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
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 40
- 试验地点
- 10
- 主要终点
- Incidence of treatment-emergent adverse events
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of NCFBE confirmed by chest CT
- •18-80 years old, inclusive at time of informed consent
- •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)
排除标准
- 未提供
研究组 & 干预措施
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
干预措施: ARINA-1 (Drug)
Placebo
Isotonic saline (0.9%); 4 mL solution inhaled twice daily via nebulization for 28 days
干预措施: Placebo (Drug)
结局指标
主要结局
Incidence of treatment-emergent adverse events
时间窗: 56 days
Incidence of participants that experience an adverse event following administration of treatment
Proportion of participants that experience each treatment-emergent adverse event
时间窗: 56 days
次要结局
- Compare changes in airway clearance techniques between ARINA-1 and placebo arms(56 days)
- Compare FEV1 between the ARINA-1 and treatment arms(56 days)
- Compare quality of life (as measured by CAAT questionnaire) between the ARINA-1 and placebo arms(56 days)
- Compare quality of life (as measured by SGRQ) between the ARINA-1 and placebo arms(56 days)
- Compare quality of life (as measured by QOL-B) between the ARINA-1 and placebo arms(56 days)
- Compare changes in mucolytic use between ARINA-1 and placebo arms(56 days)
- Compare blood inflammatory markers between the ARINA-1 and placebo arms(56 days)
- Compare FVC between the ARINA-1 and treatment arms(56 days)
- Compare FEF25-75 between the ARINA-1 and treatment arms(56 days)
- Compare PEF between the ARINA-1 and treatment arms(56 days)
- Compare sputum percent solids between the ARINA-1 and placebo arms(56 days)