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Completed

NCT Number: NCT05276570

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

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.

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Key information

Conditions

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

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Research Site 1, Nedlands, Washington, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

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

Treatment and study plan

ARO-RAGE

Drug

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

Placebo

Drug

calculated volume to match active treatment by inhalation of nebulized solution

Primary outcomes

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

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

Secondary outcomes

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

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

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

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

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

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

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

    Time frame: 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

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

    Time frame: 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

  6. PK of ARO-RAGE: Area Under the Plasma Concentration versus Time Curve From Zero to 24 Hours (AUC0-24)

    Time frame: 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

  7. PK of ARO-RAGE: 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 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

  8. PK of ARO-RAGE: Area Under the Plasma Concentration versus Time Curve From Zero to Infinity (AUCinf)

    Time frame: 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

  9. PK of ARO-RAGE: Terminal Elimination Half-Life (t1/2)

    Time frame: 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

  10. PK of ARO-RAGE: Apparent Systemic Clearance (CL/F)

    Time frame: 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

  11. PK of ARO-RAGE: Apparent Terminal-Phase Volume of Distribution (VZ/F)

    Time frame: 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

  12. PK of ARO-RAGE: Recovery of Unchanged Drug in Urine Over 0 to 24 Hours (Amount Excreted; Ae)

    Time frame: Through 24 hours post-dose

  13. PK of ARO-RAGE: Percentage of Administrated Drug Recovered in Urine Over 0 to 24 hours

    Time frame: Through 24 hours post-dose

  14. PK of ARO-RAGE: Renal Clearance (CLr)

    Time frame: 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

Sponsors and collaborators

Lead sponsor

Arrowhead Pharmaceuticals

Industry

Registry information

Official study title

A Phase 1/2a Study Evaluating the Effects of ARO-RAGE Inhalation Solution in Healthy Subjects and Patients With Inflammatory Lung Disease

Important dates

Study start
2022
Primary completion
2025
Study completion
2025
First posted
Mar 11, 2022
Registry last updated
Apr 9, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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