Long-term Follow-up of Huntington's Disease Patients Treated With Intrastriatal Fetal Neuron Allograft
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 入组人数
- 39
- 试验地点
- 2
- 主要终点
- Progression of the Unified Huntington's Disease Rating Scale (UHDRS) motor score
研究概览
简要总结
Long-term follow-up of Huntington's disease patients treated with intrastriatal allografts is essential to assess the benefit/risk ratio of grafts as well as their effectiveness. Indeed, some patients are likely to develop adverse effects and the impact of alloimmunisation phenomena remains to be explored.
详细描述
The aim is to create a specific cohort of transplant patients, to homogenise their follow-up methods, to collect all the patient data (medical, paramedical, imaging, biological) acquired from the end of their participation in MIG-HD'study (Foetal Striatal allografting in Huntington's disease: a multicentre, randomised, delayed start, phase 2 open-label cell therapy trial) until the last possible follow-up of the last patient in the study
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —(Adult, Older Adult)
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult patients transplanted under the MIG-HD protocol
- •Information and informed consent from patients or their representative
- •Affiliation with a social security scheme or beneficiary
排除标准
- •Patient under AME
- •Patient unable to express consent and not subject to legal protection
- •Knowned pregnancy or breastfeeding woman
结局指标
主要结局
Progression of the Unified Huntington's Disease Rating Scale (UHDRS) motor score
时间窗: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
For each patient
Progression of the Unified Huntington's Disease Rating Scale For Advanced Patients (UHDRS-FAP) motor score
时间窗: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
For each patient
次要结局
- Events and adverse reactions recording since the transplantation(From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed)
- Slopes comparison of neurological scores (Unified Huntington's Disease Rating Scale (UHDRS) or Unified Huntington's Disease Rating Scale For Advanced Patients (UHDRS-FAP)) before and after transplantation(From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed)
- Comparison of the clinical evolution (Unified Huntington's Disease Rating Scale (UHDRS) or Unified Huntington's Disease Rating Scale For Advanced Patients (UHDRS-FAP)) of transplanted patients with that of non-transplanted patients in the BioHD cohort(From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed)
- Slopes comparison of cognitive scores (Unified Huntington's Disease Rating Scale (UHDRS) or Unified Huntington's Disease Rating Scale For Advanced Patients (UHDRS-FAP)) before and after transplantation(From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed)
- Slopes comparison of neuropsychologic scores (Mattis Dementia Rating Scale/Trail Making test/Hopkins Verbal Learning Test/Categorical fluency/Sign barrier test/Montgomery-Åsberg Depression Rating Scale (MADRS)) before and after transplantation(From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed)
- Correlation between graft activity measured by imaging and patient performance(From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed)
- Correlation between post-mortem brain observations and patient performance before death(From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed)
- Research into predictive factors (relationships between fetal haplotypes, patient haplotypes and appearance of alloimmunization phenomena) of the evolution of transplanted patients(From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed)
- Research and identification of anti-MHC Ab from samples acquired under the MIG-HD and Post-MIGHD protocols as well as in care(From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed)
- Correlation between post-mortem observations of the brains of deceased patients and measurements obtained by MRI and PET-SCAN in studies MIG-HD and BioHD(From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed)
- Slopes comparison of psychiatric scores (Unified Huntington's Disease Rating Scale (UHDRS) or Unified Huntington's Disease Rating Scale For Advanced Patients (UHDRS-FAP)) before and after transplantation(From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed)
- Analysis of brains collected as part of MIG-HD and POST-MIGHD i) to quantify neuropathological lesions ii) to search for markers of inflammatory reactions(From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed)
