Single and Multiple Ascending Dose Study of AER-01

A Randomized, Double-blind, Placebo-controlled, Phase 1 Study of AER-01 (Solution for Inhalation, Delivered Via Nebulizer): As a First-In-Human Single Ascending Dose in Healthy Volunteers (Part A), and a 7-day Multiple Ascending Dose in Healthy Volunteers and Subjects With Chronic Obstructive Pulmonary Disease (COPD) and Moderate Airflow Limitation (Part B)

This is a first-in-human, randomized, double-blind, placebo-controlled, Phase 1 study of AER-01 (solution for inhalation, delivered via nebulizer) to assess the safety, tolerability and PK of AER-01. The study will be conducted in 2 parts: a single ascending dose (SAD) part in HVs (Part A) and a 7-day multiple ascending dose (MAD) part in HVs and subjects with COPD and moderate airflow limitation (Part B).

Study Overview

• Status: Recruiting
• Conditions: COPD
• Intervention / Treatment: Drug: AER-01; Drug: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 72
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Kelly Otto; Phone Number: 919-345-4256; Email: clinical@aertherapeutics.com

Study Locations

• Australia
  • Adelaide, Australia
    • Recruiting
    • CMAX
    • Contact: Emir Redzepagic, MD
  • Sydney, Australia
    • Recruiting
    • Scientia Clinical Research
    • Contact: Chris Argent, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria Health Volunteers:

• Healthy volunteers: Male or female aged ≥18 to ≤65 years.
• Current non-smoker or casual smoker Participants
• Females must not be pregnant or lactating

Participants with COPD and Moderate Airflow Limitation

• COPD volunteers: Male or female aged ≥35 to ≤80 years.
• Documented diagnosis of moderate COPD (GOLD 2)
• Former smoker with a history of at least 10 pack-years of cigarette smoking
• Females must not be pregnant or lactating

Exclusion Criteria:

• History of unstable medical conditions or major surgical procedures over the 12 months prior to Screening.
• History or evidence of any clinically significant cardiovascular, gastrointestinal, endocrinologic, hematologic, hepatic, immunologic, metabolic, urologic (excluding history of recurrent urinary tract infection), neurologic (excluding history of migraine), dermatologic, psychiatric, renal and/or other major disease or malignancy, or any other condition that in the opinion of the PI or designee might obfuscate the study data.
• History of malignancy of any type, other than in situ cervical cancer or surgically excised non melanomatous skin cancers, within 5 years before Screening. Part B Participants with COPD and Moderate Airflow Limitation
• Has evidence of significant lung restriction on pulmonary function testing or a history of interstitial lung disease (including sarcoidosis and idiopathic pulmonary fibrosis), severe bronchiectasis, cystic fibrosis (asthma is not an exclusion.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: AER-01; Specified dose on specified days	Drug: AER-01; AER-01 solution for inhalation delivered via nebulizer
Placebo Comparator: Placebo; Specified dose on specified days	Drug: Placebo; Placebo for solution for inhalation delivered via nebulizer

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0	Will be summarized by cohort, preferred term (PT), system organ class (SOC), severity, and relationship to IP.	Baseline to Day 2 (Part A), Baseline to Day 8 (Part B)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maximum Plasma Concentration [Cmax]	Will be summarized using descriptive statistics by cohort, timepoint, and dose	Baseline to Day 2 (Part A), Baseline to Day 7 (Part B)
Time to maximum concentration [Tmax]	Will be summarized using descriptive statistics by cohort, timepoint, and dose	Baseline to Day 2 (Part A), Baseline to Day 7 (Part B)
Area under the drug concentration-time curve from time 0 (time of dosing) extrapolated to infinity [AUC 0-inf]	Will be summarized using descriptive statistics by cohort, timepoint, and dose	Baseline to Day 2 (Part A), Baseline to Day 7 (Part B)

Collaborators and Investigators

• Sponsor: Aer Therapeutics
• Investigators: Principal Investigator: Emir Redzepagic, MD, CMAX Clinical Research

Study record dates

Study Major Dates

• Study Start (Actual): May 26, 2023
• Primary Completion (Estimated): April 1, 2024
• Study Completion (Estimated): July 1, 2024

Study Registration Dates

• First Submitted: April 28, 2023
• First Submitted That Met QC Criteria: May 8, 2023
• First Posted (Actual): May 17, 2023

Study Record Updates

• Last Update Posted (Actual): August 30, 2023
• Last Update Submitted That Met QC Criteria: August 29, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Other Study ID Numbers: AER-01-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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