Brogueira Rodrigues, Filipe;
(2024)
Enhancing clinical trials methodology in Huntington’s disease.
Doctoral thesis (Ph.D), UCL (University College London).
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Abstract
Huntington’s disease is a fatal neurodegenerative condition without cure, caused by a fully penetrant mutation in the huntingtin gene. It has a progressive course with loss of independence and quality of life, and increased disability, being devastating to patients and their families. Despite a long history of clinical drug development, most therapeutic development efforts in Huntington’s disease thus far have been unsuccessful, and no disease modifying interventions are available. It is therefore urgent to enhance clinical trials’ tools and methodologies to improve the success rate of drug development in Huntington’s disease. This will help the community design better clinical trials that are more likely to identify successful treatments, and in the long term contribute to improving the care of people with Huntington’s disease. The overarching aim of this thesis is to contribute to the advancement of instruments and knowledge intended to facilitate clinical drug development in Huntington’s disease. More specifically, this thesis systematically appraises and summarizes the evidence about the methodology and results of clinical trials in Huntington’s disease; investigates the factors associated with the feasibility of intrathecal drug delivery in Huntington’s disease clinical trials including cerebrospinal fluid dynamics and lumbar puncture safety and feasibility; and evaluates the clinically validity of mutant huntingtin protein, neurofilament light chain protein and selected kynurenine pathway metabolites as biofluid biomarkers for clinical trials use in Huntington’s disease.
| Type: | Thesis (Doctoral) |
|---|---|
| Qualification: | Ph.D |
| Title: | Enhancing clinical trials methodology in Huntington’s disease |
| Open access status: | An open access version is available from UCL Discovery |
| Language: | English |
| Additional information: | Copyright © The Author 2024. Original content in this thesis is licensed under the terms of the Creative Commons Attribution-NonCommercial 4.0 International (CC BY-NC 4.0) Licence (https://creativecommons.org/licenses/by-nc/4.0/). Any third-party copyright material present remains the property of its respective owner(s) and is licensed under its existing terms. Access may initially be restricted at the author’s request. |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology > Neurodegenerative Diseases |
| URI: | https://discovery-pp.ucl.ac.uk/id/eprint/10185154 |
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