An Open-label, Multicenter Clinical Study to Evaluate the Efficacy and Safety of Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- Savara Inc.
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- DLCO
研究概览
简要总结
The goal of this open-label study is to study molgramostim as a treatment for autoimmune pulmonary alveolar proteinosis (aPAP) in pediatric patients between age 6 and 18. The main questions it aims to answer are:
The effect of molgramostim on breathing tests and activity in pediatric patients with aPAP and the safety of molgramostim in pediatric patients with aPAP.
This is an open-label study: all participants will receive treatment with molgramostim.
Patients will:
- Take molgramostim once daily via nebulizer every day for 12 months.
- Visit the clinic approximately every 12 weeks for checkups and tests.
- Keep a diary of any oxygen use.
详细描述
This is an interventional open-label, single arm, multi-center study in pediatric subjects, age 6 through 18 years, who are diagnosed with autoimmune pulmonary alveolar proteinosis (aPAP).
The diagnosis of aPAP should be confirmed by an anti-GM-CSF antibody test and a history compatible with PAP based on patient symptoms, high resolution computed tomography of the lung, lung biopsy or bronchoalveolar lavage cytology.
The study consists of a 4-week screening period followed by a 48-week open-label treatment period. After completing the 48-week treatment or early withdrawal, subjects will enter a 4-week safety follow up period. The maximum treatment duration is 48-weeks, and the maximum study period will be 56 weeks. During the trial, lung lavage will be allowed as a rescue treatment in case of worsening of aPAP.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Be ≥6 and <18 years of age, at the time of signing the informed consent and informed assent (if applicable).
- •Have a history of pulmonary alveolar proteinosis, based on examination of a lung biopsy, bronchoalveolar lavage cytology, or a high-resolution computed tomogram of the chest.
- •Have a positive serum anti-GM-CSF autoantibody test result confirming aPAP.
- •Have a hemoglobin (Hb)-adjusted diffusing capacity of the lung for carbon monoxide (DLCO) ≤70% predicted at Screening.
排除标准
- •Have a diagnosis of hereditary (congenital) or secondary PAP, or a metabolic disorder of surfactant production.
- •Have undergone treatment with Lung Lavage (WLL) within 1 month of Baseline
研究组 & 干预措施
molgramostim
Molgramostim 300 mcg administered once daily via nebulizer for 48 weeks.
干预措施: Molgramostim (Drug)
结局指标
主要结局
DLCO
时间窗: 24 weeks
Change in Hb-adjusted % predicted DLCO from Baseline.
次要结局
- 6-minute walk distance(48-weeks)
- Oxygen Saturation (SpO2)(48 weeks)
- DLCO(48-weeks)
- PedsQL(48-weeks)
- 6-minute walk distance(24-weeks)
- PedsQL(24-weeks)
- Oxygen Saturation (SpO2)(24 weeks)