Intra-spinal Cord Delivery of Human Neural Stem Cells in ALS Patients: Proposal for a Phase I Study/ Trapianto Intramidollare di Cellule Staminali Neurali Umane Come Terapia Putativa Per la SLA: Proposta di un Trial Clinico di Fase I
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 18
- 试验地点
- 2
- 主要终点
- safety of a microsurgery human neural stem cells transplantation into spinal cord of ALS patients
研究概览
简要总结
Primary aim of the trial
- to verify safety and tolerability of expanded human fetal neural stem cells
- to verify safety and tolerability of a microsurgery human fetal neural stem cells transplantation model
- to recognize each change in patient's quality of life
Secondary aim of the trial
- assessment of the impact of human neural stem cells transplantation on the disability of ALS in a clinically significant and measurable way
- Assessment of the impact of human neural stem cells transplantation on mortality (all causes)
详细描述
18 patients diagnosed as definite ALS according to the El Escorial criteria will be recruited during two years. The patients will be divided into 3 groups with different severity. Each group will be less compromised than the group that went before them. Their breathing measurements must be at least 60%. Subjects will receive injections of the human foetal neural stem cells into the spinal cord. A variety of tests and examinations will take place every month for the first year and every three months for the next two in order to monitor the course of the disease and the adverse events.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 盲法
- None
入排标准
- 年龄范围
- 20 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of definite or possible ALS according to revised EL Escorial criteria
- •Age: 20-75 years
- •documented progression of disease during the last 6 months.
- •Absence of concomitant diseases
- •Adequate assurances of adherence to protocol
- •The patient must be able to communicate verbally or with the use of non-verbal communication system
- •maximum score of 1 on walking item of ALS functional rating scale
- •forced vital capacity > or equal to 60
- •forced vital capacity > or equal to 60
- •ambulation difficulties (maximum score of 2 on walking item of ALS functional rating scale)
- •Independent ambulation (score 4 on a scale ALS-FRS)
- •forced vital capacity > or equal to 70
排除标准
- •The presence of at least one of the following will lead to patient exclusion.
- •psychiatric diseases or other neurological diseases different from ALS
- •other systemic diseases
- •Neoplasms or other diseases reducing life expectancy
- •Antecedent polio infection
- •corticosteroids, immunoglobulin or immunosuppressive treatment
- •involvement in other clinical trials
- •2 or more critical items in MMPI
- •neuropsychological evaluation suggestive of mental deterioration or cognitive sphere disturbance.
- •Impediments to MRI
- •patients unable to understand informed consent form and study aims.
- •women who are pregnant or childbearing potential for the duration of the study. Non-pregnant status will be documented by beta-HCG negative test 7 days before treatment start
结局指标
主要结局
safety of a microsurgery human neural stem cells transplantation into spinal cord of ALS patients
时间窗: Participants will be followed for at least 3 years. Monthly in the first year and every three months the following two years
* Percentage of subjects (%) with treatment-related mortality defined as death due to procedure and not to the course of the disease * Number of adverse events related to the procedure * Changes in neuroradiological (MRI) and neurophysiological variables (SEP, MEP) * Changes in neuropsychological variables
次要结局
未报告次要终点
研究者
Angelo Luigi Vescovi
Scientific Director of Laboratorio Cellule Staminali, Cell Factory e Biobanca AOSP di Terni, Scientific Director IRCCS Casa Sollievo della Sofferenza S.Giovanni Rotondo Italy
Azienda Ospedaliera Santa Maria, Terni, Italy