AsthmaPhase 1/2NCT05292950
Study of ARO-MUC5AC in Healthy Subjects and Patients With Muco-Obstructive Lung Disease
A Phase 1/2 study testing ARO-MUC5AC in people with Asthma and Chronic Obstructive Pulmonary Disease, run by Arrowhead Pharmaceuticals at 13 sites in 7 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Asthma, Chronic Obstructive Pulmonary Disease
- What is being tested?
- ARO-MUC5AC
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 78 participants
- Where
- 13 sitesin 7 countries
- Study dates
- 27 Jun 2022to 12 Nov 2024
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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)
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- 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
Who cannot join
- 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)
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- 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 locations and contacts
13 sites in 7 countries.
13 sites in 7 countries
Poland3 sites
Research Site 1
Bialystok, 15-010
Research Site 2
Krakow, 31-455
Research Site 3
Oświęcim, 32-600
South Korea3 sites
Research Site 3
Bucheon-si, Gyeonggi-do, 14647
Research Site 2
Jeonju, 54907
Research Site 1
Seoul, 04763
Australia2 sites
Research Site 1
Nedlands, 6009
Research Site 2
South Brisbane, Queensland, 4101
New Zealand2 sites
Research Site 1
Auckland, 1010
Research Site 2
Auckland, 1051
About this study
Official title: 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.
- Conditions
- Asthma, Chronic Obstructive Pulmonary Disease
- Being tested
- ARO-MUC5AC, Placebo
- Medical subject headings
- Asthma, Pulmonary Disease, Chronic Obstructive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARO-MUC5ACDrug
single or multiple doses of ARO- MUC5AC by inhalation of nebulized solution
PlaceboDrug
calculated volume to match active treatment by inhalation of nebulized solution
| Study group | Role | What they receive |
|---|---|---|
| NHV (Single Ascending Dose [SAD]): Pooled Placebo | Placebo comparator | a single dose of placebo was administered once on Day 1 Receives: Drug: Placebo |
| NHV (SAD): ARO-MUC5AC 24 mg | Experimental | a single dose of ARO-MUC5AC 24 mg was administered once on Day 1 Receives: Drug: ARO-MUC5AC |
| NHV (SAD): ARO-MUC5AC 56 mg | Experimental | a single dose of ARO-MUC5AC 56 mg was administered once on Day 1 Receives: Drug: ARO-MUC5AC |
| NHV (SAD): ARO-MUC5AC 108 mg | Experimental | a single dose of ARO-MUC5AC 108 mg was administered once on Day 1 Receives: Drug: ARO-MUC5AC |
| NHV (SAD): ARO-MUC5AC 232 mg | Experimental | a single dose of ARO-MUC5AC 232 mg was administered once on Day 1 Receives: Drug: ARO-MUC5AC |
| NHV (Multiple Ascending Dose [MAD]): Pooled Placebo | Placebo comparator | 3 total doses of placebo were administered, with 1 dose given on each of Days 1, 15, and 29 Receives: Drug: Placebo |
| NHV (MAD): ARO-MUC5AC 24 mg | Experimental | 3 total doses of ARO-MUC5AC 24 mg were administered, with 1 dose given on each of Days 1, 15, and 29 Receives: Drug: ARO-MUC5AC |
| NHV (MAD): ARO-MUC5AC 56 mg | Experimental | 3 total doses of ARO-MUC5AC 56 mg were administered, with 1 dose given on each of Days 1, 15, and 29 Receives: Drug: ARO-MUC5AC |
| NHV (MAD): ARO-MUC5AC 108 mg | Experimental | 3 total doses of ARO-MUC5AC 108 mg were administered, with 1 dose given on each of Days 1, 15, and 29 Receives: Drug: ARO-MUC5AC |
| Asthma Patients (MAD): Pooled Placebo | Placebo comparator | 3 total doses of placebo were administered, with 1 dose given on each of Days 1, 15, and 29 Receives: Drug: Placebo |
| Asthma Patients (MAD): ARO-MUC5AC 56 mg | Experimental | 3 total doses of ARO-MUC5AC 56 mg were administered, with 1 dose given on each of Days 1, 15, and 29 Receives: Drug: ARO-MUC5AC |
| Asthma Patients (MAD): ARO-MUC5AC 108 mg | Experimental | 3 total doses of ARO-MUC5AC 108 mg were administered, with 1 dose given on each of Days 1, 15, and 29 Receives: Drug: ARO-MUC5AC |
| COPD Patients (MAD): Pooled Placebo | Placebo comparator | 3 total doses of placebo were administered, with 1 dose given on each of Days 1, 15, and 29 Receives: Drug: Placebo |
| COPD Patients (MAD): 56 mg | Experimental | 3 total doses of ARO-MUC5AC 56 mg were administered, with 1 dose given on each of Days 1, 15, and 29 Receives: Drug: ARO-MUC5AC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
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.
Measured: From first dose of study drug up to Day 29 (single dose phase), and up to Day 85 (multiple dose phase)
Secondary outcomes (18)
Change Over Time From Baseline in Forced Expiratory Volume (FEV1), SAD Cohorts
Measured: Baseline, Day 1: 15, 60, 120 minutes (mins) post-dose, Days 2,3, 8, 15, 22, 29 (end of study [EOS])
Change Over Time From Baseline in FEV1, MAD Cohorts
Measured: 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)
Change Over Time From Baseline in Forced Vital Capacity (FVC), SAD Cohorts
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
Measured: Baseline, Day 1: 15, 60, 120 minutes (mins) post-dose, Days 2, 3, 8, 15, 22, 29 (end of study [EOS])
Change Over Time From Baseline in FVC, MAD Cohorts
Measured: 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)
Pharmacokinetics (PK) of ARO-MUC5AC: Maximum Observed Plasma Concentration (Cmax), SAD Cohorts
Measured: 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
PK of ARO-MUC5AC: Time to Cmax (Tmax), SAD Cohorts
Measured: 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
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
Measured: 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
PK of ARO-MUC5AC: Area Under the Plasma Concentration Versus Time Curve From Zero to 24 Hours (AUC0-24), SAD Cohorts
Measured: Day 1: Predose, at 0.5, 1, 2, 4, 6, 8, 12, and 24 hours postdose (based on start of inhalation)
PK of ARO-MUC5AC: Area Under the Plasma Concentration Versus Time Curve From Zero to Infinity (AUCinf), SAD Cohorts
Measured: 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
PK of ARO-MUC5AC: Terminal Elimination Half-Life (t1/2), SAD Cohorts
Measured: 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
PK of ARO-MUC5AC: Apparent Systemic Clearance (CL/F), SAD Cohorts
Measured: 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
PK of ARO-MUC5AC: Apparent Terminal-Phase Volume of Distribution (Vz/F), SAD Cohorts
Measured: 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
PK of ARO-MUC5AC: Recovery of Unchanged Drug in Urine Over 24 Hours (Amount Excreted; Ae), SAD Cohorts Only
Measured: Day 1: Predose and cumulatively from 0 to 6 hours and 6 to 24 hours postdose.
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
Measured: Day 1: Predose and cumulatively from 0 to 6 hours and 6 to 24 hours postdose.
PK of ARO-MUC5AC: Renal Clearance (CLr), SAD Cohorts Only
Measured: Day 1: Predose and cumulatively from 0 to 6 hours and 6 to 24 hours postdose.
PK of ARO-MUC5AC: Maximum Observed Plasma Concentration (Cmax), MAD Cohorts
Measured: 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.
PK of ARO-MUC5AC: Tmax, MAD Cohorts
Measured: 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.
PK of ARO-MUC5AC: AUC0-t, MAD Cohorts
Measured: 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.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2022
- Study start27 Jun 2022
- Primary completion12 Nov 2024
- Study completion12 Nov 2024
Why it stopped: Sponsor business decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for AsthmaStudies of the same condition, open and under way.
- More trials run by Arrowhead PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 78 participants (actual)
Sponsor and investigators
- Lead sponsor
- Arrowhead Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05292950
- Sponsor's protocol ID
- AROMUC5AC-1001
- Other IDs
- 2022-003467-21
- Status last verified
- Aug 2026
- First submitted
- 15 Mar 2022
- Last updated
- 14 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No