Phase 2a, 28-day Investigational Use Study of ARINA-1 in Non-Cystic Fibrosis Bronchiectasis (NCFBE) With Excess Mucus and Cough

A Phase 2a, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety and Efficacy of ARINA-1 in Adult Participants With Non-cystic Fibrosis Bronchiectasis (NCFBE) With Excess Mucus and Cough

This is a randomized, Phase 2a, double-blind, placebo-controlled study to evaluate the safety and efficacy of ARINA-1 compared to placebo (isotonic saline, 0.9%) in participants with NCFBE. Study participants will receive either study treatment or placebo twice daily for 28 days. Efficacy endpoints will include quality of life, sputum rheological markers, and blood inflammatory markers. Quality of life will be measured using the following tools: Quality of Life-Bronchiectasis (QOL-B) questionnaire, St. George's Respiratory Questionnaire (SGRQ), Chronic Airways Assessment Test (CAAT), and a daily clinical global impression questionnaire.

Study Overview

• Status: Completed
• Conditions: Non-cystic Fibrosis Bronchiectasis
• Intervention / Treatment: Drug: ARINA-1; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 40
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35294
      • University of Alabama - Birmingham
  • Colorado
    • Denver, Colorado, United States, 80206
      • National Jewish Hospital
  • District of Columbia
    • Washington, District of Columbia, United States, 20007
      • Georgetown University
  • Louisiana
    • New Orleans, Louisiana, United States, 70803
      • Louisiana State University
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Johns Hopkins
  • North Carolina
    • Winston-Salem, North Carolina, United States, 27103
      • Southeastern Research Center
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health and Science University
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19107
      • Jefferson Hospital
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina
  • Texas
    • Tyler, Texas, United States, 29425
      • University of Texas - Tyler

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Diagnosis of NCFBE confirmed by chest CT
• 18-80 years old, inclusive at time of informed consent
• BMI >18
• Percent predicted FEV1 > 40%, pre-bronchodilator
• Stable for 90 days with any airway clearance technique (ACT) method(s)
• Stable on standard of care (SOC) therapy for 90 days (includes cycling antibiotics, such as inhaled tobramycin, and long-term macrolide therapy) and not likely, in the opinion of the investigator, to require any changes to therapy during the duration of study participation
• Fully vaccinated for COVID-19 (second dose of a 2-shot regimen or single dose of 1-shot regimen completed >14 days prior to the screening visit)
• Must be able to produce a sputum sample
• If female and of childbearing potential, must be willing to use contraception for the duration of the study.

Exclusion Criteria

• Positive urine pregnancy test for women of childbearing potential (WOCBP) at screening and baseline visit
• Active exacerbation ≤28 days prior to the baseline visit
• Initiating or changing antibiotic, antiviral, or antifungal therapy ≤ 28 days prior to the baseline visit
• Changing or initiating any vitamin C, glutathione or N-acetyl-cysteine-containing therapy or multivitamin within 30 says prior to the screening visit.
• Positive COVID-19 diagnostic test (PCR or antigen) within 90 days prior to the screening visit.
• Participated in other interventional drug or device studies within 30 days of the screening visit (Note: observational studies are acceptable)
• Significant unstable comorbidities (in the opinion of the site investigator), such as heart failure, cardiovascular disease, diabetes, renal disease, liver disease
• Current tobacco or marijuana smoker (those with active smoking exposure <180 days prior to the screening visit) (Note: edibles are acceptable)
• Requiring the use of any supplemental oxygen
• Currently on cycled antibiotics (e.g., tobramycin) or have been on a cycled antibiotic regimen within 90 days prior to the screening visit.
• Current diagnosis of non-tuberculous mycobacteria (NTM) requiring antibiotic treatment. (Participants who test positive for NTM but who are not currently on antibiotic therapy are eligible for screening)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ARINA-1; ARINA-1 (88 mg/mL ascorbic acid, ASC; 150 mg/mL reduced glutathione, GSH); fixed dose, 4 mL solution inhaled twice daily via nebulization for 28 days	Drug: ARINA-1; ARINA-1 (88 mg/mL ascorbic acid, ASC; 150 mg/mL reduced glutathione, GSH)
Placebo Comparator: Placebo; Isotonic saline (0.9%); 4 mL solution inhaled twice daily via nebulization for 28 days	Drug: Placebo; Isotonic saline (0.9%)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of treatment-emergent adverse events	Incidence of participants that experience an adverse event following administration of treatment	56 days
Proportion of participants that experience each treatment-emergent adverse event		56 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Compare quality of life (as measured by CAAT questionnaire) between the ARINA-1 and placebo arms	8 questions rated on a 0-5 scale	56 days
Compare quality of life (as measured by SGRQ) between the ARINA-1 and placebo arms	42 questions, ranked on a 5-point descriptive scale or true/false	56 days
Compare quality of life (as measured by QOL-B) between the ARINA-1 and placebo arms	37 questions ranked on a 4-point descriptive scale	56 days
Compare blood inflammatory markers between the ARINA-1 and placebo arms	C-reactive protein	56 days
Compare changes in mucolytic use between ARINA-1 and placebo arms	Compare the initiation or changes to regimen for drugs such as n-acetylcysteine, dornase alfa, etc. This information will be collected at all clinic visits and phone calls.	56 days
Compare changes in airway clearance techniques between ARINA-1 and placebo arms	Compare the initiation or changes to regimen for airway clearance techniques such as chest physical therapy, high-frequency oscillating vest therapy, etc. This information will be collected at all clinic visits and phone calls.	56 days
Compare FEV1 between the ARINA-1 and treatment arms	measured in L	56 days
Compare FVC between the ARINA-1 and treatment arms	measured in L	56 days
Compare FEF25-75 between the ARINA-1 and treatment arms	measured in L/sec	56 days
Compare PEF between the ARINA-1 and treatment arms	measured in L/min	56 days
Compare sputum percent solids between the ARINA-1 and placebo arms		56 days

Collaborators and Investigators

• Sponsor: Renovion, Inc.
• Investigators: Principal Investigator: Charles L Daley, MD, National Jewish Health

Study record dates

Study Major Dates

• Study Start (Actual): October 3, 2022
• Primary Completion (Actual): February 1, 2024
• Study Completion (Actual): February 1, 2024

Study Registration Dates

• First Submitted: June 24, 2022
• First Submitted That Met QC Criteria: August 8, 2022
• First Posted (Actual): August 10, 2022

Study Record Updates

• Last Update Posted (Actual): July 23, 2025
• Last Update Submitted That Met QC Criteria: July 21, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Keywords: bronchiectasis
• Additional Relevant MeSH Terms: Pathologic Processes; Respiratory Tract Diseases; Bronchial Diseases; Fibrosis; Bronchiectasis
• Other Study ID Numbers: RVN-301

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No