NCT02835742已完成2 期
Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 78
- 试验地点
- 1
- 主要终点
- Change value of AaDO2 between baseline and 24 weeks
研究概览
简要总结
Objective: Determine the safety and efficacy of GM-CSF inhalation in patients with aPAP.
Study Design: multi-center, randomized, double-blind, placebo- controlled, safety/efficacy study.
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
- 16 Years 至 80 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age over 16 years and below 80 years (as of the date of registration).
- •Can provide signed informed consent.
- •Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures specified in the protocol (including short-term hospital admission).
- •Autoimmune pulmonary alveolar proteinosis diagnosed by both HR-CT and biopsy and/or BAL as well as GM-CSF antibodies in serum positive.
- •PaO2 < 70 mmHg after 5 minutes spine position at room air, or PaO2 < 75 mmHg after 5 minutes spine position at room air and with symptom(s) including cough, sputum and exertional dyspnea
排除标准
- •Diagnosed as secondary or hereditary pulmonary alveolar proteinosis
- •WBC of 12,000/mm3 or more
- •Fever of 38 degree celsius or more
- •Severe edema
- •History of malignant disease within recent 5 years (not applied to the treated cases of uterine carcinoma in situ and local basal cell carcinoma)
- •Complication of cardiovascular diseases including congestive heart failure, angina pectoris, hemorrhagic tendency, etc with severe condition.
- •Complication of respiratory diseases such as pulmonary infectious disease(incl. pulmonary tuberculosis), bronchial asthma, lung fibrosis ,interstitial pneumonitis, or bronchiectasis, in which the evaluations of safety and efficacy of GM-CSF therapy are considered as difficult.
- •History or complication of infectious diseases which require systemic administration of antibiotics, antifungal or antiviral agents within recent 2 weeks.
- •Treatment with other cytokines
- •Pregnant or possibly pregnant women, lactating women, and women who desire to become pregnant during the study period
- •Patients who have been treated with whole-lung lavage, repeated segmental-lung lavage, or rituximab within 6 months before the start of the study (this criterion does not apply to patients for whom 6 months or more have elapsed after their last lavage or rituximab)
- •Severe liver dysfunction (AST > 100 IU/L and/or ALT > 100 IU/L and/or T-bil >3.0mg/dL)
- •Severe renal dysfunction (Ccr < 30 mL/min, calculated by Cockcroft-Gault (CG) formula)
- •Previous experience of severe and unexplained side-effects during aerosol delivery of any kind of medicinal product
- •Treatment with oral or intravenous administration or inhalation of corticosteroids.
- •Treatment with other inhaled drugs.
- •Previously treated with GM-CSF before the start of the study.
- •Demonstrate hypersensitivity to GM-CSF agent.
- •Other patients judged to be inappropriate for the study by the attending physician (e.g., patients who are unlikely to complete treatment or are uncooperative).
研究组 & 干预措施
Group1
Active Comparator
Treatments for Group 1 include GM-CSF inhalation with 250 mcg/day/body of sargramostim (125 mcg BID on Days 1-7, none on Days 8-14) for twelve 2-week cycles.
干预措施: Sargramostim (Drug)
Group2
Placebo Comparator
Treatments for Group 2 include placebo inhalation (Placebo BID on Days 1-7, none on Days 8-14) for twelve 2-week cycles.
干预措施: Placebo (Drug)
结局指标
主要结局
Change value of AaDO2 between baseline and 24 weeks
时间窗: 24 weeks
次要结局
未报告次要终点
研究者
研究点 (1)
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