Huntington's disease Nervous System Diseases Huntington disease
Conditions
Interventions
A subset of participants within a comparator cohort will be assessed for suitablilty and safety of undergoing transplant surgery. Those deemed to be eligible for surgery will be randomly ordered and 5
Sponsors
Cardiff University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Confirmed diagnosis of HD through genetic testing (CAG=39) 2. =18 years of age 3. Stage I or II disease (assessed using the Total Functional Capacity Scale with a score equal to 12 or below) and diagnosed as having motor onset 4. Participant is ambulatory
Exclusion criteria
Exclusion criteria: 1. People without capacity to consent to the trial 2. Any ongoing major psychiatric disorder that would preclude the ability to take part in functional assessments and the ability to give informed consent 3. Lack of carer, significant other or family member to support attendance at regular assessments. Exclusion criteria for the surgical intervention are: 4. Participants on any long-term anticoagulant medication (to include aspirin, warfarin, clexane) 5. Any significant medical condition that would compromise the safety of anaesthesia and/ or surgery including; Q-T interval prolongation, torsades des pointes or ventricular arrhythmia 6. Participants not deemed to be suitable for transplant surgery (e.g. inadequate striatal volume on MRI) 7. Pregnancy and breastfeeding Added 31/07/2019: 8. Previous immunizing event such as blood transfusion or previous transplant 9. Contraindications to 3T MRI (such as a fitted pacemaker) 10. Contraindications to PET 11. Any contraindication to immunosuppressive therapy 12. Positive blood tests for; Human Immunodeficiency Virus (HIV), Hepatitis B Virus (HBV), Hepatitis C Virus (HCV), active Cytomegalovirus (CMV)infection, active Toxoplasma Gondii (Toxo G), Human T-cell lymphotropic virus type 1 (HTLV 1), serology for active treponema pallidum 13. Women of childbearing potential and male participants with partners of childbearing potential who will not commit to the prevention of and active monitoring for pregnancy by using a highly effective form of contraception and undertaking regular pregnancy tests whilst enrolled in the study/or on immunosuppressant
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety will be assessed at 4 weeks by the trial steering committee, who will review pre and post-operative 3T MRI scans, surgical records from the transplant procedure, medical records from the participants inpatient stay and any case report forms completed during the postoperative inpatient stay, including blood results, vital signs and any serious adverse events reported. Defined by: 1. The incidence of significant additional, permanent neurological deficits 2. The incidence of a clinically significant intracranial haemorrhage 3. The incidence of clinically significant intracranial infection | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Feasibility and acceptability of clinical trial processes as determined by: 1.1. Recruitment rates assessed using screening and recruitment logs at the end of the trial 1.2. Retention of participants assessed from any participant withdrawal forms at the end of the trial 1.3. Participant and carer experiences elicited from the pre- and post-operative interviews 2. Fidelity of neurosurgery is measured using the evaluation of MRI and PET scanning to measure the successful delivery of cells and accurate neurosurgical graft placement at 1 month post-operatively (3T MRI) and 12 months postoperatively (PET) 3. Long-term (12 months) safety of transplantation is measured using MRI and PET scans to assess the growth profile of the graft and the development of clinically significant inflammatory or immune reactions as assessed by clinician and advisory groups at 12 months 4. Research, treatment and immunosuppression costs will be documented across the duration of the trial using a mix of standard unit costs and detailed research costs for all research procedures 5. Development of fidelity markers through analysis of video-data capture of the surgery and graft survival over 1 year as determined by structural MRI/PET | — |
Countries
United Kingdom, Wales
Contacts
Public ContactCheney Drew
Outcome results
None listed