Study of ARO-MUC5AC in Healthy Subjects and Patients With Muco-Obstructive Lung Disease

NCT05292950 | Terminated Phase 1 DMC FDA Drug

• 2 other identifiers: AROMUC5AC-10012022-003467-21
• Study Type: interventional
• Target: 78
• Locations: 7 countries
• Sites: 13

Brief Summary

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of ARO-MUC5AC in normal healthy volunteers (NHVs), patients with moderate-to-severe asthma and patients with moderate-to-severe chronic obstructive pulmonary disease (COPD). In part 1 NHVs will receive a single dose of ARO-MUC5AC or placebo. In part 2 of the study, NHVs, adult patients with asthma, and adult patients with COPD will receive 3 doses of ARO-MUC5AC or placebo.

Conditions

Asthma; Chronic Obstructive Pulmonary Disease

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Treatment-Emergent Adverse Events (TEAEs)

  single dose phase: up to Day 29; multiple dose phase: up to Day 85

Secondary Outcomes (12)

• Change Over Time from Baseline in Forced Expiratory Volume (FEV1)

  single dose phase: up to Day 29; multiple dose phase: up to Day 85

• Change Over Time from Baseline in Forced Vital Capacity (FVC)

  single dose phase: up to Day 29; multiple dose phase: up to Day 85

• PK of ARO-MUC5AC: Maximum Observed Plasma Concentration (Cmax)

  single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1 and 29 for NHVs; and up to 6 hours post-dose on Days 1 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD

• PK of ARO-MUC5AC: Area Under the Plasma Concentration versus Time Curve From Zero to 24 Hours (AUC0-24)

  single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1 and 29 for NHVs; and up to 6 hours post-dose on Days 1 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD

• PK of ARO-MUC5AC: Area Under the Plasma Concentration versus Time Curve From Zero to the Last Quantifiable Plasma Concentration (AUClast)

  single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1 and 29 for NHVs; and up to 6 hours post-dose on Days 1 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD

Study Arms (2)

ARO-MUC5AC

EXPERIMENTAL

ARO-MUC5AC Inhalation

Drug: ARO-MUC5AC

Placebo

PLACEBO COMPARATOR

(0.9% NaCl)

Drug: Placebo

Interventions

ARO-MUC5AC DRUG

single or multiple doses of ARO- MUC5AC by inhalation of nebulized solution

ARO-MUC5AC

Placebo DRUG

calculated volume to match active treatment by inhalation of nebulized solution

Placebo

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Normal pulmonary function tests at Screening (NHVs only)
• Confirmed diagnosis of asthma or COPD based on source verifiable medical record (asthma and COPD patients only)
• No abnormal finding of clinical relevance at Screening (NHVs only)
• Stable dose of asthma controller medications for at least 28 days prior to Screening (asthma patients only)
• Documented treatment with an inhaled corticosteroid and at least 1 additional maintenance asthma controller medication for at least 3 months prior to Screening (asthma patients only)
• Non-smoking (NHVs and asthma patients)
• Current smoker or ex-smoker with smoking history of ≥ 10 pack-years (COPD patients only)
• All COPD treatments have been stable for at least one month prior to Screening (COPD patients only)
• Able to produce an induced sputum sample at Screening
• Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception. Males must not donate sperm during the study and for at least 90 days following the last dose of study drug
• Willing to provide written informed consent and to comply with study requirements

You may not qualify if:

• Acute lower respiratory infection within 30 days prior to first dose and/or acute upper respiratory infection within 7 days prior to first dose
• Positive COVID-19 test during Screening window
• Any history of chronic pulmonary disease (NHVs only)
• Any concomitant pulmonary disease in asthma or COPD patients that could interfere with the evaluation of the study drug or interpretation of patient safety or study results
• Use of theophylline within 30 days prior to first dose
• History of lung volume reduction surgery or pneumonectomy (COPD patients)
• Need for chronic oxygen support at Screening
• Clinically significant health concerns (other than asthma in asthma patients)
• Human immunodeficiency virus (HIV) infection, seropositive for hepatitis B virus (HBV), seropositive for hepatitis C virus (HCV)
• Uncontrolled hypertension
• Unwilling to limit alcohol consumption to within moderate limits for the duration of the study
• Use of illicit drugs
• Use of an investigational agent or device within 30 days prior to first dose

Sponsors & Collaborators

• Arrowhead Pharmaceuticals: lead

Study Sites (13)

Research Site 2

South Brisbane, Queensland, 4101, Australia

Location

Research Site 1

Nedlands, 6009, Australia

Location

Research Site 1

Auckland, 1010, New Zealand

Location

Research Site 2

Auckland, 1051, New Zealand

Location

Research Site 1

Bialystok, 15-010, Poland

Location

Research Site 2

Krakow, 31-455, Poland

Location

Research Site 3

Oświęcim, 32-600, Poland

Location

Research Site 3

Bucheon-si, Gyeonggi-do, 14647, South Korea

Location

Research Site 2

Jeonju, 54907, South Korea

Location

Research Site 1

Seoul, 04763, South Korea

Location

Research Site 1

Barcelona, 08017, Spain

Location

Research Site 1

Bangkok, 10700, Thailand

Location

Research Site 1

Wythenshawe, Manchester, M23 9QZ, United Kingdom

Location

MeSH Terms

Conditions

Asthma; Pulmonary Disease, Chronic Obstructive

Condition Hierarchy (Ancestors)

Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Respiratory Hypersensitivity; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 15, 2022
• First Posted: March 23, 2022
• Study Start: June 27, 2022
• Primary Completion: November 12, 2024
• Study Completion: November 12, 2024
• Last Updated: November 5, 2025

Record last verified: 2025-11

Locations

AU (2); KR (3); TH (1); ES (1); GB (1); PL (3); NZ (2)