulmonary disease, asthma, COVID-19, etc.) or psychiatric condition or disease
• Abnormal vital signs, after minutes rest, defined as any of the following (SBP > 140 mmHg, Diastolic blood pressure (DBP) > 90 mmHg, Heart rate < 40 or > 99 beats per minute)
• Prolonged QTcF > 450 ms or family history of long QT syndrome

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Basic Science
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Placebo; Other Names: 0.9% Sodium Chloride; Saline
Experimental: Intervention/Treatment; Single dose, oral inhalation (nebuliser solution)	Drug: APR002; Active Investigational Product; Other Names: TLR7 agonist

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety and tolerability of APR002 in healthy subjects as assessed by incidence of treatment-emergent adverse events according to CTCAE v5.0 criteria	Safety assessments will include evaluation of incidence of treatment-emergent adverse events (AEs) according to CTCAE v5.0 criteria, including vital signs, resting electrocardiogram (ECG) parameters, standard hematology, chemistry, urinalysis and other tests	Screening up to Day 14

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Pharmacokinetics of APR002 in healthy subjects as assessed by maximum plasma concentration (Cmax) towards determination of the optimal pharmacokinetic dose	Determination of maximum plasma concentration (Cmax)	Day 1 to Day 3
Pharmacokinetics of APR002 in healthy subjects as assessed by time to maximum concentration (Tmax) towards determination of the optimal pharmacokinetic dose	Determination of time to maximum concentration	Day 1 to Day 3
Pharmacokinetics of APR002 in healthy subjects as assessed by plasma exposure (AUC0-t, AUC0-inf) determination of the optimal pharmacokinetic dose	Determination of plasma exposure (AUC0-t, AUC0-inf)	Day 1 to Day 3
Pharmacokinetics of APR002 in healthy subjects as assessed by terminal elimination half life (t1/2) determination of the optimal pharmacokinetic dose	Determination of terminal half life	Day 1 to Day 3
Pharmacokinetics of APR002 in healthy subjects as assessed by apparent volume of distribution during the terminal elimination phase after inhalation (extravascular) administration for determination of the optimal pharmacokinetic dose	Determination of volume of distribution (CL/F and Vz/F)	Day 1 to Day 3

Collaborators and Investigators

• Sponsor: Global Health Drug Discovery Institute
• Collaborators: CMAX Clinical Research Pty Limited; Avance Clinical
• Investigators: Study Director: Aaron Weitzman, Global Health Drug Discovery Institute

Study record dates

Study Major Dates

• Study Start (Actual): March 20, 2023
• Primary Completion (Actual): June 29, 2023
• Study Completion (Actual): July 27, 2023

Study Registration Dates

• First Submitted: February 17, 2022
• First Submitted That Met QC Criteria: February 24, 2022
• First Posted (Actual): March 7, 2022

Study Record Updates

• Last Update Posted (Actual): August 2, 2023
• Last Update Submitted That Met QC Criteria: August 1, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Other Study ID Numbers: APR002-NEB-001

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No