Study of RHB-204 for the Treatment of Pulmonary Mycobacterium Avium Complex (MAC) Disease in Adults With Nodular Bronchiectasis (CleaR-MAC Trial)
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 12
- 试验地点
- 3
- 主要终点
- Quality of Life Questionnaire - Bronchiectasis (QoL B) Respiratory Symptoms domain score from baseline to Month 6 for RHB-204 compared to placebo
研究概览
简要总结
A 2-part multi-center, Phase 3, randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of RHB-204 in adult subjects with underlying nodular bronchiectasis and documented MAC lung infection.
详细描述
A 2-part multi-center, Phase 3, randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of RHB-204 in adult subjects with underlying nodular bronchiectasis and documented MAC lung infection.
The co-primary efficacy endpoints at the end of Part 1 evaluate the proportion of patients with sputum culture conversion after 6 months of treatment defined as three consecutive monthly negative sputum cultures at Months 4, 5, and 6 for RHB-204 compared to placebo and the mean change in the Quality of Life Questionnaire - Bronchiectasis (QoL-B) Respiratory Symptoms domain score from baseline to Month 6 for RHB-204 compared to placebo.
At the Month 6 visit (end of Part 1), after all assessments including questionnaires and sputum samples have been collected, subjects will enter Part 2 of the study and receive open-label RHB-204.
In order to comply with current treatment duration guidelines (Nontuberculous Mycobacterial Pulmonary Disease Caused by Mycobacterium avium Complex: Developing Drugs for Treatment - FDA Draft Guidance September 2021) in this protocol subjects will receive active treatment for 12 months after sputum culture conversion. As such, each subject's first negative culture that defines conversion will determine the total duration of their active study drug treatment. The longest duration of active study drug treatment will be 16 months. At the end of study treatment, all subjects will enter a 3-month post-treatment follow-up period (see study design section below for additional details).
To maintain the blind and integrity of the data, an independent team (Sputum Culture Monitors) will inform sites on each subject's treatment duration based on sputum culture conversion. Sputum Culture Monitors will also have access to randomized treatment allocation. All other study related personnel will remain blinded to sputum culture results and treatment allocation (Sponsor, CRO, medical monitors, safety, ECG and sputum laboratories, site personnel as well as participating subjects).
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 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
RHB-204
Each capsule contains clarithromycin 158.3mg; rifabutin 40mg; clofazimine 13.3mg.
干预措施: RHB-204 (Drug)
Placebo
Matching placebo will contain riboflavin, a type of B vitamin, which may discolor urine in a similar fashion as RHB-204.
干预措施: Placebo (Drug)
结局指标
主要结局
Quality of Life Questionnaire - Bronchiectasis (QoL B) Respiratory Symptoms domain score from baseline to Month 6 for RHB-204 compared to placebo
时间窗: 6 months
The mean change in the Quality of Life Questionnaire - Bronchiectasis (QoL B) Respiratory Symptoms domain score from baseline to Month 6 for RHB-204 compared to placebo
Sputum culture conversion (SCC)
时间窗: 6 months
The proportion of subjects who achieve SCC by Month 6, defined by 3 consecutive monthly negative sputum cultures, without reversion, at Months 4, 5 \& 6 for THB-204 compared to placebo.
次要结局
- Part 1 Secondary efficacy objective - Reduction of fatigue(6 months)
- Part 1 Secondary efficacy objective - Time to culture conversion(6 months)
- Part 1 Secondary efficacy objective - Improvement in Physical Functioning(6 months)
- Part 2 Secondary efficacy objective - Durable Sputum culture conversion at end of study(19 months)
- Part 2 Secondary efficacy objective - Durable Sputum culture conversion at end of treatment(16 months)
- QoL B Respiratory Symptoms domain score mean change from baseline to Month 16(16 months)
- Part 2 Secondary efficacy objective - Improvement in Quality of Life - Fatigue 16 months(16 months)
- Part 2 Secondary efficacy objective - Improvement in Quality of Life - Fatigue 19 Months(19 months)
- Part 2 Secondary efficacy objective - Quality of Life Physical Functioning Symptoms 19 Months(19 Months)
- Part 2 Secondary efficacy objective - Quality of Life Physical Functioning Symptoms 16 Months(16 months)
- QoL-B Respiratory Symptoms domain scores at Month 19(19 Months)