Phase I/II Clinical Trial on The Use of Autologous Bone Marrow Stem Cells in Amyotrophic Lateral Sclerosis (Extension CMN/ELA)
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 63
- 试验地点
- 1
- 主要终点
- Forced vital capacity
研究概览
简要总结
The purpose of this clinical trial is to assess the feasibility and the security of the intraspinal and intrathecal infusion of autologous bone marrow stem cells for the treatment of Amyotrophic Lateral Sclerosis patients.
详细描述
Patients with Amyotrophic Lateral Sclerosis (ALS) typically endure a progressive paralysis due to the continued loss of motoneurons that leads them to death in less than 5 years. No treatment has changed its natural history. Intraspinal injections of bone marrow mononuclear cells (MNC) have been able to ameliorate the course of ALS in murine models, acting as pumps of trophic factors that keep the motoneurons functional. Moreover, the clinical trial (Study NCT00855400 on www.ClinicalTrials.gov) conducted by our research group to determine the safety and efficacy of Autologous Stem Cell transplantation in Amyotrophic Lateral Sclerosis in humans, found that this procedure is feasible and safe. Continuing with that study, we have designed a phase I/II clinical trial to check the feasibility of the intraspinal and intrathecal infusion of autologous bone marrow stem cells.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnose established following the World Federation of Neurology criteria
- •More than 6 and less than 36 months of evolution of the disease
- •Medullar onset of the disease
- •More than 18 and less than 70 years old
- •Forced Vital Capacity ≥ 50%
- •Total time of oxygen saturation <90% inferior to 5% of the sleeping time
- •Signed informed consent
排除标准
- •Neurological or psychiatric concomitant disease
- •Need of parenteral or enteral nutrition through percutaneous endoscopic gastrostomy or nasogastric tube
- •Concomitant systemic disease
- •Treatment with corticosteroids, immunoglobulins or immunosuppressors during the last 12 months
- •Inclusion in other clinical trials
- •Unability to understand the informed consent
结局指标
主要结局
Forced vital capacity
时间窗: Every 3 months
次要结局
- Absence of adverse events(Every week / month depending on the study phase)
- Neurophysiological variables: Electromyography, polysomnography, evoked potentials(Every 3 months)
- Neuroradiological variables: Spinal Magnetic Resonance Imaging (MRI)(Every 3 months)
- Respiratory variables: Maximal inspiratory pressure (PIM), Maximal expiratory pressure (PEM), sniff nasal, oxymetry.(Every 3 months)
- Psychological variables: Health Questionnaire (EuroQol-5D), The Profile of Mood States (POMS)(Every 3 months)
- Neurological variables: The Amyotrophic Lateral Sclerosis Functional Rating Scale (ALS-FRS), Medical Research Council (MRC) and Norris scales(Every 3 months)