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Terminated

NCT Number: NCT05292950

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

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.

Why the study stopped: Sponsor business decision
Terminated

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Key information

Conditions

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

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Research Site 2, South Brisbane, Queensland, Australia

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Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

ARO-MUC5AC

Drug

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

Placebo

Drug

calculated volume to match active treatment by inhalation of nebulized solution

Primary outcomes

  1. Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

    Time frame: From first dose of study drug up to Day 29 (single dose phase), and up to Day 85 (multiple dose phase)

    An adverse event (AE) is any untoward medical occurrence, which does not necessarily have to have a causal relationship with treatment. TEAEs will be defined as AEs with onset after administration of the study drug, or when a preexisting medical condition increases in severity or frequency after study drug administration. A serious AE (SAE) is an AE occurring during any study phase, and at any dose of study drug that: results in death; is immediately life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability or incapacity; results in a congenital abnormality or birth defect; is an important medical event that may jeopardize the subject or may require medical intervention to prevent one of the outcomes listed above.

Secondary outcomes

  1. Change Over Time From Baseline in Forced Expiratory Volume (FEV1), SAD Cohorts

    Time frame: Baseline, Day 1: 15, 60, 120 minutes (mins) post-dose, Days 2,3, 8, 15, 22, 29 (end of study [EOS])

  2. Change Over Time From Baseline in FEV1, MAD Cohorts

    Time frame: Baseline, Day 1 (15, 60, 120 mins post-dose), Days 2, 3, 8, 15 (15, 60, 120 mins post-dose), 22, 29 (15, 60, 120 mins post-dose), 30, 35, 36, 43, 50, 57, 71, 85 (EOS)

  3. Change Over Time From Baseline in Forced Vital Capacity (FVC), SAD Cohorts

    Time frame: Baseline, Day 1: 15, 60, 120 minutes (mins) post-dose, Days 2, 3, 8, 15, 22, 29 (end of study [EOS])

    Day 1: Predose, at 0.5, 1, 2, 4, 6, 8, and 12 hours postdose (based on start of inhalation); Day 2: 24 hours postdose; Day 3: 48 hours postdose

  4. Change Over Time From Baseline in FVC, MAD Cohorts

    Time frame: Baseline, Day 1 (15, 60, 120 mins post-dose), Days 2, 8, 15 (15, 60, 120 mins post-dose), 22, 29 (15, 60, 120 mins post-dose), 30, 35, 36, 43, 50, 57, 71, 85 (EOS)

  5. Pharmacokinetics (PK) of ARO-MUC5AC: Maximum Observed Plasma Concentration (Cmax), SAD Cohorts

    Time frame: Day 1: Predose, at 0.5, 1, 2, 4, 6, 8, and 12 hours postdose (based on start of inhalation); Day 2: 24 hours postdose; Day 3: 48 hours postdose

  6. PK of ARO-MUC5AC: Time to Cmax (Tmax), SAD Cohorts

    Time frame: Day 1: Predose, at 0.5, 1, 2, 4, 6, 8, and 12 hours postdose (based on start of inhalation); Day 2: 24 hours postdose; Day 3: 48 hours postdose

  7. PK of ARO-MUC5AC: Area Under the Plasma Concentration Versus Time Curve From Time 0 to the Time of the Last Measurable Concentration (AUC0-t), SAD Cohorts

    Time frame: Day 1: Predose, at 0.5, 1, 2, 4, 6, 8, and 12 hours postdose (based on start of inhalation); Day 2: 24 hours postdose; Day 3: 48 hours postdose

  8. PK of ARO-MUC5AC: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24), SAD Cohorts

    Time frame: Day 1: Predose, at 0.5, 1, 2, 4, 6, 8, 12, and 24 hours postdose (based on start of inhalation)

  9. PK of ARO-MUC5AC: Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUCinf), SAD Cohorts

    Time frame: Day 1: Predose, at 0.5, 1, 2, 4, 6, 8, and 12 hours postdose (based on start of inhalation); Day 2: 24 hours postdose; Day 3: 48 hours postdose

  10. PK of ARO-MUC5AC: Terminal Elimination Half-Life (t1/2), SAD Cohorts

    Time frame: Day 1: Predose, at 0.5, 1, 2, 4, 6, 8, and 12 hours postdose (based on start of inhalation); Day 2: 24 hours postdose; Day 3: 48 hours postdose

  11. PK of ARO-MUC5AC: Apparent Systemic Clearance (CL/F), SAD Cohorts

    Time frame: Day 1: Predose, at 0.5, 1, 2, 4, 6, 8, and 12 hours postdose (based on start of inhalation); Day 2: 24 hours postdose; Day 3: 48 hours postdose

  12. PK of ARO-MUC5AC: Apparent Terminal-Phase Volume of Distribution (Vz/F), SAD Cohorts

    Time frame: Day 1: Predose, at 0.5, 1, 2, 4, 6, 8, and 12 hours postdose (based on start of inhalation); Day 2: 24 hours postdose; Day 3: 48 hours postdose

  13. PK of ARO-MUC5AC: Recovery of Unchanged Drug in Urine Over 24 Hours (Amount Excreted; Ae), SAD Cohorts Only

    Time frame: Day 1: Predose and cumulatively from 0 to 6 hours and 6 to 24 hours postdose.

  14. PK of ARO-MUC5AC: Fraction of Respirable Delivered Dose (RDD) Excreted Unchanged in Urine Over 24 Hours Postdose, as a Percentage (Fe), SAD Cohorts Only

    Time frame: Day 1: Predose and cumulatively from 0 to 6 hours and 6 to 24 hours postdose.

  15. PK of ARO-MUC5AC: Renal Clearance (CLr), SAD Cohorts Only

    Time frame: Day 1: Predose and cumulatively from 0 to 6 hours and 6 to 24 hours postdose.

  16. PK of ARO-MUC5AC: Maximum Observed Plasma Concentration (Cmax), MAD Cohorts

    Time frame: NHV Cohorts: Day 1: Predose, 0.5, 1, 2, 4, and 6 hours postdose (based on start of inhalation); Day 15: Predose; Day 29: Predose, 0.5, 1, 2, 4, and 6 hours postdose.

  17. PK of ARO-MUC5AC: Tmax, MAD Cohorts

    Time frame: NHV Cohorts: Day 1: Predose, 0.5, 1, 2, 4, and 6 hours postdose (based on start of inhalation); Day 15: Predose; Day 29: Predose, 0.5, 1, 2, 4, and 6 hours postdose.

  18. PK of ARO-MUC5AC: AUC0-t, MAD Cohorts

    Time frame: NHV Cohorts: Day 1: Predose, 0.5, 1, 2, 4, and 6 hours postdose (based on start of inhalation); Day 15: Predose; Day 29: Predose, 0.5, 1, 2, 4, and 6 hours postdose.

Sponsors and collaborators

Lead sponsor

Arrowhead Pharmaceuticals

Industry

Registry information

Official study title

A Phase 1/2a Study Evaluating the Effects of ARO-MUC5AC Inhalation Solution in Healthy Subjects and Patients With Muco-Obstructive Lung Disease

Important dates

Study start
2022
Primary completion
2024
Study completion
2024
First posted
Mar 23, 2022
Registry last updated
Sep 14, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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