CAPIT-HD 2
Research type
Research Study
Full title
Beta Testing of a Revised Core Assessment Protocol for Intracranial transplantation in Huntington’s disease
IRAS ID
190391
Contact name
Anne Rosser
Contact email
Sponsor organisation
Cardiff University
Duration of Study in the UK
1 years, 11 months, 30 days
Research summary
Research Summary
Huntington’s disease (HD) is an inherited neurodegenerative disease for which there are no existing disease-modifying treatments. Repair-HD is a EU FP7 consortium that aims to establish all the preclinical requirements for transplantation of stem cell-derived neurons in HD in order to replace those lost to the disease process. These requirements include the generation of new clinical assessments for detailed monitoring of patients with HD who have undergone cell replacement therapy. This protocol describes the beta testing of a new clinical assessment battery: Core Assessment Protocol for Intrastriatal Transplanation in HD version 2 (CAPIT-HD2). CAPIT-HD2 represents a substantial revision of a previous CAPIT-HD battery published over 20 years ago, which is in need of updating in order to accommodate knowledge from clinical transplant studies over this time and to take advantage of technological advances in patient assessment.
HD is a complex disorder in which there is relentless deterioration of motor, cognitive and behavioural functions, usually from mid-life onwards. The original CAPIT battery aimed to capture elements of change in all three domains, but was based predominantly on subjective semi-quantitative assessment tools that have poor inter-rater reliability. Moreover, a number of deficits, such as impairments in social cognition, were not recognised when the CAPIT-HD battery was constructed, so we have developed novel assessments of these deficits, some of which are included in CAPIT-HD2. The beta testing will take place in established HD clinical centres in Cardiff, Manchester, Paris, and Munster by teams of researchers who are experienced in leading clinic research in HD. Patients will early to moderate HD will be assessed at baseline, and at one and twelve months later, to assess the reliability and sensitivity of the CAPIT-HD2 battery. Arrangements for data storage and analysis are in place.Summary of Results
Huntington’s disease (HD) is an inherited neurodegenerative disease for which there are no existing disease-modifying treatments. It is a complex disorder in which there is relentless deterioration of motor, cognitive and behavioural functions, usually from mid-life onwards. Repair-HD was a EU FP7 consortium that aimed to establish all the preclinical requirements for transplantation of stem cell-derived neurons in HD in order to replace those lost to the disease process. These requirements included the generation of new clinical assessments for detailed monitoring of patients with HD who have undergone cell replacement therapy. In this project we developed and beta-tested a new clinical assessment battery: Core Assessment Protocol for Intrastriatal Transplantation in HD version 2 (CAPIT-HD2). CAPIT-HD2 represents a substantial revision of a previous CAPIT-HD battery published over 20 years ago, which needed updating in order to accommodate knowledge from clinical transplant studies over this time and to take advantage of technological advances in patient assessment.
The original CAPIT battery aimed to capture elements of change in all three domains (motor cognitive and behaviour) but was based predominantly on subjective semi-quantitative assessment tools that have poor inter-rater reliability. Moreover, a number of deficits, such as impairments in social cognition, were not recognised when the CAPIT-HD battery was constructed, so we have developed novel assessments of these deficits, some of which are included in CAPIT-HD2. The beta testing took place in established HD clinical centres in Cardiff, Manchester, Paris, and Munster by teams of researchers who are experienced in leading clinic research in HD. Up to 20 patients at each site with early to moderate HD were assessed at baseline, and at one and twelve months later, to assess the reliability and sensitivity of the CAPIT-HD2 battery. Controls were also recruited and assessed. Assessments were performed in the same order on each patient at each centre, along with 4 questionnaires. Statistical analysis of results was undertaken to establish the a pool of tests which were the most sensitive to motor, cognitive, behavioural and psychiatric changes in the participants over time. In Cardiff, eligible and interested participants were reconsented and transitioned to the TRIDENT surgical trial. Some of these novel tests have now been published:
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Different Depression: Motivational Anhedonia Governs Antidepressant Efficacy in Huntington’s Disease, in press Brain Communications Lunven et al - A new approach to digitized cognitive monitoring: Validity of the SelfCog in Huntington’s Disease submitted Brain.
Hare E et al. Antecedent processes of apathy in Huntington’s disease are a sensitive biomarker for disease progression. Submitted to Plos one A modified form of the CAPIT-HD battery has been used in our ongoing clinical trial of neural transplantation in Huntington’s disease (Drew et al. Protocol for an open label: phase I trial within a cohort of foetal cell transplants in people with Huntington's disease. Brain Commun. 2021 Jan 19;3(1):fcaa230.REC name
Wales REC 7
REC reference
15/WA/0428
Date of REC Opinion
30 Nov 2015
REC opinion
Favourable Opinion