Multicentric Intracerebral Grafting in Huntington's Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 54
- 试验地点
- 2
- 主要终点
- Motor UHDRS rating scale, at randomization, 20 month after transplant
研究概览
简要总结
The aim of this study is to assess the clinical benefit of intrastriatal grafting of human cells from the foetal ganglionic eminence in patients with Huntington's disease. The duration of the study will be 52 months. A first group of patients will be grafted at M13-14 (early G group) and a second group of patients will be grafted at M33-34 (late G group). The principal criterion is the comparison of the progression between M12 and M32 of the motor score (TMS) of the UHDRS between grafted patients (early G group) and not yet grafted patients (late G group). An additional evaluation will be performed to compare the progression in individual patients over the 52-month study period. We will thus be able to compare the pre and post-graft TMS progression for all patients.
详细描述
The aim of this study is to show the existence of a clinical benefit rising from a substitution of the striatal neurons degenerated among a large cohort of Huntington's patient at early stage by homologous neurons coming from human foetuses, This effect will be estimated, compared with a group of patients not treated at first, on the results of the motor scale of the Unified Huntington Disease Rating Scale (UHDRS, Huntington study group, on 1996).
Transplants will be realized in two surgical times to avoid the risk of hurts per-operating, BI-CAUDES, if transplants were realized at single time. The minimal interval between both transplants will be of 2 weeks, so as to let the patient recover of the first general anaesthesia It's a multicentric study of phase II randomised and controlled, with direct individual benefit.
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
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Disease clinically declared since at least 1 year ,UHDRS motor > or =5
- •TFC > or =
- •CAG > or = 36
- •Age between 18 and 65
- •Family and socially integrated subject
- •Informed consent.
排除标准
- •Severe intellectual deterioration or neuropsychiatric disorders making the follow-up longitudinal too complicated (score MATTIS < 120).
- •Not-observance of the appointments and the symptomatic treatments in pre-surgical period.
- •Intercurrent disease making a surgical operation impossible.
- •Associated disease having a neurological repercussion, intercurrent cerebral lesion with the IRM.
- •Visceral affection engraves, evolutionary, which brings into play the vital forecast or makes risks for general anaesthesia.
- •Mental Affection likely to disturb adhesion with the protocol, and in particular antecedents of hallucinations spontaneous and/or induced by the drugs; antecedents of serious depression having required repeated hospitalizations; antecedents of repeated suicide attempts.
- •Cerebral morphological anomalies, others that those characteristic of the disease, noted with the IRM or the tomodensitometry.
- •Participation in progress, or stopped since less than three months, with a therapeutic protocol of the Huntington's disease Exclusion Criteria (pre-randomization)
- •Not-observance of the appointments and the symptomatic treatments in pre-surgical period.
- •Intercurrent disease returning the surgery or impossible immunosuppression. v Subject completely isolated with his family and socially..
- •UHDRS motor <
- •Positives serologies for HIV1, HIV2, AgP24, HTLV1 et 2, HEPATITE B, HEPATITE C, syphilis
- •Psychiatric disorders being able to compromise the follow-up.
- •Signs other than Huntington with the IRM.
研究组 & 干预措施
1
Early Graft (Early G)
干预措施: graft intracerebral of foetal neurons (Biological)
2
Late Graft (Late G)
干预措施: graft intracerebral of foetal neurons (Biological)
结局指标
主要结局
Motor UHDRS rating scale, at randomization, 20 month after transplant
时间窗: during de study
次要结局
- Neurologic, Cognitive, Neurophysiologic, Psychiatric, MRI and Pet-scan evaluation at randomization, 20 month after and at the end of the protocol(during the study)