Huntington Disease
Conditions
Keywords
Grafted patients, Long term follow-up
Brief summary
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.
Detailed description
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
Interventions
OTHERFollow-up
Follow-up grafted patients as long as their condition allows
Sponsors
Assistance Publique - Hôpitaux de Paris
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* 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
Exclusion criteria
* Patient under AME * Patient unable to express consent and not subject to legal protection * Knowned pregnancy or breastfeeding woman
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Progression of the Unified Huntington's Disease Rating Scale For Advanced Patients (UHDRS-FAP) motor score | 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 (UHDRS) motor score | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | For each patient |
| 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 | For each patient |
| 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 | Grafted patients and cohort patients (BIOHD) |
| 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 | For each patient |
| 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 | For each patient |
| 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 | For each patient |
| 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 | For each patient |
| 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 | For each patient |
| 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 | For all patients whose brains have been recovered |
| 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 | For each patient |
| 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 | For each patient |
| 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 | For each patient |
Countries
France
Contacts
Primary ContactAnne-Catherine BACHOUD-LEVI, MD, PhD
Outcome results
None listed