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临床试验 NCT05858268 针对脑瘫目前进行中（不再招募）。请查看临床试验雷达卡片视图和 AI 发现工具了解所有详情，或在此提出任何问题。 | ||
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NeuralNET Cerebral Palsy Pilot Study 66
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临床试验NCT05858268是一项针对脑瘫的观察研究试验，目前试验状态为进行中（不再招募）。试验始于2023年4月14日，计划招募66名患者。该研究由University of Cambridge主导，预计于2026年1月31日完成。试验数据来源于ClinicalTrials.gov，最后更新时间为2025年3月30日。
简要概括
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, ...显示更多
官方标题
The NeuralNET: Research to Impact Diagnosis, Mechanistic Understanding and Treatment of Children's Brain and Mental Health Disorders - A Pilot Study in Cerebral Palsy
疾病
脑瘫其他研究标识符
- IRAS: 319781
试验组/干预措施
| 参与者组/试验组 | 干预措施/治疗方法 |
|---|---|
不适用 | 全基因组测序 Whole-genome sequencing |
主要终点
次要终点
| 结果指标 | 度量标准描述 | 时间框架 |
|---|---|---|
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 |
| 结果指标 | 度量标准描述 | 时间框架 |
|---|---|---|
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 |
参与助手
资格标准
适龄参与研究
儿童
适龄性别
全部
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
研究责任方
Heather Pierce, 主要研究者, Research Study Coordinator, University of Cambridge
没有联系数据。
1 位于 1 个国家/地区的研究中心
Cambridge University Hospitals NHS Trust, Cambridge, CB2 0QQ, United Kingdom