A Phase 2b, 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
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 63
- 试验地点
- 14
- 主要终点
- Change in sputum Pseudomonas aeruginosa (PsA) burden at 8 weeks (EOT)
研究概览
简要总结
The goal of this Phase 2b clinical trial is to see if nebulized phage (BX004) can treat chronic Pseudomonas aeruginosa (PsA) lung infection in CF subjects. The primary goal is to see if 8 weeks of twice daily BX004 can reduce the amount of PsA in the sputum compared to placebo (on top of background CF therapy).
详细描述
This is a randomized, double-blind, placebo-controlled, multicenter study to evaluate BX004 in CF subjects with chronic PsA pulmonary infection. The main purpose of the study is to evaluate whether BX004 reduces the PsA burden in the sputum of CF subjects with chronic PsA pulmonary infection. Secondary endpoints are to see how well BX004 works in improving lung function and quality of life, reducing the amount of PsA in the sputum, getting negative sputum cultures for PsA, and safety and tolerability. Clinically stable CF subjects with a confirmed diagnosis of CF and chronic PsA pulmonary infection will be enrolled. Subjects will be included in a 6-month post-dose safety follow-up. A Data Safety Monitoring Board of the CF Foundation will monitor safety.
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 inhaled antibiotics (cycling or continuous regimen) or no inhaled antibiotics
- •Age ≥ 18 years
- •FEV1 40%-80% 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 or Day 1
- •Detection of Burkholderia cenocepacia from respiratory tract within 1 year prior to Screening or from Screening culture
- •Currently receiving systemic treatment for allergic bronchopulmonary aspergillosis
- •Currently receiving treatment for active infection with non-tuberculous mycobacteria or prior detection of Mycobacterium abscessus in 12 months prior to Screening
- •History of severe neutropenia
- •History of lung transplant
- •History of solid organ transplant
- •Acquired or primary immunodeficiency syndrome
- •Initiation or change in type of CFTR modulator less than 3 months prior to Screening
- •Pregnant or breastfeeding female
研究组 & 干预措施
BX004
Participants will be randomized to receive standard dose of nebulized bacteriophage
干预措施: BX004 (Biological)
Placebo
Participants will be randomized to receive nebulized placebo
干预措施: Placebo (Other)
结局指标
主要结局
Change in sputum Pseudomonas aeruginosa (PsA) burden at 8 weeks (EOT)
时间窗: 8 weeks
Change from baseline in PsA colony-forming units (CFU) per g of sputum
次要结局
- Change in lung function at D8, D29, D43, D57 (EOT), D85, 3 months post-dose, and 6 months post-dose(from Day 8 until 6 months after last dose (end of study))
- Change from Baseline in CFQ-R respiratory domain(until 6 months after last dose of study drug)
- Change from Baseline in CFRSD-CRISS(until 6 months after last dose of study drug)
- Change in sputum PsA burden(until 6 months after last dose of study drug)
- Efficacy of BX004 on obtaining negative sputum cultures for PsA(until 6 months after last dose of study drug)
- Incidence of treatment-emergent adverse events [safety and tolerability](until 6 months after last dose of study drug)