F55 Repair-hd project – WP5: preparation for clinical intervention

Background The core assessment for intra-cerebral transplantation in HD (Quinn et al, 1996) was never validated and used as a whole in previous transplant studies. Aims The main objective of Repair-HD WP5 is to develop and validate the CAPIT-HD2 as a reference battery for surgical trial. Method Through a series of workshops and in-depth review of the relevant literature, members of the consortium selected existing tests the most sensitive to change, which were retained in the provisional protocol, and identified domains in which additional tasks were required for longitudinal assessments in small cohort of patients. The new tasks were assessed in the centers in which they were developed: Creteil for cognition, Munster for motor deficits, Manchester for psychiatry and Cardiff for functional aspects and adapted for English, French and German participants. Most of them were digitalized to replace existing tasks which are heavily questionnaire-based and subject to rater bias. The development of a specified GCP and EMA compliant eCRF allowed centralization of data entry. The proposed provisional CAPIT-HD2 consisting of the best old and new tasks was then proposed for beta-testing in France, UK and Germany. 80 HD carriers and 40 controls were enrolled with the objective of assessing the psychometric properties and sensitivity to change of the core assessment battery. Participants were assessed at baseline, month 1 and month 12. Final results from all centers are expected in October 2018. Conclusion While no firm conclusion can be drawn as of now, preliminary analyses provided promising results as the new tests have proven to be efficient in discriminating between controls and patients or between stages of the disease. Moreover, decline in cognitive functions could be detected over a period of 11 months with a population as low as 20 patients and 18 controls at Creteil. Validation from the other centers is expected.