A Phase I Multicenter Study of Allogenic, Intracerebroventricular Human Neural Stem Cells Transplantation for the Experimental Treatment of Secondary Progressive Multiple Sclerosis Patients
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 24
- 试验地点
- 3
- 主要终点
- Incidence of Treatment Emergent AE
研究概览
简要总结
This will be a phase I, open, multicenter, international study performed by 3 participating centres across two countries (Italy and Switzerland). Fifteen to 24 patients affected by SPMS will be enrolled, according to a "standard" phase I design over 18 months. All patients will enter a 3 months run in phase. Thereafter they will receive one of four different doses of allogenic hNSCs (dose A=5 millions hNSCs; dose B=10 millions hNSCs; dose C=16 millions hNSCs; dose D=24 millions hNSCs). Following hNSCs injection, all SPMS patients will receive immunosuppression with tacrolimus for 6 months. Patients will be clinically followed monthly for 1 year and then every 6 months for the 5 years following the study completion (possibly all life long).
MRI assessments will be performed monthly for the first 6 months and then every 3 months for 5 years following the study completion.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 60 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •SPMS with progressive accumulation of disability after initial relapsing course, with or without disease activity (Lublin et al. 2014).
- •EDSS ≥ 6.5 and ≤ 8
- •EDSS progression over the 2 years prior to study start of ≥ 1.0 point for patients with EDSS =6.5 at the time of inclusion , and of ≥ 0.5 points for patients with EDSS > 6.5 at the time of inclusion
- •Age ≥ 18 and ≤ 60 years
- •Failure of best medical treatment as judged by the treating neurologist and declared absence of therapeutic alternatives
排除标准
- •Neurological conditions other than MS.
- •Psychiatric disorders, severe cognitive decline and personality and relational disorders.
- •History or known presence of significant systemic, infectious, oncologic or metabolic disorders.
- •Presence of any other autoimmune disease.
- •Chronic infections (HBV, HCV, HIV, tuberculosis).
- •Inability to perform MRI scans.
- •Immunomodulant/immunosuppressive treatments in the last 6 months before inclusion.
- •Current participation to other experimental studies.
- •Inability to provide informed consent.
- •Any contra-indication to lumbar puncture and the surgical procedure (e.g. use of anticoagulants)
- •Pregnancy and breast feeding.
研究组 & 干预措施
Human Neural Stem Cells Suspension
Intraventricular injections of Allogenic human Neural Stem Cells (hNSCs) in four different dosages (5, 10,16 or 24 millions)
干预措施: Human Neural Stem Cells (Biological)
结局指标
主要结局
Incidence of Treatment Emergent AE
时间窗: 1 year
To Evaluate the Feasibility, Safety and Tolerability of intracerebroventricular injection of allogenic hNSCs
Percentage of Mortality in treated patients
时间窗: 1 year
Percentage of subjects (%) with death due to procedure (mortality correlated to treatment)
次要结局
- Relapses Rate(Up to 1 year)
- Change in Functional disability(Up to 1 year)
- Activity of Cognitive function(Up to 1 year)
- Alteration in Neurophysiological parameters(Up to 1 year)
- MS Biomarkers(Up to 1 year)