Study of ARO-RAGE in Healthy Subjects and Patients With Inflammatory Lung Disease

NCT05276570 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: ARORAGE-1001
• Study Type: interventional
• Target: 127
• Locations: 5 countries
• Sites: 8

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.

Conditions

Asthma

Outcome Measures

Primary Outcomes (1)

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

  From first dose of study drug through the end of study (EOS; up to 113 days)

Secondary Outcomes (14)

• Change from Baseline Over Time in Forced Expiratory Volume (FEV1)

  Baseline through EOS (up to 113 days) or until serum soluble receptor for advance glycation end products (sRAGE) is ≥ 70% of baseline value

• Change from Baseline Over Time in Forced Vital Capacity (FVC)

  Baseline through EOS (up to 113 days) or until serum sRAGE is ≥ 70% of baseline value

• Change from Baseline Over Time in Diffusing Capacity for Carbon Monoxide (DLCO)

  Baseline through EOS (up to 113 days) or until serum sRAGE is ≥ 70% of baseline value

• PK of ARO-RAGE: Maximum Observed Plasma Concentration (Cmax)

  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

• PK of ARO-RAGE: Time to Maximum Observed Plasma Concentration (Tmax)

  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

Study Arms (2)

ARO-RAGE

EXPERIMENTAL

ARO-RAGE Inhalation

Drug: ARO-RAGE

Placebo

PLACEBO COMPARATOR

(0.9% NaCl)

Drug: Placebo

Interventions

ARO-RAGE DRUG

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

ARO-RAGE

Placebo DRUG

calculated volume to match active treatment by inhalation of nebulized solution

Placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Arrowhead Pharmaceuticals: lead

Study Sites (8)

Research Site 1

Nedlands, Washington, 6009, Australia

Location

Research Site 1

Auckland, 1010, New Zealand

Location

Research Site 2

Auckland, 1051, New Zealand

Location

Research Site 3

Auckland, 622, New Zealand

Location

Research Site 2

Krakow, 31-455, Poland

Location

Research Site 3

Oświęcim, 32-600, Poland

Location

Research Site 1

Barcelona, 08017, Spain

Location

Research Site 1

Bangkok Noi, Bangkok, 10700, Thailand

Location

MeSH Terms

Conditions

Asthma

Condition Hierarchy (Ancestors)

Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Respiratory Hypersensitivity; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 3, 2022
• First Posted: March 11, 2022
• Study Start: June 29, 2022
• Primary Completion: April 16, 2025
• Study Completion: April 16, 2025
• Last Updated: April 9, 2026

Record last verified: 2026-04

Locations

NZ (3); ES (1); TH (1); PL (2); AU (1)