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

February 2, 2024 updated by: Arrowhead Pharmaceuticals

A Phase 1/2a Study Evaluating the Effects of ARO-MUC5AC Inhalation Solution 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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

104

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
      • Nedlands, Australia, 6009
        • Recruiting
        • Institute For Respiratory Health - Perth
        • Principal Investigator:
          • John Blakey, MD
        • Contact:
  • Korea, Republic of
      • Jeonju, Korea, Republic of, 54907
        • Recruiting
        • Jeonbuk National University Hospital
        • Contact:
        • Principal Investigator:
          • Yonchul Lee, MD
      • Seoul, Korea, Republic of, 04763
        • Recruiting
        • Hanyang University Seoul Hospital
        • Contact:
        • Principal Investigator:
          • Sang Heon Kim, MD
  • New Zealand
      • Auckland, New Zealand, 1010
        • Recruiting
        • New Zealand Research Sleep Institute
        • Contact:
        • Contact:
        • Principal Investigator:
          • Michelle Baker, MD
      • Auckland, New Zealand, 1051
        • Recruiting
        • New Zealand Clinical Research-Auckland
        • Contact:
        • Principal Investigator:
          • Mark O'Carroll, MD
  • Poland
      • Białystok, Poland, 15-010
        • Recruiting
        • Prywatny Gabinet Internistyczno-Alergologiczny
        • Contact:
        • Principal Investigator:
          • Zenon Siergiejko, MD
      • Kraków, Poland, 31-455
        • Recruiting
        • Krakmed.NZOZ
        • Principal Investigator:
          • Elzbieta Hajol, MD
        • Contact:
      • Oświęcim, Poland, 32-600
        • Recruiting
        • Medicome SP.ZO.O
        • Principal Investigator:
          • Iwona Kobielsz-Gembala, MD
        • Contact:
  • Spain
      • Barcelona, Spain, 08017
        • Recruiting
        • Giromed Institute Barcelona
        • Principal Investigator:
          • Juan Roldan Sanchez, MD
        • Contact:
  • Thailand
      • Bangkok, Thailand, 10700

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 60 years (Adult, Older Adult)

Accepts Healthy Volunteers

Yes

Description

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 12 weeks 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ARO-MUC5AC
ARO-MUC5AC Inhalation
Drug: ARO-MUC5AC
single or multiple doses of ARO- MUC5AC by inhalation of nebulized solution
Placebo Comparator: Placebo
(0.9% NaCl)
Drug: Placebo
calculated volume to match active treatment by inhalation of nebulized solution

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
Time Frame: single dose phase: up to Day 29; multiple dose phase: up to Day 85
single dose phase: up to Day 29; multiple dose phase: up to Day 85

Secondary Outcome Measures

Outcome Measure
Time Frame
Change Over Time from Baseline in Forced Expiratory Volume (FEV1)
Time Frame: single dose phase: up to Day 29; multiple dose phase: up to Day 85
single dose phase: up to Day 29; multiple dose phase: up to Day 85
Change Over Time from Baseline in Forced Vital Capacity (FVC)
Time Frame: single dose phase: up to Day 29; multiple dose phase: up to Day 85
single dose phase: up to Day 29; multiple dose phase: up to Day 85
PK of ARO-MUC5AC: Recovery of Unchanged Drug in Urine Over 24 Hours (Amount Excreted; Ae)
Time Frame: through 24 hours post-dose
through 24 hours post-dose
PK of ARO-MUC5AC: Percentage of Administered Drug Recovered in Urine Over 0-24 Hours
Time Frame: through 24 hours post-dose
through 24 hours post-dose
PK of ARO-MUC5AC: Maximum Observed Plasma Concentration (Cmax)
Time Frame: single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD
single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 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)
Time Frame: single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD
single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 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)
Time Frame: single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD
single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 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 Infinity (AUCinf)
Time Frame: single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD
single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD
PK of ARO-MUC5AC: Terminal Elimination Half-Life (t1/2)
Time Frame: single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD
single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD
PK of ARO-MUC5AC: Apparent Systemic Clearance (CL/F)
Time Frame: single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD
single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD
PK of ARO-MUC5AC: Apparent Terminal-Phase Volume of Distribution (VZ/F)
Time Frame: single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD
single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD
PK of ARO-MUC5AC: Renal Clearance (CLr)
Time Frame: single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD
single dose phase: up to 48 Hours; multiple dose phase: up to 6 hours post-dose on Days 1, 15 and 29 for NHVs; and up to 6 hours post-dose on Days 1, 15 and 29, and up to 24 hours post-dose on Days 2 and 30 for participants with asthma or COPD

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Arrowhead Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 27, 2022

Primary Completion (Estimated)

November 1, 2024

Study Completion (Estimated)

November 1, 2024

Study Registration Dates

First Submitted

March 15, 2022

First Submitted That Met QC Criteria

March 15, 2022

First Posted (Actual)

March 23, 2022

Study Record Updates

Last Update Posted (Estimated)

February 5, 2024

Last Update Submitted That Met QC Criteria

February 2, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AROMUC5AC-1001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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