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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/NCT05276570




Registration number
NCT05276570
Ethics application status
Date submitted
3/03/2022
Date registered
11/03/2022
Date last updated
8/03/2024

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

Health condition
Health condition(s) or problem(s) studied:
Asthma 0 0
Condition category
Condition code

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

Experimental: ARO-RAGE - ARO-RAGE Inhalation

Placebo Comparator: Placebo - (0.9% NaCl)


Treatment: Drugs: ARO-RAGE
single or multiple doses of ARO-RAGE 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
From first dose of study drug through the end of study (EOS; up to 113 days)
Secondary outcome [1] 0 0
Change from Baseline Over Time in Forced Expiratory Volume (FEV1)
Timepoint [1] 0 0
Baseline through EOS (up to 113 days) or until serum soluble receptor for advance glycation end products (sRAGE) is = 70% of baseline value
Secondary outcome [2] 0 0
Change from Baseline Over Time in Forced Vital Capacity (FVC)
Timepoint [2] 0 0
Baseline through EOS (up to 113 days) or until serum sRAGE is = 70% of baseline value
Secondary outcome [3] 0 0
Change from Baseline Over Time in Diffusing Capacity for Carbon Monoxide (DLCO)
Timepoint [3] 0 0
Baseline through EOS (up to 113 days) or until serum sRAGE is = 70% of baseline value
Secondary outcome [4] 0 0
PK of ARO-RAGE: Maximum Observed Plasma Concentration (Cmax)
Timepoint [4] 0 0
single dose phase: up to 48 hours post-dose; multiple dose phase: up to 6 hours post-dose on Days 1 and 29 for NHVs, and up to 24 hours post-dose for participants with asthma
Secondary outcome [5] 0 0
PK of ARO-RAGE: Time to Maximum Observed Plasma Concentration (Tmax)
Timepoint [5] 0 0
single dose phase: up to 48 hours post-dose; multiple dose phase: up to 6 hours post-dose on Days 1 and 29 for NHVs, and up to 24 hours post-dose for participants with asthma
Secondary outcome [6] 0 0
PK of ARO-RAGE: Area Under the Plasma Concentration versus Time Curve From Zero to 24 Hours (AUC0-24)
Timepoint [6] 0 0
single dose phase: up to 48 hours post-dose; multiple dose phase: up to 6 hours post-dose on Days 1 and 29 for NHVs, and up to 24 hours post-dose for participants with asthma
Secondary outcome [7] 0 0
PK of ARO-RAGE: Area Under the Plasma Concentration versus Time Curve From Zero to the Last Quantifiable Plasma Concentration (AUClast)
Timepoint [7] 0 0
single dose phase: up to 48 hours post-dose; multiple dose phase: up to 6 hours post-dose on Days 1 and 29 for NHVs, and up to 24 hours post-dose for participants with asthma
Secondary outcome [8] 0 0
PK of ARO-RAGE: Area Under the Plasma Concentration versus Time Curve From Zero to Infinity (AUCinf)
Timepoint [8] 0 0
single dose phase: up to 48 hours post-dose; multiple dose phase: up to 6 hours post-dose on Days 1 and 29 for NHVs, and up to 24 hours post-dose for participants with asthma
Secondary outcome [9] 0 0
PK of ARO-RAGE: Terminal Elimination Half-Life (t1/2)
Timepoint [9] 0 0
single dose phase: up to 48 hours post-dose; multiple dose phase: up to 6 hours post-dose on Days 1 and 29 for NHVs, and up to 24 hours post-dose for participants with asthma
Secondary outcome [10] 0 0
PK of ARO-RAGE: Apparent Systemic Clearance (CL/F)
Timepoint [10] 0 0
single dose phase: up to 48 hours post-dose; multiple dose phase: up to 6 hours post-dose on Days 1 and 29 for NHVs, and up to 24 hours post-dose for participants with asthma
Secondary outcome [11] 0 0
PK of ARO-RAGE: Apparent Terminal-Phase Volume of Distribution (VZ/F)
Timepoint [11] 0 0
single dose phase: up to 48 hours post-dose; multiple dose phase: up to 6 hours post-dose on Days 1 and 29 for NHVs, and up to 24 hours post-dose for participants with asthma
Secondary outcome [12] 0 0
PK of ARO-RAGE: Recovery of Unchanged Drug in Urine Over 0 to 24 Hours (Amount Excreted; Ae)
Timepoint [12] 0 0
Through 24 hours post-dose
Secondary outcome [13] 0 0
PK of ARO-RAGE: Percentage of Administrated Drug Recovered in Urine Over 0 to 24 hours
Timepoint [13] 0 0
Through 24 hours post-dose
Secondary outcome [14] 0 0
PK of ARO-RAGE: Renal Clearance (CLr)
Timepoint [14] 0 0
single dose phase: up to 48 hours post-dose; multiple dose phase: up to 6 hours post-dose on Days 1 and 29 for NHVs, and up to 24 hours post-dose for participants with asthma

Eligibility
Key inclusion criteria
- Normal pulmonary function tests at Screening (NHVs only)

- Confirmed diagnosis of asthma based on source verifiable medical record (asthma
patients only)

- No abnormal finding of clinical relevance at Screening (other than asthma for asthma
patients)

- Stable dose of asthma controller medications prior to Screening (asthma patients only)

- If on allergen-specific immunotherapy, participants must be on a stable maintenance
dose

- Non-smoking

- 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
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
- Acute lower respiratory infection or asthma exacerbation within 30 days prior to first
dose

- Positive COVID-19 test during Screening window

- Use of immunosuppressive medication within 90 days prior to first dose

- Receipt of any intranasal vaccine within 30 days prior to first dose

- Use of systemic corticosteroid therapy within 90 days prior to first dose

- 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
29/06/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/02/2025
Actual
Sample size
Target
149
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
WA
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 of
ARO-RAGE in normal healthy volunteers (NHVs) and in participants with inflammatory lung
disease (asthma). In Part 1 of the study, NHVs will receive a single dose of ARO-RAGE or
placebo. In Part 2 of the study, adult participants with asthma will receive 2 doses of
ARO-RAGE or placebo. Additional NHVs may be randomized to receive 1 or 2 doses of ARO-RAGE or
placebo at Sponsor discretion. Dose levels in Part 2 will be determined based on cumulative
safety and pharmacodynamic data from Part 1.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05276570
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
ARORAGE@arrowheadpharma.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05276570