Phase I, Single Center, Prospective, Non-randomized, Open Label, Safety/Efficacy Study of the Infusion of Autologous Bone Marrow-derived Stem Cells, in Patients With Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 1 期
- 状态
- 暂停
- 发起方
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- Safety
研究概览
简要总结
A Phase I, single center, prospective, non-randomized, open label, safety/efficacy study of the infusion of autologous bone marrow-derived stem cells, in 6 patients with Amyotrophic Lateral Sclerosis according to established criteria (1), (2) with a moderate to severe diagnosis of ALS according to the World Federation of Neurology El Escorial criteria.
The primary purpose of this study is to evaluate safety of the infusion procedure, as assessed by absence of complications at the site of infusion or the appearance of new neurologic deficit not attributed to the natural progression of the disease.
Secondary outcomes will include a)neurological evidence of trends toward a slowing down of the decline of the forced vital capacity (FVC) (3) and of the functional rating scale (ALS-FRS) scores, as assessed at 3-month intervals, b)evidence of a decline of the maximum voluntary isometric contraction-arm (MVIC-arm) and MVIC-grip Z (4) scores and c)patient evaluation that the treatment was effective and consider the possibility of a new cell product stem cell infusion.
Subjects who fulfill inclusion/exclusion criteria and sign informed consent will undergo an aspiration of bone marrow from the iliac crest for preparation of the cellular product.
The day of infusion, the investigational product will be injected into the patient's intrathecal space.
After cell infusion patients will be followed at WK 2, MN 1, MN 2, MN 6 and a long-term followup at MN 12 in the clinic and/or office. Electromyographic (EMG) studies, Forced vital capacity (FVC), functional rating scale (FRS) and maximum voluntary isometric contraction-arm (MVIC-arm) and MVIC-grip Z scores will have been used to assess the status of the disease before (historical record acceptable if done within three months of Screening Visit) and during the 12-month study period after cell infusion.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult male and female subjects > 18 years of age.
- •Good understanding of the protocol and willingness to consent.
- •Moderate to severe Diagnosis of ALS according to the World Federation of Neurology El Escorial criteria.
- •Vital capacity at least 50% predicted value for gender, height and age.
- •More than 6 and less than 36 months of evolution of the disease.
- •Hematocrit greater than 30 % prior to bone marrow aspiration.
- •Platelet count greater than 100 Thousand/uL at screening.
- •INR less than or equal to 1.5.
排除标准
- •Any concurrent illness, which affects the bone marrow.
- •Any concomitant medication that affects the bone marrow.
- •Previous stem cell therapy.
- •Any lymphoproliferative disease.
- •Riluzole with 4 weeks of study entry and at any time during the study.
- •Hemophiliacs or subjects with bleeding disorders.
- •Known hypersensitivity to fetal bovine serum
- •HIV infection.
- •Serum creatinine > 3.0 in subjects not on hemodialysis.
- •Skin infection at the infusion site or systemic infection
- •Current smoker.
- •Active drug or alcohol addiction
- •Pregnant, planning to become pregnant or not on accepted birth control method if subject is of child bearing potential.
- •Subjects that are breast feeding.
- •Any condition that the Principal Investigator considers would render the subject unfit for the study.
结局指标
主要结局
Safety
时间窗: one year
Safety of the infusion procedure, as assessed by absence of complications at the site of infusion or the appearance of new neurologic deficit not attributed to the natural progression of the disease.
次要结局
- Efficacy(one year)