Characterizing circulating biomarkers for childhood dementia disorders: A scoping review of clinical trials
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 ar...
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Elsevier
2025-03-01
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| Series: | Neurotherapeutics |
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| Online Access: | http://www.sciencedirect.com/science/article/pii/S1878747925000248 |
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| author | Arlene D'Silva James Barnes Jason Djafar Kaustuv Bhattacharya Jingya Yan Shekeeb Mohammad Sushil Bandodkar Alexandra Johnson Michel Tchan Christina Miteff Kristina L. Elvidge Russell C. Dale Michelle Farrar |
| author_facet | Arlene D'Silva James Barnes Jason Djafar Kaustuv Bhattacharya Jingya Yan Shekeeb Mohammad Sushil Bandodkar Alexandra Johnson Michel Tchan Christina Miteff Kristina L. Elvidge Russell C. Dale Michelle Farrar |
| author_sort | Arlene D'Silva |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-cbd89c34b1c24a05b0afdf6cb3d7c197 |
| institution | OA Journals |
| issn | 1878-7479 |
| language | English |
| publishDate | 2025-03-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Neurotherapeutics |
| spelling | doaj-art-cbd89c34b1c24a05b0afdf6cb3d7c1972025-08-20T02:18:00ZengElsevierNeurotherapeutics1878-74792025-03-01222e0054610.1016/j.neurot.2025.e00546Characterizing circulating biomarkers for childhood dementia disorders: A scoping review of clinical trialsArlene D'Silva0James Barnes1Jason Djafar2Kaustuv Bhattacharya3Jingya Yan4Shekeeb Mohammad5Sushil Bandodkar6Alexandra Johnson7Michel Tchan8Christina Miteff9Kristina L. Elvidge10Russell C. Dale11Michelle Farrar12Department of Neurology, The Sydney Children's Hospitals Network, Sydney, Australia; Discipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, The University of New South Wales, Sydney, Australia; UNSW RNA Institute, The University of New South Wales, Sydney, Australia; Corresponding author.Department of Neurology, The Sydney Children's Hospitals Network, Sydney, Australia; Discipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, The University of New South Wales, Sydney, AustraliaDepartment of Neurology, The Sydney Children's Hospitals Network, Sydney, Australia; Discipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, The University of New South Wales, Sydney, AustraliaSydney Children's Hospitals' Network, Westmead, NSW 2145, Australia; Clinical School, The Children's Hospital at Westmead, Faculty of Medicine and Health, University of Sydney, NSW, AustraliaClinical School, The Children's Hospital at Westmead, Faculty of Medicine and Health, University of Sydney, NSW, Australia; Kids Neuroscience Centre, The Children's Hospital at Westmead, Faculty of Medicine and Health, University of Sydney, Clinical School, NSW, AustraliaClinical School, The Children's Hospital at Westmead, Faculty of Medicine and Health, University of Sydney, NSW, Australia; Kids Neuroscience Centre, The Children's Hospital at Westmead, Faculty of Medicine and Health, University of Sydney, Clinical School, NSW, AustraliaSydney Children's Hospitals' Network, Westmead, NSW 2145, Australia; Clinical School, The Children's Hospital at Westmead, Faculty of Medicine and Health, University of Sydney, NSW, AustraliaDepartment of Neurology, The Sydney Children's Hospitals Network, Sydney, Australia; Discipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, The University of New South Wales, Sydney, AustraliaDepartment of Genetic Medicine, Westmead Hospital, Westmead, NSW 2145, Australia; Faculty of Medicine and Health, University of Sydney, NSW, AustraliaChildren, Young People and Families Directorate of Hunter New England Local Health District and John Hunter Children's Hospital, New Lambton Heights, NSW 2305, AustraliaChildhood Dementia Initiative, Brookvale, NSW, 2100, AustraliaClinical School, The Children's Hospital at Westmead, Faculty of Medicine and Health, University of Sydney, NSW, Australia; Kids Neuroscience Centre, The Children's Hospital at Westmead, Faculty of Medicine and Health, University of Sydney, Clinical School, NSW, AustraliaDepartment of Neurology, The Sydney Children's Hospitals Network, Sydney, Australia; Discipline of Paediatrics and Child Health, School of Clinical Medicine, UNSW Medicine and Health, The University of New South Wales, Sydney, Australia; UNSW RNA Institute, The University of New South Wales, Sydney, AustraliaChildhood 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.http://www.sciencedirect.com/science/article/pii/S1878747925000248BiomarkersChildhood dementiaClinical trialsEndpointsMetabolomicsProteomics |
| spellingShingle | Arlene D'Silva James Barnes Jason Djafar Kaustuv Bhattacharya Jingya Yan Shekeeb Mohammad Sushil Bandodkar Alexandra Johnson Michel Tchan Christina Miteff Kristina L. Elvidge Russell C. Dale Michelle Farrar Characterizing circulating biomarkers for childhood dementia disorders: A scoping review of clinical trials Neurotherapeutics Biomarkers Childhood dementia Clinical trials Endpoints Metabolomics Proteomics |
| title | Characterizing circulating biomarkers for childhood dementia disorders: A scoping review of clinical trials |
| title_full | Characterizing circulating biomarkers for childhood dementia disorders: A scoping review of clinical trials |
| title_fullStr | Characterizing circulating biomarkers for childhood dementia disorders: A scoping review of clinical trials |
| title_full_unstemmed | Characterizing circulating biomarkers for childhood dementia disorders: A scoping review of clinical trials |
| title_short | Characterizing circulating biomarkers for childhood dementia disorders: A scoping review of clinical trials |
| title_sort | characterizing circulating biomarkers for childhood dementia disorders a scoping review of clinical trials |
| topic | Biomarkers Childhood dementia Clinical trials Endpoints Metabolomics Proteomics |
| url | http://www.sciencedirect.com/science/article/pii/S1878747925000248 |
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