Phase I Clinical Trial on Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis.
试验速览
- 阶段
- 1 期
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Rate of serious and non-serious adverse events related to the use of bone marrow mononuclear cells in patients with Amyotrophic Lateral Sclerosis.
研究概览
简要总结
The purpose of this study is to evaluate the safety of Intramuscular Infusion of Autologous Bone Marrow Stem Cells in Patients With Amyotrophic Lateral Sclerosis by a prospective, single-center, randomized, parallel, double-blind, placebo-controlled phase I clinical trial.
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
- •Diagnosis of definite or probable ALS according to the criteria established by the World Federation of Neurology
- •Patient that provides reasonable assurance of adherence to protocol.
- •Neurophysiological data confirming affectation of lower motor neurons in the lumbar region.
- •Assessment of motor deficits in dorsiflexion of both feet (4 or 5 points on the MRC scale)
- •The patient must fulfill all inclusion criteria.
排除标准
- •Diabetes Mellitus.
- •Other diseases that may present with polyneuropathy.
- •Previous history of stroke.
- •Prior Pathology of the peripheral nervous system affecting one or both lower limbs with or without clinically evident neurological sequelae.
- •Pregnant or breastfeeding patients active.
- •Patients physiologically capable of becoming pregnant, unless they are using reliable contraception.
- •Patients with cardiac disease, renal, hepatic, systemic, immune that may influence patient survival during the test.
- •Positive serology for hepatitis B, hepatitis C or HIV.
- •Clinical and anesthesiologic Criteria, contraindicating either sedation or extraction of MO (Altered coagulation system or anticoagulated patient with inability to withdraw anticoagulation, hemodynamic instability, altered skin puncture site, etc.)
- •Included in other clinical trials in the last 6 months.
研究组 & 干预措施
MNC (Mononuclear cells)
All patients included in the clinical trial will receive an intramuscular infusion of autologous mononuclear cells (MNC) of Bone Marrow (BM) in TA muscle of one of the lower limb (experimental group). The lower limb on the CMN infuse autologous BM will be determined randomly.
The average dose is 550 millions of cells (100-1200 million) diluted in 2 ml. saline
干预措施: MNC (Mononuclear cells) (Biological)
Saline
All patients included in the clinical trial will receive an intramuscular infusion of 2 mL of saline (placebo) in the TA muscle of the contralateral limb (group control).
干预措施: Saline (Other)
结局指标
主要结局
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: 24 months from baseline
次要结局
- Muscle force MRC (Medical Research Council) score(24 months from baseline)
- Estimated number of motor units (MUNE)(24 months from baseline)
- Compound muscle action potential (CMAP)(24 months from baseline)
- Fiber density (FD)(24 months from baseline)
- Maximum force developed in an isometric contraction of the tibialis anterior (TA) muscle.(24 months from baseline)
- Maximum transversal area of the tibialis anterior (TA)(24 months from baseline)