Clinical Trial in Phase II of Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis.
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 100
- 试验地点
- 1
- 主要终点
- D50 index obtained from stimulus intensity curves
研究概览
简要总结
Our working hypothesis is that the injection of autologous bone marrow mononuclear cells (BMNC) has a positive effect on the natural loss of motor units and on the increase in the size of the motor unit that occurs in patients with ALS during the evolution of the disease
详细描述
Our working hypothesis is that the injection of autologous bone marrow mononuclear cells (BMNC) has a positive effect on the natural loss of motor units and on the increase in the size of the motor unit that occurs in patients with ALS during the evolution of the disease.
This hypothesis is based on experimental work done in animal models of ALS and the results of our previous phase I clinical trial. In this clinical trial (TCIM / ALS, ClinicalTrials.gov Identifier: NCT02286011) we studied with electrophysiological techniques (in a small number of patients with ALS) the effects of intramuscular injection of a single dose of BMSC in the tibialis anterioris (TA) muscle. . The results indicate that this is a safe procedure and show the presence of a positive and apparently transitory effect on the size and number of motor units of the TA muscle.
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)
盲法说明
double-blind
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
- •Diagnosis of ALS defined or probable according to the criteria established by the World Federation of Neurology
- •Age between 18 and 70 years.
- •Patient who offers sufficient guarantees of adherence to the protocol.
- •Neurophysiological data confirming lower motor neuron involvement at the lumbar and cervical level.
- •Assessment of the motor deficit in the dorsal flexion of both feet (between 3 and 5 points on the MRC scale).
排除标准
- •Mellitus diabetes.
- •Other diseases that may be associated with polyneuropathies.
- •Previous history of cerebral stroke.
- •Previous pathology of the peripheral nervous system that affected one or both lower or upper limbs, with or without clinically evident neurological sequelae.
- •Pregnant or actively breastfeeding patients
- •Patients physiologically capable of becoming pregnant, unless they are using a reliable contraceptive method (Annex III)
- •Patients with cardiac, renal, hepatic, systemic, immune disease that may influence the survival of the patient during the test.
- •Positive serology for hepatitis B, hepatitis C or HIV.
- •Clinical and anesthesiological criteria that contraindicate either sedation or the extraction of BM itself (Alteration of the coagulation system or anticoagulated patient with inability to withdraw anticoagulation, hemodynamic instability, skin alteration in the puncture site, etc.)
- •Inclusion in other clinical trials in the last 6 months.
- •Inability to understand informed consent.
研究组 & 干预措施
MNC (Mononuclear cells)
MNC (Mononuclear cells) (patients in which stem cells will be injected into the two muscles on one side and placebo -vehicle- in the two contralateral muscles).
This group would consist of 74 patients.
干预措施: MNC (Mononuclear cells) (Drug)
MNC (Mononuclear cells)
MNC (Mononuclear cells) (patients in which stem cells will be injected into the two muscles on one side and placebo -vehicle- in the two contralateral muscles).
This group would consist of 74 patients.
干预措施: Placebo / Saline (Drug)
Saline
(patients in which placebo -vehicle- will be injected into both muscles on both sides). This group would consist of 26 patients.
干预措施: Placebo / Saline (Drug)
结局指标
主要结局
D50 index obtained from stimulus intensity curves
时间窗: 48 months from baseline
This parameter quantifies the number and size of the functional motor units of a given muscle.
Rate of serious and non-serious adverse events related to the use of bone marrow mononuclear cells in patients with Amyotrophic Lateral Sclerosis
时间窗: 48 months from baseline
次要结局
- Fiber density (FD)(48 months from baseline)
- Maximum force developed in an isometric contraction of the muscles(48 months from baseline)
- Fuerza muscular (MRC Medical Research Council) score(48 months from baseline)
- Motor unit number index" (MUNIX)(48 months from baseline)
- PAMC area: mV / ms(48 months from baseline)
- Motor unit size index" (MUSIX)(48 months from baseline)
- PAMC amplitude: mV(48 months from baseline)