A Phase 1b/2a, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate Nebulized Bacteriophage Treatment in Outpatient Adult Cystic Fibrosis (CF) Subjects With Chronic Pseudomonas Aeruginosa (PsA) Pulmonary Infection
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- BiomX, Inc.
- 入组人数
- 43
- 试验地点
- 28
- 主要终点
- Safety and tolerability
研究概览
简要总结
This is a Phase 1b/2a study with the primary objective to determine if BX004-A is safe and tolerable. Exploratory objectives include whether BX004-A reduces sputum Pseudomonas aeruginosa (PsA) bacterial load in CF subjects with chronic PsA pulmonary infection.
详细描述
This is a randomized, double-blind, placebo-controlled, multicenter study to evaluate the safety and tolerability of BX004-A in CF subjects with chronic PsA pulmonary infection. The study is divided into two parts, a single-ascending and multiple-dose phase (Part 1) and a multiple dose phase (Part 2). Subjects in both parts will be included in a 6-month safety follow-up. A Data Safety Monitoring Board will monitor safety in both parts. The purpose of the study is to evaluate safety and tolerability of BX004-A, and whether BX004-A reduces the PsA burden in the sputum of CF subjects with chronic PsA pulmonary infection. Clinically stable CF subjects with a confirmed diagnosis of CF and chronic PsA pulmonary infection will be enrolled.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Cystic fibrosis patients with chronic Pseudomonas aeruginosa pulmonary infection receiving standard of care CF medications
- •Age ≥ 18 years
- •FEV1 ≥ 40% predicted
- •Clinically stable lung disease
- •Willing and able to provide adequate sputum samples, using any method (spontaneously expectorated, induced, from home or clinic) at designated study visits.
排除标准
- •Known hypersensitivity to bacteriophages or excipients in the formulation.
- •Receipt of prior bacteriophage therapy within the 6 months prior to Screening
- •Recovery of Burkholderia species from respiratory tract within 1 year prior to screening
- •Currently receiving treatment for allergic bronchopulmonary aspergillosis
- •Currently receiving treatment for active infection with non-tuberculous mycobacteria
- •History of severe neutropenia
- •History of lung transplant
- •History of solid organ transplant
- •Acquired or primary immunodeficiency syndrome
- •Initiation or change in CF modulator therapy less than 3 months prior to screening
- •Pregnant or breastfeeding female
研究组 & 干预措施
BX004-A
Participants will be randomized to receive standard dose of nebulized bacteriophage
干预措施: BX004-A (Drug)
Placebo
Participants will be randomized to receive nebulized placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Safety and tolerability
时间窗: 6 months
Incidence of treatment emergent adverse events following single and multiple doses of BX004-A administered by inhalation
次要结局
未报告次要终点