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

Phase 2

Completed

Brief Summary: 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.

Recruitment & Eligibility

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)

Exclusion Criteria

Not provided

Arm && Interventions

Group	Intervention	Description
ARINA-1	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
Placebo	Placebo	Isotonic saline (0.9%); 4 mL solution inhaled twice daily via nebulization for 28 days

Primary Outcome Measures

Name	Time	Method
Incidence of treatment-emergent adverse events	56 days	Incidence of participants that experience an adverse event following administration of treatment
Proportion of participants that experience each treatment-emergent adverse event	56 days

Secondary Outcome Measures

Name	Time	Method
Compare changes in airway clearance techniques between ARINA-1 and placebo arms	56 days	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.
Compare FEV1 between the ARINA-1 and treatment arms	56 days	measured in L
Compare quality of life (as measured by CAAT questionnaire) between the ARINA-1 and placebo arms	56 days	8 questions rated on a 0-5 scale
Compare quality of life (as measured by SGRQ) between the ARINA-1 and placebo arms	56 days	42 questions, ranked on a 5-point descriptive scale or true/false
Compare quality of life (as measured by QOL-B) between the ARINA-1 and placebo arms	56 days	37 questions ranked on a 4-point descriptive scale
Compare changes in mucolytic use between ARINA-1 and placebo arms	56 days	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.
Compare blood inflammatory markers between the ARINA-1 and placebo arms	56 days	C-reactive protein
Compare FVC between the ARINA-1 and treatment arms	56 days	measured in L
Compare FEF25-75 between the ARINA-1 and treatment arms	56 days	measured in L/sec
Compare PEF between the ARINA-1 and treatment arms	56 days	measured in L/min
Compare sputum percent solids between the ARINA-1 and placebo arms	56 days

Locations (10): Louisiana State University
🇺🇸
New Orleans, Louisiana, United States
Georgetown University
🇺🇸
Washington, District of Columbia, United States
Medical University of South Carolina
🇺🇸
Charleston, South Carolina, United States
University of Texas - Tyler
🇺🇸
Tyler, Texas, United States
University of Alabama - Birmingham
🇺🇸
Birmingham, Alabama, United States
Johns Hopkins
🇺🇸
Baltimore, Maryland, United States
Oregon Health and Science University
🇺🇸
Portland, Oregon, United States
National Jewish Hospital
🇺🇸
Denver, Colorado, United States
Jefferson Hospital
🇺🇸
Philadelphia, Pennsylvania, United States
Southeastern Research Center
🇺🇸
Winston-Salem, North Carolina, United States