Long Term Follow-up Grafted Huntington's Disease Patients

Not Applicable

Not yet recruiting

• Conditions: Huntington Disease
• Interventions: Other: Follow-up

• Registration Number: NCT06194006
• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

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

Study Timeline

• Study First Submitted: 2022-03-09
• Status Verified: 2023-05
• Study First Submitted QC: 2023-12-20
• Last Update Submitted: 2023-12-20
• Study Start: 2024-01-01
• Study First Posted: 2024-01-08
• Last Update Posted: 2024-01-08
• Primary Completion: 2034-01-01
• Study Completion: 2034-01-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 39

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

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Exclusion Criteria

• Patient under AME
• Patient unable to express consent and not subject to legal protection
• Knowned pregnancy or breastfeeding woman

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Long term follow-up	Follow-up	* Formalized and standardized follow-up * Retrospective data collection * Biological collections * Biological collections's centralisation

Primary Outcome Measures

Name	Time	Method
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
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

Secondary Outcome Measures

Name	Time	Method
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
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
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)
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
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
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
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
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
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
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
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
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

Trial Locations

Locations (1)

Assistance Publique Hôpitaux de Paris - Hôpital Henri Mondor; 🇫🇷 Créteil, France