Safety and feasibility of neural transplantation in early to moderate Huntington's disease in the UK

ISRCTN	ISRCTN36485475
DOI	https://doi.org/10.1186/ISRCTN36485475
Protocol serial number	G9825903 and 071659
Sponsor	Medical Research Council (UK)
Funders	The Wellcome Trust (UK) (grant ref: 071659), Medical Research Council (UK) (grant ref: G9825903)

Submission date: 23/10/2000
Registration date: 23/10/2000
Last edited: 27/08/2019

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Mental and Behavioural Disorders

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English summary of protocol

Not provided at time of registration

Contact information

Prof Steve Dunnett
Scientific

The Brain Repair Group
Biomedical Science Building
Cardiff University
Museum Avenue
PO Box 911
Cardiff
CF10 3US
United Kingdom

Phone	+44 (0)29 2087 5188
Email	dunnett@cf.ac.uk

Study information

Primary study design	Interventional
Study design	Multicentre, randomised controlled trial
Secondary study design	Randomised controlled trial
Scientific title	Safety and feasibility of neural transplantation in early to moderate Huntington's disease in the UK
Study objectives	To determine the safety, feasibility and efficacy of foetal tissue transplantation as a restorative therapy for Huntington's disease
Ethics approval(s)	Not provided at time of registration
Health condition(s) or problem(s) studied	Neuroscience, psychiatry
Intervention	Patients in matched pairs randomly allocated to experimental and control conditions; the pairs are selected from a larger research cohort undergoing longitudinal assessment but not (yet) selected for participation in the surgical trial. All subjects will be examined using Positron Emission Tomography (PET) to assess survival and functional integration of the implanted grafts. The imaging component of the study, entitled 'Pre- and post-operative PET studies of Huntington's disease patients receiving human fetal striatal cell implants' (RTF - 071659), is funded by Wellcome trust, as part of the research training fellowship (for Y F Tai). As of September 2007, this trial is still open, but temporarily suspended following introduction of EU Tissue directive and pending upgrading of facilities to GMP compliance and MHRA accreditation. The end date of the follow up period will be extended to two years after thelast patient was operated on. Please also note that as of 22nd January 2007 the Sponsor institution of this trial changed. The previous sponsor was: Cardiff University (UK) Cardiff CF10 3XQ United Kingdom +44 (0)29 2087 4000 Http://www.cardiff.ac.uk
Intervention type	Other
Primary outcome measure(s)	Safety trial: 1. Presence/absence of adverse events 2. Feasibility: a. co-ordination of an effective and efficient programme for foetal tissue collection b. biosafety assessment c. preparation and implantation into identified patients and their long term evaluation on the relevant structural and functional criteria of graft survival and efficacy Efficacy trial: Retardation or reversal of the progress of the disease in the affected patients as determined by the neurological, neuropsychological and neuropsychiatric and imaging criteria of neuropsychological tests of cognition using the CAmbridge Neuropsychological Test Automated Battery (CANTAB) test battery.
Key secondary outcome measure(s)	No secondary outcome measures
Completion date	30/06/2008
Reason abandoned (if study stopped)	This trial is still open, but temporarily suspended following introduction of EU Tissue directive and pending upgrading of facilities to GMP compliance and MHRA accreditation

Eligibility

Participant type(s)	Patient
Age group	Adult
Sex	Not Specified
Target sample size at registration	20
Total final enrolment	17
Key inclusion criteria	1. Genetically confirmed Huntington's disease 2. Early to moderate stage of disease 3. Presence of motor signs 4. Availability of one close primary informant
Key exclusion criteria	1. Advanced disease 2. Still employable in usual occupation 3. Other concurrent major illness 4. Current serious psychiatric disturbance 5. Inadequate social support 6. Below the age of 18
Date of first enrolment	01/10/1999
Date of final enrolment	30/06/2008

Locations

Countries of recruitment

United Kingdom
Wales

Study participating centre

The Brain Repair Group

Cardiff
CF10 3US
United Kingdom

Results and Publications

Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Results article	results	01/12/2002		Yes	No
Results article	results	01/06/2013		Yes	No
Results article	results	01/12/2018	27/08/2019	Yes	No

Editorial Notes

27/08/2019: Publication reference and total final enrolment added.