Phase 2a, 28-day Investigational Use Study of ARINA-1 in Non-CF Bronchiectasis With Excess Mucus and Cough

Non-cystic Fibrosis Bronchiectasis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05495243
• Other study ID #: RVN-301
• Status: Completed
• Phase: Phase 2
• Start date: October 3, 2022
• Est. completion date: March 1, 2024

Study information

• Verified date: January 2024
• Source: Renovion, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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 information / eligibility

• Status: Completed
• Enrollment: 40
• Est. completion date: March 1, 2024
• Est. primary completion date: January 11, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 80 Years

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)

Related Conditions & MeSH terms

• Bronchiectasis
• Non-cystic Fibrosis Bronchiectasis

Intervention

Drug:
• ARINA-1
ARINA-1 (88 mg/mL ascorbic acid, ASC; 150 mg/mL reduced glutathione, GSH)
• Placebo
Isotonic saline (0.9%)

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Renovion, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of treatment-emergent adverse events	Incidence of participants that experience an adverse event following administration of treatment	56 days
Primary	Proportion of participants that experience each treatment-emergent adverse event		56 days
Secondary	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
Secondary	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
Secondary	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
Secondary	Compare blood inflammatory markers between the ARINA-1 and placebo arms	C-reactive protein	56 days
Secondary	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
Secondary	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
Secondary	Compare FEV1 between the ARINA-1 and treatment arms	measured in L	56 days
Secondary	Compare FVC between the ARINA-1 and treatment arms	measured in L	56 days
Secondary	Compare FEF25-75 between the ARINA-1 and treatment arms	measured in L/sec	56 days
Secondary	Compare PEF between the ARINA-1 and treatment arms	measured in L/min	56 days
Secondary	Compare sputum percent solids between the ARINA-1 and placebo arms		56 days