Challenges and constraints in evaluating novel therapies for neurodegeneration
Brighton, UK

Speaker: Kerry Hood

This is a talk at the International Clinical Trials Methodology Conference (ICTMC), Brighton, UK, 6th – 9th October 2019.  Venue is the Hilton Brighton Metropole.

Parallel Session 10D – UKCRN Showcase


Kerry Hood, Monica Busse, Cheney Drew, Philip Pallmann, Liam Gray and Anne Rosser.


Background and Aim;

Huntington’s Disease (HD) is a progressive, inherited neurological disorder characterised by a triad of motor, cognitive and psychiatric symptoms. It is an incurable, progressive condition that seriously erodes quality of life and has a high societal cost. HD research is entering an exciting phase, with considerable experimental advanced therapies such as huntingtin lowering strategies and cell therapies on the horizon.  Many of these potential therapeutic strategies require direct delivery of the therapeutic agents into the brain.  This is complex and presents multiple challenges. Our aim was to design and implement a trial that would set standards for the evaluation of novel therapeutics in HD and other neurodegenerative disorders.



HD principle investigators, international authorities on trial design, bio-ethics, cell transplantation and public and patient involvement representatives contributed to consensus discussion in a workshop setting held in London, October 2016



Workshop participants agreed that: it is critical to learn from the collaborative efforts in the field of primary fetal cell transplantation over the last 20 years;  a stabilized procedure including device-specific accuracy and safety needs to be assured; participant consent and information support to be considered as an integral part of any trial design; and, critically, there needs to be an alternative to the ‘gold-standard’ randomised, double-blind placebo-controlled trial design if regulatory approvals are to be achieved in a timely and efficient manner, and the field of complex surgical therapies is to progress in an ethical and efficient manner.



Consensus views generated at the REPAIR-HD workshop were input into the design of a Phase 1 safety and feasibility trial of foetal cell transplants in HD (TRIDENT: ISRCTN52651778). This uses a variation of the Trial Within Cohort design, involving 3-stage consent, an embedded qualitative evaluation and a novel selection process for transplant allocation.

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