Clinical trial NCT06194006: Long Term Follow-up Grafted Huntington's Disease Patients
Official title: Long-term Follow-up of Huntington's Disease Patients Treated With Intrastriatal Fetal Neuron Allograft
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: NA
Conditions: Huntington Disease
Keywords: Grafted patients
Long term follow-up
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
First posted: 2024-01-08
Enrollment: 39
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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

Eligibility criteria: 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

Primary outcomes:
measure: Progression of the Unified Huntington's Disease Rating Scale (UHDRS) motor score
timeFrame: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
measure: Progression of the Unified Huntington's Disease Rating Scale For Advanced Patients (UHDRS-FAP) motor score
timeFrame: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
Secondary outcomes:
measure: 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
timeFrame: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
measure: 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
timeFrame: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
measure: 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
timeFrame: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
measure: 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
timeFrame: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
measure: 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
timeFrame: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
measure: Events and adverse reactions recording since the transplantation
timeFrame: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
measure: Research and identification of anti-MHC Ab from samples acquired under the MIG-HD and Post-MIGHD protocols as well as in care
timeFrame: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
measure: Correlation between graft activity measured by imaging and patient performance
timeFrame: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
measure: 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
timeFrame: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
measure: Correlation between post-mortem brain observations and patient performance before death
timeFrame: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
measure: 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
timeFrame: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
measure: Research into predictive factors (relationships between fetal haplotypes, patient haplotypes and appearance of alloimmunization phenomena) of the evolution of transplanted patients
timeFrame: From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed
Other outcomes:
Locations:
city: Créteil
country: France
facility: Assistance Publique Hôpitaux de Paris - Hôpital Henri Mondor
Study officials:
affiliation: Assistance Publique - Hôpitaux de Paris
name: Anne-Catherine BACHOUD-LEVI, MD, PhD
role: STUDY_DIRECTOR
References: