2024-512039-66-00招募中3 期
An Open-label, Multicenter Clinical Study to Evaluate the Efficacy and Safety of Inhaled Molgramostim in Pediatric Subjects with Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- Savara ApS
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Change in Hb-adjusted % predicted DLCO from Baseline to Week 24.
研究概览
简要总结
To investigate efficacy of inhaled molgramostim in participants with aPAP, from 6 years to less than 18 years of age.
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
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 prior to screening.
- •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 WLL within 1 month of Baseline.
结局指标
主要结局
Change in Hb-adjusted % predicted DLCO from Baseline to Week 24.
Change in Hb-adjusted % predicted DLCO from Baseline to Week 24.
次要结局
- Change in Hb-adjusted % predicted DLCO from Baseline to Week 48.
- Absolute change from Baseline in oxygen saturation (SpO2) at rest after 24 and 48 weeks of treatment.
- Absolute change from Baseline in 6-minute walk distance (6MWD) after 24 and 48 weeks of treatment.
- Change from Baseline in Pediatric Quality of Life (PedsQLTM) Generic Core Scale score after 24 and 48 weeks of treatment.
- AEs, including clinically significant findings on pulmonary function tests and safety laboratory assessments and adverse events of special interest (AESIs; hypersensitivity and chest pain).
- Titers of anti-GM-CSF antibodies at Baseline and after 4, 12, 24, 48, and 52 weeks.
- Change from Baseline in forced expiratory volume in one second (FEV1) (% predicted) and forced vital capacity (FVC) (% predicted) after 24 and 48 weeks of treatment.
研究者
Savara ApS
Scientific
Savara ApS
研究点 (1)
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