A Phase I-II Study to Evaluate Safety and Efficacy of Allogeneic Bone-Marrow Mesenchymal Stem Cells in Patients With Rapidly Progressive Interstitial Lung Disease
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Safety: Number of serious adverse events
研究概览
简要总结
The study evaluates the safety and the efficacy of the addition of intravenous transplantation of donor bone marrow mesenchymal stem cells in patients with idiopathic interstitial pneumonia or connective tissue disease associated with interstitial lung disease, which have actively progressing disease with rapid loss of pulmonary function on the background of routine treatment.
详细描述
Despite significant progress in the treatment of interstitial lung disease, achieved thanks to new drugs, such as pirfenidone and nintedanib, there are many patients for whom these drugs are not available or poorly tolerated. In addition significant evidence of their effectiveness and safety is valid only for idiopathic pulmonary fibrosis. The transplantation of allogeneic stem cells is a promising direction in the modern medicine with the proven safety for different diseases. But the effectiveness of this therapy is still under research. We believe that in most severe cases of a rapidly progressive interstitial lung disease the transplantation of mesenchymal stem cells may be an effective technology due to their immunomodulatory properties.
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
- None
入排标准
- 年龄范围
- 20 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female patients 20-80 years old
- •Diagnosis of idiopathic interstitial pneumonia or secondary to connective tissue diseases interstitial lung disease, based on:
- •Clinical symptoms > 12 months duration,
- •Histologically diagnosed or diagnostic chest HRCT features of interstitial pneumonia
- •Forced Vital Capacity (FVC) ≥ 40% predicted and Diffusing Lung Capacity (DLCO) ≥20%
- •Loss more than 10% of FVC (L) and DLCO during the last 12 months
- •Signed informed consent.
排除标准
- •Diagnosis of an interstitial lung disease other than idiopathic interstitial pneumonia or connective tissue disease associated with interstitial lung disease (sarcoidosis, pulmonary alveolar proteinosis, lymphangioleiomyomatosis, pulmonary amyloidosis, exposure-related lung disease etc)
- •Obstructive lung disease: FEV1/FVC < 0.70
- •Clinically significant medical condition, in the opinion of the investigator, may compromise the results of the study
- •Evidence of active infection within 4 week prior to enrollment
- •History of malignancy < 5 years prior to enrollment
- •Unable to cooperate with any study procedures
- •Pregnant or breast-feeding
- •Treatment with antiinflammatory or antifibrotic drugs including oral steroids, cytostatic drugs, pirfenidone, D penicillamine, colchicine, tumor necrosis factor α blockers, imatinib, interferon γ, monoclonal antibodies < 6 months prior to randomization.
- •Active listing for transplant of any organ.
- •Known history of alcohol abuse within 1 year prior to enrollment
- •Participation in another clinical trial
研究组 & 干预措施
MSCs
2 intravenous infusions of suspension of 200 000 000 MSCs each at interval of 7 days. Infusions will be repeated every 3 months for 1 year.
干预措施: Bone marrow mesenchymal stem cells (Drug)
placebo
2 intravenous infusions of 400 mL saline each at interval of 7 days. Infusions will be repeated every 3 months for 1 year.
干预措施: Placebo (Drug)
结局指标
主要结局
Safety: Number of serious adverse events
时间窗: 12 months
registration of adverse events related to infusion and 12 months follow-up
次要结局
- FVC changes from baseline(12 months)
- DLCO changes from baseline(12 months)
- exercise capacity changes(12 months)