Single and Multiple Ascending Dose Study of AER-01

Study Overview
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Brief Summary
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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).

Official Title
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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)

Conditions
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COPD

Intervention / Treatment
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* Drug: AER-01
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

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

What is the study measuring?
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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) |