Study of Two Intrathecal Doses of Autologous Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 1 期
- 入组人数
- 28
- 试验地点
- 2
- 主要终点
- Safety
研究概览
简要总结
The study aims to evaluate primarily safety of two injections of autologous mesenchymal stem cells in Amyotrophic Lateral Sclerosis patients. Secondary outcomes of efficacy will also be evaluated
详细描述
The study is an open-label, unmasked, uncontrolled phase I and II trial to evaluate safety and efficacy of two injections of autologous mesenchymal stem cells (MSC) in Amyotrophic Lateral Sclerosis (ALS) patients. Patients will be recruited trough a web-based registration system, open for all ALS Brazilian patients. The patients will be enrolled after in-person screening at the University of Sao Paulo Medical School Clinics Hospital and inclusion criteria fulfilled. There will be 9 national calls for registration, two months apart each. The enrolled patients will be followed for 3 months before bone marrow aspiration (BMA). Patients will receive 2 intrathecal MSC injections, 1 and 2 months after BMA. The patients will be followed for 6 months after the interventions.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Age between 18 and 70
- •Possible, probable or definite ALS following El Escorial Revised Criteria (Brooks, 2000)
- •ALSFRS-r ≥30 at enrollment
- •Forced Vital Capacity ≥65% of the height and weight standard
- •No-pregnancy agreement
- •Regional accessibility to the study site
- •Capability to give away agreed consensus
- •Patients will be followed at Academic Institutions at their hometown
排除标准
- •Previous cellular therapy
- •Incapacity to lay still during bone marrow aspirate or intrathecal MSC injections
- •Personal history of auto-Immune, myeloproliferative or myelodysplastic diseases, leukemia, lymphoma, whole-body irradiation, hip fracture, severe scoliosis or incapacity to undergo any of the study's proposed procedures
- •Any other disease that may interfere with the study
- •Any other neurological diseases
- •Aspartate or alanine aminotransferases elevated >3x normality upper limit
- •Serum creatinine >2x normality upper limit
- •Hepatitis B and C, HIV, HTLV I and II and syphilis
- •Immunosuppressant drug use within 6 weeks from the study's screening
- •Pregnancy or breast-feeding
- •Acquired or inherited Immunodeficiency
- •Participation in other clinical trials
- •Non-invasive ventilation, tracheostomy or diaphragm pacing
- •Substance abuse within one year and other unstable mental health diseases according to researcher's judgement
- •Gastrostomy or any alternative feeding means
- •Inappropriate in-vitro MSC expansion
结局指标
主要结局
Safety
时间窗: 10 months
Clinical and laboratory monitoring of possible reactions to intrathecal MSC delivery
次要结局
- Changes in morphometric parameters of MRI of encephalon and spinal cord(10 months)
- Change in Forced Vital Capacity (FVC)(10 months)
- Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R)(10 months)
- Change in Isometric Strength(10 months)
- Change in Handheld dynamometry(10 months)
- Change in Electrical Impedance Myography(10 months)
研究者
Gerson Chadi
Professor
University of Sao Paulo General Hospital