Clinical trial NCT05495243: Phase 2a, 28-day Investigational Use Study of ARINA-1 in Non-Cystic Fibrosis Bronchiectasis (NCFBE) With Excess Mucus and Cough
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-cystic Fibrosis Bronchiectasis (NCFBE) With Excess Mucus and Cough
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Non-cystic Fibrosis Bronchiectasis
Keywords: bronchiectasis
Lead sponsor: Renovion, Inc.
Sponsor class: INDUSTRY
First posted: 2022-08-10
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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)

Primary outcomes:
measure: Incidence of treatment-emergent adverse events
timeFrame: 56 days
measure: Proportion of participants that experience each treatment-emergent adverse event
timeFrame: 56 days
Secondary outcomes:
measure: Compare quality of life (as measured by CAAT questionnaire) between the ARINA-1 and placebo arms
timeFrame: 56 days
measure: Compare quality of life (as measured by SGRQ) between the ARINA-1 and placebo arms
timeFrame: 56 days
measure: Compare quality of life (as measured by QOL-B) between the ARINA-1 and placebo arms
timeFrame: 56 days
measure: Compare blood inflammatory markers between the ARINA-1 and placebo arms
timeFrame: 56 days
measure: Compare changes in mucolytic use between ARINA-1 and placebo arms
timeFrame: 56 days
measure: Compare changes in airway clearance techniques between ARINA-1 and placebo arms
timeFrame: 56 days
measure: Compare FEV1 between the ARINA-1 and treatment arms
timeFrame: 56 days
measure: Compare FVC between the ARINA-1 and treatment arms
timeFrame: 56 days
measure: Compare FEF25-75 between the ARINA-1 and treatment arms
timeFrame: 56 days
measure: Compare PEF between the ARINA-1 and treatment arms
timeFrame: 56 days
measure: Compare sputum percent solids between the ARINA-1 and placebo arms
timeFrame: 56 days
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama - Birmingham
state: Alabama
city: Denver
country: United States
facility: National Jewish Hospital
state: Colorado
city: Washington
country: United States
facility: Georgetown University
state: District of Columbia
city: New Orleans
country: United States
facility: Louisiana State University
state: Louisiana
city: Baltimore
country: United States
facility: Johns Hopkins
state: Maryland
city: Winston-Salem
country: United States
facility: Southeastern Research Center
state: North Carolina
city: Portland
country: United States
facility: Oregon Health and Science University
state: Oregon
city: Philadelphia
country: United States
facility: Jefferson Hospital
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Tyler
country: United States
facility: University of Texas - Tyler
state: Texas
Study officials:
affiliation: National Jewish Health
name: Charles L Daley, MD
role: PRINCIPAL_INVESTIGATOR
References: