An Open-label, Non-controlled, Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 发起方
- Savara Inc.
- 入组人数
- 60
- 试验地点
- 13
- 主要终点
- Number of Treatment-emergent Adverse Events (TEAEs)
研究概览
简要总结
SAV006-03 is an open-label extension study for participants who had completed the IMPALA study.
At the baseline visit, eligible participants may continue or re-start treatment with 300 µg inhaled molgramostim (recombinant human Granulocyte-Macrophage Colony Stimulating Factor; GM-CSF) administered intermittently in cycles of seven days molgramostim, administered once daily, and seven days off treatment.
Participants will be treated with inhaled molgramostim for up to 36 months.
During the trial, whole lung lavage will be applied as rescue therapy.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completer of the IMPALA trial.
- •Females who have been post menopausal for >1 year, or females of child-bearing potential who are not pregnant or lactating and are using acceptable contraceptive methods.
- •Males agreeing to use using acceptable contraceptive methods.
- •Willing and able to provide signed informed consent.
排除标准
- •Treatment with GM-CSF products other than molgramostim nebuliser solution within three months of Baseline.
- •Treatment with any investigational medicinal product other than inhaled molgramostim within four weeks of Baseline.
- •History of allergic reactions to GM-CSF.
- •Connective tissue disease, inflammatory bowel disease or other autoimmune disorder requiring treatment associated with significant immunosuppression, e.g. more than 10 mg/day systemic prednisolone.
- •Previous experience of severe and unexplained side effects during aerosol delivery of any kind of medicinal product.
- •History of, or present, myeloproliferative disease or leukaemia.
- •Apparent pre-existing concurrent pulmonary fibrosis.
- •Any other serious medical condition which in the opinion of the investigator would make the subject unsuitable for the trial.
研究组 & 干预措施
Molgramostim nebulizer solution (300 μg)
Open-label treatment with molgramostim nebulizer solution (300 μg) administered intermittently (repetitive cycles of 7 days of treatment followed by 7 days off-treatment).
干预措施: Molgramostim (Drug)
结局指标
主要结局
Number of Treatment-emergent Adverse Events (TEAEs)
时间窗: 139 weeks
The primary objective of this trial was safety assessed by adverse event (AE) reporting. Definitions and reporting procedures for AEs were done according to current regulatory standards. AEs were collected by the investigator by a non-leading question and by reporting events directly observed or spontaneously volunteered by participants. Participants were also encouraged to contact the clinic in between visits if they experienced AEs or had any concerns. Treatment-emergent was defined as events occurring on study drug and up to 7 days after last dose of study drug.
Number of TEAEs Leading to Treatment Discontinuation
时间窗: 139 weeks
Number of Serious TEAEs
时间窗: 139 weeks
Serious TEAEs were defined as any untoward medicinal occurrence or effect that at any dose: * Results in death * Is life-threatening * Requires hospitalisation or prolongation of existing hospitalisation * Results in persistent or significant disability or incapacity * Is a congenital abnormality or birth defect * May jeopardise the participant or may require medical intervention to prevent one or more of the outcomes listed above (Important Medical Events).
Number of Treatment-emergent Adverse Drug Reactions (ADRs)
时间窗: 139 weeks
All AEs were assessed by the investigator for causality (unlikely, possible, probable, not applicable) according to current regulatory standards. AEs which had a 'possible' or 'probable' causality were classified as ADRs.
次要结局
未报告次要终点