A Multicenter Phase I/II Clinical Trial, Randomized, Controlled With Placebo, Triple Blind to Evaluate Safety, and Indications of Efficiency of the Intravenous Administration of the Therapy With 3 Doses of MSC in Patients With ASL Moderated to Severe
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 52
- 试验地点
- 4
- 主要终点
- Number of adverse serious unexpected reactions or not, attributable to the treatment (SUSSARs or SAE)
研究概览
简要总结
A multicenter phase I/II Clinical trial,randomized, controlled with placebo, triple blind to evaluate the safety of the intravenous administration of 3 doses of autologous mesenchymal stem cells cells from adipose tissue in patients with Amyotrophic Lateral Sclerosis (ALS).
详细描述
A multicenter phase I/II Clinical trial,randomized, controlled with placebo, triple blind to evaluate the safety of the intravenous administration of 3 doses of autologous mesenchymal stem cells cells from adipose tissue in patients with Amyotrophic Lateral Sclerosis (ALS).
40 patients will be enrolled and will be randomized into one of the following 4 arms:
- 10 patients in the control group (placebo)
- 10 patients received a dose of 1 million MSC / kg
- 10 patients received a dose of 2 million MSC / kg.
- 10 patients received a dose of 4 million MSC / kg
The follow-up phase of each patient from the cell infusion/placebo will be 6 months.
At the time that each patient completed the follow-up period (i.e., 6 months after the infusion of the cellular product or placebo), the blind will be open, and patients who have been assigned to the control group, will receive the cell product as secondary treatment. These patients will be randomized to receive each of the doses used in the first phase. From this point, they begin a second period of follow up of 6 months.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
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
- •Women and males over 18-year-old.
- •Good understanding of the protocol and aptitude to grant the informed assent.
- •Diagnosis of sporadic ALS, with diagnosis of certainty, that is to say, definite or probable, in agreement with the criteria of "El Escorial", of the World Federation of Neurology.
- •Forced vital capacity of at least 50 % of the one that would correspond to them for sex, height and age.
- •More than 6 and less than 36 months of evolution of the disease (from the beginning of the symptoms).
- •Possibility of obtaining, at least, 50gr of adipose tissue.
- •Treatment with riluzole, for at least, a month before the inclusion.
排除标准
- •Any concomitant disease that under investigator's criteria could concern the measures of the clinical variables of the trial (hepatic, renal or cardiac insufficiency, diabetes mellitus, etc).
- •Previous therapy with stem cells.
- •Participation in another clinical trial during 3 months previous to the entry in this trial.
- •Any disease lymphoproliferative
- •Tracheostomy and /or gastrostomy.
- •Haemophilia, diathesis hemorrhagic or anticoagulative current therapy.
- •Hypersensitivity known to the bovine foetal whey or the gentamicin.
- •Medical precedents of infection of the HIV or any serious condition of immunocompromised.
- •Positive HBV or HCV serology
- •Levels of creatinine in whey > 3.0 in subjects not submitted to haemodialysis.
研究组 & 干预措施
Placebo
Intravenous administration of placebo
干预措施: Intravenous administration of placebo (Other)
1 million of MSC
Intravenous administration of 1 million of MSC/ kg
干预措施: Intravenous administration of 1 million of MSC (Drug)
2 million of MSC
Intravenous administration of 2 million of MSC/ kg
干预措施: Intravenous administration of 2 million of MSC (Drug)
4 million of MSC
Intravenous administration of 4 million of MSC/ kg
干预措施: Intravenous administration of 4 million of MSC (Drug)
结局指标
主要结局
Number of adverse serious unexpected reactions or not, attributable to the treatment (SUSSARs or SAE)
时间窗: 6 months
To evaluate the safety of the intravenous administration of 3 doses of autologous mesenchymal stem cells (MSC) from adipose tissue in patients with Amyotrophic lateral Sclerosis (ALS) ABSENCE of: complications in the place of the infusion, appearance of a new neurological effect not attributable to the natural progression of this pathology and adverse serious unexpected reactions or not, attributable to the treatment (SUSSARs or SAE)
Appearance of a new neurological effect not attributable to the natural progression of this pathology
时间窗: 6 months
To evaluate the safety of the intravenous administration of 3 doses of autologous
Complications in the place of the infusion
时间窗: 6 months
To evaluate the safety of the intravenous administration of 3 doses of autologous
次要结局
- Changes in neurophysiological parameters and of quality of life(6 months)
- Changes in the degree of muscular force(6 months)
- Changes in the vital forced capacity(6 months)
- Changes of the muscular mass estimated by Nuclear Magnetic Resonance (NMR) of the upper and low extremities(6 months)
- Need and time to tracheotomy or permanent assisted ventilation(6 months)
- Changes in the progression of the disease (modifications in the scale of functionality of the ALS)(6 months)