A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 447
- 试验地点
- 105
- 主要终点
- Pulmonary Exacerbation (PEx) Rate Over 28 Weeks
研究概览
简要总结
This is a Phase 2 multicenter, double-blind, randomized, placebo-controlled study assessing the efficacy and safety of lenabasum for the treatment of cystic fibrosis in patients 12 years of age or older. Approximately 415 subjects will be enrolled in this study at about 100 sites in North America, and Europe. The planned duration of treatment with study drug is 28 weeks.
Study drug will be lenabasum 20 mg BID, lenabasum 5 mg BID, and placebo in a 2:1:2 ratio.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •≥ 12 years of age at the time Informed Consent/ Assent is signed.
- •Weight ≥ 40 kg.
- •FEV1 ≥ 40% predicted and < 100% predicted in the last 12 months.
- •Physician-initiated treatment with an IV antibiotic 2 or 3 times in the last 12 months for a new PEx or physician-initiated treatment with an IV antibiotic 1 time in the last 12 months plus physician-initiated treatment with oral antibiotic(s) 1 or more times in the past 12 months for a new PEX.
排除标准
- •Severe or unstable CF at screening or Visit
- •Any of the following values for laboratory tests at screening:
- •A positive pregnancy test.
- •Hemoglobin < 10 g/dL in males and < 9 g/dL in females.
- •Neutrophils < 1.0 x 10^9 /L.
- •Platelets < 75 x 10^9/L.
- •Creatinine clearance < 50 mL/min according to Modification of Diet in Renal Disease (MDRD) Study equation.
- •Serum transaminases > 2.5 x upper limit of normal.
- •Any medical condition or concurrent medical therapies at screening or Visit 1 that may put the subject at greater safety risk, influence response to study drug or interfere with study assessments.
研究组 & 干预措施
Lenabasum 20 mg BID
干预措施: Lenabasum 20 mg (Drug)
Lenabasum 5 mg BID
干预措施: Lenabasum 5 mg (Drug)
Placebo BID
干预措施: Placebo (Other)
结局指标
主要结局
Pulmonary Exacerbation (PEx) Rate Over 28 Weeks
时间窗: 28 weeks (Baseline Day 0 to Week 28)
Rate of PEx using the primary PEx definition with lenabasum 20 mg BID compared to placebo, during the treatment period. An primary PEx is defined on the physician's decision to treat with oral, intraveneous or inhaled antibiotics in the presence 4/12 Fuch's criteria (Change in sputum, new/increased hemoptysis, increased cough, increased dyspnea, malaise, fatigue/lethargy, Temperature greater than 38C, weight loss, sinus pain, change in sinus discharge, change in exam of chest, decrease in FEV1 of more than 10%, radiographic change). This excludes prophylactic antibiotics. A new PEx is a one that occurs at 28 days after the previous PEx.
次要结局
- Pulmonary Exacerbation (PEx) Rate(28 weeks (Baseline Day 0 to Week 28))
- Time to First New Pulmonary Exacerbation (PEx)(28 weeks (Baseline Day 0 to Week 28))
- Pulmonary Exacerbation (PEx)(28 weeks (Baseline Day 0 to Week 28))
- CFQ-R Respiratory Symptom Domain(28 weeks (Change from Baseline Day 0 to Week 28))
- FEV1 % Predicted(28 weeks (Change from Baseline Day 0 to Week 28))