Long Term Follow-up Grafted Huntington's Disease Patients

NCT06194006 | Not Yet Recruiting

• 2 other identifiers: K140601J2018-A02569-46
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 1

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.

Conditions

Huntington Disease

Keywords

Grafted patients; Long term follow-up

Outcome Measures

Primary Outcomes (2)

• Progression of the Unified Huntington's Disease Rating Scale (UHDRS) motor score

  For each patient

  From the end of participation at MIGHD's study until the death from any cause or assessed up to 10 years after consentement signed

• Progression of the Unified Huntington's Disease Rating Scale For Advanced Patients (UHDRS-FAP) motor score

  For each patient

  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 (12)

• 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

• 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 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

• 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

• 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

Study Arms (1)

Long term follow-up

OTHER

* Formalized and standardized follow-up * Retrospective data collection * Biological collections * Biological collections's centralisation

Other: Follow-up

Interventions

Follow-up OTHER

Follow-up grafted patients as long as their condition allows

Long term follow-up

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (1)

Assistance Publique Hôpitaux de Paris - Hôpital Henri Mondor

Créteil, 94010, France

Location

MeSH Terms

Conditions

Huntington Disease

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Chorea; Dyskinesias; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Study Officials

• Anne-Catherine BACHOUD-LEVI, MD, PhD

  Assistance Publique - Hôpitaux de Paris

  STUDY DIRECTOR

Central Study Contacts

Anne-Catherine BACHOUD-LEVI, MD, PhD

CONTACT

1 49 81 43 01; anne-catherine.bachoud-levi@aphp.fr

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 9, 2022
• First Posted: January 8, 2024
• Study Start: January 1, 2024
• Primary Completion (Estimated): January 1, 2034
• Study Completion (Estimated): January 1, 2034
• Last Updated: January 8, 2024

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)