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L'essai clinique NCT05858268 pour Paralysie cérébrale est actif, ne recrute pas. Consultez la vue en carte du Radar des Essais Cliniques et les outils de découverte par IA pour tous les détails, ou posez vos questions ici. | ||
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NeuralNET Cerebral Palsy Pilot Study 66
Les détails de l'essai clinique sont principalement disponibles en anglais. Cependant, l'IA Trial Radar peut vous aider ! Cliquez simplement sur 'Expliquer l'étude' pour voir et discuter des informations sur l'étude dans la langue sélectionnée.
L'essai clinique NCT05858268 est une étude observationnel pour Paralysie cérébrale. Son statut actuel est : actif, ne recrute pas. L'étude a débuté le 14 avril 2023 et vise à recruter 66 participants. Dirigée par University of Cambridge, l'étude devrait être terminée d'ici le 31 janvier 2026. Les données du site ClinicalTrials.gov ont été mises à jour pour la dernière fois le 30 mars 2025.
Résumé succinct
The NeuralNET Cerebral Palsy Pilot Study is testing a genetic testing pathway in the NHS for children with cerebral palsy (CP). Other studies suggest that almost one in three peoples' CP is caused by a change in their genes, but more studies are needed to confirm this. A genetic test called whole genome sequencing (WGS) will be used for children who have CP to look for rare changes in genes that cause the condition, ...Afficher plus
Titre officiel
The NeuralNET: Research to Impact Diagnosis, Mechanistic Understanding and Treatment of Children's Brain and Mental Health Disorders - A Pilot Study in Cerebral Palsy
Pathologies
Paralysie cérébraleAutres identifiants de l'étude
- IRAS: 319781
Numéro NCT
Date de début (réel)
2023-04-14
Dernière mise à jour publiée
2025-03-30
Date de fin (estimée)
2026-01-31
Inscription (estimée)
66
Type d'étude
Observationnel
Statut
Actif, ne recrute pas
Bras / Interventions
| Groupe de participants/Bras | Intervention/Traitement |
|---|---|
N/A | Séquençage du génome entier Whole-genome sequencing |
Critère principal d'évaluation
Critère secondaire d'évaluation
| Critères d'évaluation | Description de la mesure | Période |
|---|---|---|
Feasibility of rapid whole-genome sequencing of children with cerebral palsy | The investigators will measure feasibility of rapid whole-genome sequencing of children with cerebral palsy by successful delivery of WGS results to 66 children with a clinical diagnosis of CP within 12 weeks of blood sample receipt in the laboratory. | 16 months |
| Critères d'évaluation | Description de la mesure | Période |
|---|---|---|
Uptake of WGS testing by families with a child with CP | The percentage of uptake of WGS testing by families with a child with CP will be measured by comparing the number of families invited to the study to the number who proceed with testing at baseline | Baseline |
Reasons for declining offer of WGS | The reasons for declining the offer of WGS provided by families voluntarily to the referring clinician will be measured in aggregate using a questionnaire at the close of the recruitment | 16 months |
Identification of specific genetic contributors to CP in the UK | Specific genetic contributors to CP in children in the UK will be measured using the collation of WGS results at the close of the recruitment | 16 months |
Parent/guardian intolerance for uncertainty | Intolerance of uncertainty of parent/guardian will be measured with the short version of the validated Intolerance for Uncertainty scale, which is a 12-item self-administered questionnaire where items are rated on a 5-point Likert scale where a higher score means higher intolerance of uncertainty. | baseline |
Parent/guardian attitude to genome sequencing | Four-item scale via self-administered questionnaire examining general attitudes (eg harmful vs. beneficial, unimportant vs. important, etc.) of parent/guardian to genome sequencing measured on a five-point Likert scale, where a higher score means more positive attitude. | baseline and 16 months |
Parent/guardian decisional conflict | Parent/guardian decisional conflict will be measured with validated sixteen-item self-administered questionnaire which assesses decisional certainty or conflict about a healthcare decision on a five-point Likert scale, where a higher score indicates higher uncertainty or conflict. | baseline |
Parent/guardian empowerment | Parent/guardian empowerment relating to genomic medicine will be measured with the Genomics Outcome Scale, a validated six-item self-administered questionnaire with a 5-point Likert scale, where a higher score indicates higher theoretical construct of empowerment. | baseline, 16 months |
Parent/guardian decisional regret | Parent/guardian decisional regret about WGS will be measured with a validated 5-item self-administered Decisional Regret Scale, which uses a 5-point Likert scale where higher scores indicate greater decisional regret. | 16 months |
Parent/guardian psychological impact of WGS | Parent/guardian psychological impact of WGS will be measured with adapted 10-item version of the validated Feelings About genomic Testing Results (FACToR) scale, which uses a 5-point Likert scale to measure agreement with specific feelings about the impact of result disclosure after genomic testing, where a higher score indicates greater agreement with those feelings. | 16 months |
Impact of WGS on family quality of life | Impact of WGS on the family's quality of life will be measured using the Family Impact Module of The PEDS-QL, which is a 36-item validated self-administered scale. It uses a 5-point Likert scale where higher scores indicated greater agreement with specific items. | 16 months |
Clinical utility of WGS testing in children with CP | The clinical utility of WGS testing in children with CP from the Paediatrician's perspective will measured using the validated Clinician-reported Genetic testing Utility InDEx (C-GUIDE), a self-administered questionnaire which includes 17 items related to results received for the primary indication for testing and 9 items related to secondary variant results received. Individual items scores range from -1 to 2. An item score \>0 indicates positive utility, item scores \<0 indicate presence of negative utility, and item scores of 0 indicate absence of utility. | 16 months |
Number of candidate variants which warrant further investigation of pathogenicity | Number of candidate variants which warrant further investigation of pathogenicity via collaborative studies will be measured by collating potentially pathogenic variants of uncertain significance at the completion of the study. | 16 months |
Generation of data to support the refinement of clinical criteria for WGS for CP | Data to support the refinement of clinical criteria for assessing CP patient suitability for WGS testing will be measured using the correlation of clinical features with identification of causative variants by WGS | 16 months |
Assistant à la participation
Critères d'éligibilité
Âges éligibles
Enfant
Sexes éligibles
Tous
Children with cerebral palsy (CP):
- Has a clinical diagnosis of CP in the medical record
- Any GMFCS score (GMFCS 1-5)
- Does not have a known genetic diagnosis that explains the CP phenotype
- Has a parent/legal guardian available who can consent and is willing to complete study questionnaires
- Invited to participate by a clinician at a participating recruitment site
Biological parents of children with CP will also be included in the study if they are:
- A biological parent of the child
- Aged 18 years or above
- Willing and able to give informed consent for participation in the study Participant type
Children with cerebral palsy (CP):
- Children that have a pre-existing genetic diagnosis from whole genome sequencing or whole-exome sequencing
- Children not matching the inclusion criteria
Biological parents of children with CP will be excluded from the study if they do not meet the biological parent inclusion criteria i.e. they ARE NOT:
- A biological parent of the child
- Aged 18 years or above
- Willing and able to give informed consent for participation in the study
University of Cambridge- 🏛️Rosetrees Trust
Partie responsable de l'étude
Heather Pierce, Investigateur principal, Research Study Coordinator, University of Cambridge
Aucune donnée de contact disponible
1 Centres de l'étude dans 1 pays
Cambridge University Hospitals NHS Trust, Cambridge, CB2 0QQ, United Kingdom