MIG-HD: Multicentric Intracerebral Grafting in Huntington's Disease

Phase 2

Completed

• Conditions: Huntington Disease
• Interventions: Biological: graft intracerebral of foetal neurons

• Registration Number: NCT00190450
• Lead Sponsor: Assistance Publique - Hôpitaux de Paris

Brief Summary

The aim of this study is to assess the clinical benefit of intrastriatal grafting of human cells from the foetal ganglionic eminence in patients with Huntington's disease. The duration of the study will be 52 months. A first group of patients will be grafted at M13-14 (early G group) and a second group of patients will be grafted at M33-34 (late G group). The principal criterion is the comparison of the progression between M12 and M32 of the motor score (TMS) of the UHDRS between grafted patients (early G group) and not yet grafted patients (late G group). An additional evaluation will be performed to compare the progression in individual patients over the 52-month study period. We will thus be able to compare the pre and post-graft TMS progression for all patients.

Detailed Description

The aim of this study is to show the existence of a clinical benefit rising from a substitution of the striatal neurons degenerated among a large cohort of Huntington's patient at early stage by homologous neurons coming from human foetuses, This effect will be estimated, compared with a group of patients not treated at first, on the results of the motor scale of the Unified Huntington Disease Rating Scale (UHDRS, Huntington study group, on 1996).

Transplants will be realized in two surgical times to avoid the risk of hurts per-operating, BI-CAUDES, if transplants were realized at single time. The minimal interval between both transplants will be of 2 weeks, so as to let the patient recover of the first general anaesthesia It's a multicentric study of phase II randomised and controlled, with direct individual benefit.

Study Timeline

• Study Start: 2002-01
• Study First Submitted: 2005-09-15
• Study First Submitted QC: 2005-09-15
• Study First Posted: 2005-09-19
• Primary Completion: 2013-12
• Study Completion: 2013-12
• Status Verified: 2017-10
• Last Update Submitted: 2017-10-12
• Last Update Posted: 2017-10-13

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 54

Inclusion Criteria

• Disease clinically declared since at least 1 year ,UHDRS motor > or =5
• TFC > or = 10.
• CAG > or = 36
• Age between 18 and 65
• Family and socially integrated subject
• Informed consent.

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Exclusion Criteria

• Severe intellectual deterioration or neuropsychiatric disorders making the follow-up longitudinal too complicated (score MATTIS < 120).
• Not-observance of the appointments and the symptomatic treatments in pre-surgical period.
• Intercurrent disease making a surgical operation impossible.
• Associated disease having a neurological repercussion, intercurrent cerebral lesion with the IRM.
• Visceral affection engraves, evolutionary, which brings into play the vital forecast or makes risks for general anaesthesia.
• Mental Affection likely to disturb adhesion with the protocol, and in particular antecedents of hallucinations spontaneous and/or induced by the drugs; antecedents of serious depression having required repeated hospitalizations; antecedents of repeated suicide attempts.
• Cerebral morphological anomalies, others that those characteristic of the disease, noted with the IRM or the tomodensitometry.
• Participation in progress, or stopped since less than three months, with a therapeutic protocol of the Huntington's disease Exclusion Criteria (pre-randomization)
• TFC < 8
• Not-observance of the appointments and the symptomatic treatments in pre-surgical period.
• Intercurrent disease returning the surgery or impossible immunosuppression. v Subject completely isolated with his family and socially..
• UHDRS motor < 5.
• Positives serologies for HIV1, HIV2, AgP24, HTLV1 et 2, HEPATITE B, HEPATITE C, syphilis
• Psychiatric disorders being able to compromise the follow-up.
• Signs other than Huntington with the IRM.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
1	graft intracerebral of foetal neurons	Early Graft (Early G)
2	graft intracerebral of foetal neurons	Late Graft (Late G)

Primary Outcome Measures

Name	Time	Method
Motor UHDRS rating scale, at randomization, 20 month after transplant	during de study

Secondary Outcome Measures

Name	Time	Method
Neurologic, Cognitive, Neurophysiologic, Psychiatric, MRI and Pet-scan evaluation at randomization, 20 month after and at the end of the protocol	during the study

Trial Locations

Locations (1)

Hopital Henri Mondor; 🇫🇷 Paris, Ile de France, France