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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05292950




Registration number
NCT05292950
Ethics application status
Date submitted
15/03/2022
Date registered
23/03/2022
Date last updated
12/04/2024

Titles & IDs
Public title
Study of ARO-MUC5AC in Healthy Subjects and Patients With Muco-Obstructive Lung Disease
Scientific title
A Phase 1/2a Study Evaluating the Effects of ARO-MUC5AC Inhalation Solution in Healthy Subjects and Patients With Muco-Obstructive Lung Disease
Secondary ID [1] 0 0
2022-003467-21
Secondary ID [2] 0 0
AROMUC5AC-1001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Asthma 0 0
Chronic Obstructive Pulmonary Disease 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Chronic obstructive pulmonary disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ARO-MUC5AC
Treatment: Drugs - Placebo

Experimental: ARO-MUC5AC - ARO-MUC5AC Inhalation

Placebo Comparator: Placebo - (0.9% NaCl)


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

Treatment: Drugs: Placebo
calculated volume to match active treatment by inhalation of nebulized solution
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
single dose phase: up to Day 29; multiple dose phase: up to Day 85
Secondary outcome [1] 0 0
Change Over Time from Baseline in Forced Expiratory Volume (FEV1)
Timepoint [1] 0 0
single dose phase: up to Day 29; multiple dose phase: up to Day 85
Secondary outcome [2] 0 0
Change Over Time from Baseline in Forced Vital Capacity (FVC)
Timepoint [2] 0 0
single dose phase: up to Day 29; multiple dose phase: up to Day 85
Secondary outcome [3] 0 0
PK of ARO-MUC5AC: Maximum Observed Plasma Concentration (Cmax)
Timepoint [3] 0 0
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
Secondary outcome [4] 0 0
PK of ARO-MUC5AC: Area Under the Plasma Concentration versus Time Curve From Zero to 24 Hours (AUC0-24)
Timepoint [4] 0 0
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
Secondary outcome [5] 0 0
PK of ARO-MUC5AC: Area Under the Plasma Concentration versus Time Curve From Zero to the Last Quantifiable Plasma Concentration (AUClast)
Timepoint [5] 0 0
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
Secondary outcome [6] 0 0
PK of ARO-MUC5AC: Area Under the Plasma Concentration versus Time Curve From Zero to Infinity (AUCinf)
Timepoint [6] 0 0
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
Secondary outcome [7] 0 0
PK of ARO-MUC5AC: Terminal Elimination Half-Life (t1/2)
Timepoint [7] 0 0
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
Secondary outcome [8] 0 0
PK of ARO-MUC5AC: Apparent Systemic Clearance (CL/F)
Timepoint [8] 0 0
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
Secondary outcome [9] 0 0
PK of ARO-MUC5AC: Apparent Terminal-Phase Volume of Distribution (VZ/F)
Timepoint [9] 0 0
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
Secondary outcome [10] 0 0
PK of ARO-MUC5AC: Recovery of Unchanged Drug in Urine Over 24 Hours (Amount Excreted; Ae)
Timepoint [10] 0 0
through 24 hours post-dose
Secondary outcome [11] 0 0
PK of ARO-MUC5AC: Percentage of Administered Drug Recovered in Urine Over 0-24 Hours
Timepoint [11] 0 0
through 24 hours post-dose
Secondary outcome [12] 0 0
PK of ARO-MUC5AC: Renal Clearance (CLr)
Timepoint [12] 0 0
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

Eligibility
Key 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
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key 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 design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
27/06/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/11/2024
Actual
Sample size
Target
104
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Institute For Respiratory Health - Perth - Nedlands
Recruitment postcode(s) [1] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
Korea, Republic of
State/province [1] 0 0
Jeonju
Country [2] 0 0
Korea, Republic of
State/province [2] 0 0
Seoul
Country [3] 0 0
New Zealand
State/province [3] 0 0
Auckland
Country [4] 0 0
Poland
State/province [4] 0 0
Bialystok
Country [5] 0 0
Poland
State/province [5] 0 0
Kraków
Country [6] 0 0
Poland
State/province [6] 0 0
Oswiecim
Country [7] 0 0
Spain
State/province [7] 0 0
Barcelona
Country [8] 0 0
Thailand
State/province [8] 0 0
Bangkok

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Arrowhead Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05292950
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Medical Monitor
Address 0 0
Country 0 0
Phone 0 0
626-304-3400
Fax 0 0
Email 0 0
AROMUC5AC@arrowheadpharma.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05292950