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临床试验/NCT06194006
NCT06194006尚未招募不适用

Long-term Follow-up of Huntington's Disease Patients Treated With Intrastriatal Fetal Neuron Allograft

Assistance Publique - Hôpitaux de Paris2 个研究点 分布在 1 个国家目标入组 39 人开始时间: 2024年1月1日最近更新:
适应症

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

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)

研究者

申办方类型
Other
责任方
Sponsor

研究点 (2)

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