A single site, open label, phase I study to assess the safety and feasibility of foetal cell transplants in the striatum of people with Huntington’s disease

Not Applicable

Completed

• Conditions: Huntington's disease; Nervous System Diseases; Huntington disease

• Registration Number: ISRCTN52651778
• Lead Sponsor: Cardiff University

Brief Summary

2021 Protocol article in https://pubmed.ncbi.nlm.nih.gov/33543141/ protocol (added 08/02/2021)

Detailed Description

Not available

Study Timeline

• Study Start: 2018-04-04
• Study Completion: 2023-02-28
• Last Update Posted: 8 July 2024

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 22

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

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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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: <br>1. The incidence of significant additional, permanent neurological deficits<br>2. The incidence of a clinically significant intracranial haemorrhage<br>3. The incidence of clinically significant intracranial infection