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

Arrowhead Pharmaceuticals

Status and phase: Enrolling; Phase 1

Conditions: Asthma; Chronic Obstructive Pulmonary Disease

Treatments: Drug: Placebo; Drug: ARO-MUC5AC

Study type: Interventional

Funder types: Industry

Identifiers

NCT05292950

2022-003467-21 (EudraCT Number)

AROMUC5AC-1001

Details and patient eligibility

About

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.

Enrollment: 104 estimated patients

Sex: All

Ages: 18 to 70 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• 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

Exclusion criteria

• 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

Note: additional inclusion/exclusion criteria may apply per protocol

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: Triple Blind

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104 participants in 2 patient groups, including a placebo group

ARO-MUC5AC

Experimental group

Description:

ARO-MUC5AC Inhalation

Treatment:

Drug: ARO-MUC5AC

Placebo

Placebo Comparator group

Description:

(0.9% NaCl)

Treatment:

Drug: Placebo