Characterizing circulating biomarkers for childhood dementia disorders: A scoping review of clinical trials.

D'Silva, A; Barnes, J; Djafar, J; Bhattacharya, K; Yan, J; Mohammad, S; Bandodkar, S; Johnson, A; Tchan, M; Miteff, C; Elvidge, KL; Dale, RC; Farrar, M

Characterizing circulating biomarkers for childhood dementia disorders: A scoping review of clinical trials.

D'Silva, A Barnes, J Djafar, J Bhattacharya, K Yan, J

Mohammad, S Bandodkar, S Johnson, A Tchan, M Miteff, C Elvidge, KL Dale, RC Farrar, M

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Publisher:: Elsevier
Publication Type:: Journal Article
Citation:: Neurotherapeutics, 2025, 22, (2), pp. e00546
Issue Date:: 2025-02-12

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Field	Value	Language
dc.contributor.author	D'Silva, A
dc.contributor.author	Barnes, J
dc.contributor.author	Djafar, J
dc.contributor.author	Bhattacharya, K
dc.contributor.author	Yan, J https://orcid.org/0000-0002-0970-1570
dc.contributor.author	Mohammad, S
dc.contributor.author	Bandodkar, S
dc.contributor.author	Johnson, A
dc.contributor.author	Tchan, M
dc.contributor.author	Miteff, C
dc.contributor.author	Elvidge, KL
dc.contributor.author	Dale, RC
dc.contributor.author	Farrar, M
dc.date.accessioned	2025-03-09T02:53:53Z
dc.date.available	2025-01-29
dc.date.available	2025-03-09T02:53:53Z
dc.date.issued	2025-02-12
dc.identifier.citation	Neurotherapeutics, 2025, 22, (2), pp. e00546
dc.identifier.issn	1933-7213
dc.identifier.issn	1878-7479
dc.identifier.uri	http://hdl.handle.net/10453/185594
dc.description.abstract	Childhood dementias, a group of neurological disorders are characterised by neurocognitive decline, with physical and psychosocial impacts for individuals. With therapy available for <5 % of childhood dementias, there is a high level of unmet need. Integration of biomarkers in clinical trials are important to characterize distinctive biological activities and interrogate targets for therapeutic development. This study reviewed four clinical trial registries to examine circulating biomarkers in childhood dementias. Findings from 262 studies were synthesized across 49/72 (68 %) childhood dementia disorders. Disease-related biomarkers were associated with 1) the primary pathophysiology 2) downstream pathogenic events 3) drug-related pharmacokinetics, safety and/or tolerability. The predominant biological measures were metabolites linked to the primary pathophysiological pathway (102 measures, 185 studies), while use of cytoskeletal proteins (3 measures, 15 studies), inflammatory mediators (19 measures, 24 studies), oxidative stress-related analytes (15 measures, 8 studies), neurotransmitters or related neuro-metabolites (3 measures, 5 studies) were limited. A range of potential biomarkers are used in clinical trials; however, their use is inconsistent and under utilised among conditions. Development of a panel of biomarkers has potential to interrogate and link shared biological pathways across the heterogeneity of childhood dementias to exert a significant impact for the development of disease-modifying therapies.
dc.format	Print-Electronic
dc.language	eng
dc.publisher	Elsevier
dc.relation.ispartof	Neurotherapeutics
dc.relation.isbasedon	10.1016/j.neurot.2025.e00546
dc.rights	info:eu-repo/semantics/openAccess
dc.subject	1109 Neurosciences, 1115 Pharmacology and Pharmaceutical Sciences, 1117 Public Health and Health Services
dc.subject.classification	Neurology & Neurosurgery
dc.subject.classification	3209 Neurosciences
dc.subject.classification	3214 Pharmacology and pharmaceutical sciences
dc.subject.classification	5202 Biological psychology
dc.title	Characterizing circulating biomarkers for childhood dementia disorders: A scoping review of clinical trials.
dc.type	Journal Article
utslib.citation.volume	22
utslib.location.activity	United States
utslib.for	1109 Neurosciences
utslib.for	1115 Pharmacology and Pharmaceutical Sciences
utslib.for	1117 Public Health and Health Services
pubs.organisational-group	University of Technology Sydney
pubs.organisational-group	University of Technology Sydney/Faculty of Science
pubs.organisational-group	University of Technology Sydney/Faculty of Science/School of Mathematical and Physical Sciences
utslib.copyright.status	open_access	*
dc.rights.license	This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0). To view a copy of this license, visit https://creativecommons.org/licenses/by/4.0/
dc.date.updated	2025-03-09T02:53:48Z
pubs.issue	2
pubs.publication-status	Published online
pubs.volume	22
utslib.citation.issue	2

Abstract:

Childhood dementias, a group of neurological disorders are characterised by neurocognitive decline, with physical and psychosocial impacts for individuals. With therapy available for <5 % of childhood dementias, there is a high level of unmet need. Integration of biomarkers in clinical trials are important to characterize distinctive biological activities and interrogate targets for therapeutic development. This study reviewed four clinical trial registries to examine circulating biomarkers in childhood dementias. Findings from 262 studies were synthesized across 49/72 (68 %) childhood dementia disorders. Disease-related biomarkers were associated with 1) the primary pathophysiology 2) downstream pathogenic events 3) drug-related pharmacokinetics, safety and/or tolerability. The predominant biological measures were metabolites linked to the primary pathophysiological pathway (102 measures, 185 studies), while use of cytoskeletal proteins (3 measures, 15 studies), inflammatory mediators (19 measures, 24 studies), oxidative stress-related analytes (15 measures, 8 studies), neurotransmitters or related neuro-metabolites (3 measures, 5 studies) were limited. A range of potential biomarkers are used in clinical trials; however, their use is inconsistent and under utilised among conditions. Development of a panel of biomarkers has potential to interrogate and link shared biological pathways across the heterogeneity of childhood dementias to exert a significant impact for the development of disease-modifying therapies.

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http://hdl.handle.net/10453/185594