Human Epilepsy Project 3: Newly Diagnosed Idiopathic Generalized Epilepsy
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 316
- 试验地点
- 36
- 主要终点
- Average heart rate
研究概览
简要总结
By carrying a careful, large-scale and ambitious prospective study of a cohort of participants with generalized epilepsy, the study team hopes to clarify the likelihood of response and remission in this type of epilepsy, and try to explore the underlying biological drivers of treatment response, including novel realms of exploration such as impact of the microbiome, and genetics. The identification of biomarkers that predict the likelihood of disease response would allow epilepsy patients to make more informed decisions about the factors affecting their quality of life, including plans for driving, relationships, pregnancy, schooling, work, and play. In addition to its impact on clinical care, the data and specimens collected in HEP3, including sequential electrophysiology, biochemical profiles and neuroimaging and banked DNA for future genomics studies, have the potential to provide new insights into the biological basis of IGE, thereby advancing the discovery of effective treatments and cures. By enrolling both newly diagnosed subjects (prognosis unknown) as well as subjects with established IGE who are already determined to be treatment resistant or treatment responsive, the study team can immediately test potential biomarkers in a confirmation cohort, which will accelerate identification of predictive biomarkers.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 13 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Cohort 1: Newly Diagnosed IGE
- •Inclusion criteria:
- •Age ≥13 years at time of enrollment
- •Age ≥8 years at time of seizure onset
- •Clinical seizure(s) and history consistent with IGE. The only permitted seizure types are absence, myoclonus or generalized tonic-clonic convulsions
- •Occurrence of at least 1 seizure of any type in the 6 months prior to treatment
- •Patients must have one of the following:
- •GTCSs alone accompanied by generalized spike-wave consistent with IGE per adjudication review
- •GTCSs with a history of absence and/or myoclonus, accompanied by generalized spike-wave consistent with IGE per adjudication review
- •GTCSs associated with a history of absence and/or myoclonus, not accompanied by generalized spike-wave consistent with IGE per adjudication review
- •A clear history of absence and/or myoclonus, accompanied by generalized spike-wave consistent with IGE per adjudication review
- •Availability of a complete medication history since initiation of treatment, including doses and date of initiation
- •No competing cause of epilepsy (e.g. traumatic brain injury)
- •AED treatment (for seizures) instituted not more than 12 months before enrollment
排除标准
- •Focal epilepsy
- •Generalized/focal epilepsy mixed syndromes
- •Progressive neurological disorder (brain tumor, Alzheimer's disease, progressive myoclonic epilepsy, etc.)
- •Epileptic or developmental encephalopathy
- •Major medical co-morbidities (e.g. renal failure requiring dialysis, metastatic cancer, HIV, or significant liver or renal disease)
- •Autism Spectrum Disorder
- •History of chronic drug or alcohol abuse (misuse or excessive use that interferes with activities of daily living) within the last 2 years
- •Seizures only during pregnancy
- •History of previous or current significant psychiatric disorder that would interfere with study requirements
- •Cohort 2: Longstanding Treatment Responsive
- •Inclusion Criteria:
- •Age ≥13 years at time of enrollment
- •Age ≥8 years at time of seizure onset
- •Clinical seizure(s) and history consistent with IGE. The only permitted seizure types are absence, myoclonus or generalized tonic-clonic convulsions
- •Patients must have had one of the following:
- •GTCSs alone accompanied by generalized spike-wave consistent with IGE per adjudication review
- •GTCSs with a history of absence and/or myoclonus, accompanied by generalized spike-wave consistent with IGE per adjudication review
- •GTCSs associated with a history of absence and/or myoclonus, not accompanied by generalized spike-wave consistent with IGE per adjudication review
- •A clear history of absence and/or myoclonus, accompanied by generalized spike-wave consistent with IGE per adjudication review
- •Availability of a complete medication history since initiation of treatment, including doses and date of initiation
- •No competing cause of epilepsy (e.g. traumatic brain injury)
- •Two years of well-controlled seizures.
- •No convulsive seizures in the last two years
- •Myoclonic or absence seizures must be rare (<2 per year) and non-disabling
- •Ongoing therapy with > 1 antiseizure medication
- •Exclusion Criteria:
- •Focal epilepsy
- •Paroxysmal nonepileptic seizures
- •Generalized/focal epilepsy mixed syndromes
- •Progressive neurological disorder (brain tumor, Alzheimer's disease, progressive myoclonic epilepsy, etc.)
- •Epileptic or developmental encephalopathy
- •Major medical co-morbidities (e.g. renal failure requiring dialysis, metastatic cancer, HIV, or significant liver or renal disease)
- •Autism Spectrum Disorder
- •History of chronic drug or alcohol abuse (misuse or excessive use that interferes with activities of daily living) within the last 2 years
- •Seizures only during pregnancy
- •History of previous or current significant psychiatric disorder that would interfere with study requirements
- •Cohort 3: Longstanding IGE, Treatment Resistant
- •Inclusion Criteria:
- •Age ≥13 years at time of enrollment
- •Age ≥8 years at time of seizure onset
- •Clinical seizure(s) and history consistent with IGE. The only permitted seizure types are absence, myoclonus or generalized tonic-clonic convulsions
- •Occurrence of at least 1 seizure of any type in the 6 months prior to treatment onset
- •Patients must have had one of the following:
- •GTCSs alone accompanied by generalized spike-wave consistent with IGE per adjudication review
- •GTCSs with a history of absence and/or myoclonus, accompanied by generalized spike-wave consistent with IGE per adjudication review
- •GTCSs associated with a history of absence and/or myoclonus, not accompanied by generalized spike-wave consistent with IGE per adjudication review
- •A clear history of absence and/or myoclonus, accompanied by generalized spike-wave consistent with IGE per adjudication review
- •Availability of a complete medication history since initiation of treatment. This should include doses and date of initiation if possible, but minimum information would include name of drug, approximate duration of administration and reason for discontinuation, if applicable.
- •No competing cause of epilepsy (e.g. traumatic brain injury)
- •Treatment resistant IGE
- 另有 16 项未显示
研究组 & 干预措施
Cohort 1: Newly Diagnosed Idiopathic Generalized Epilepsy (IGE)
Cohort 1 will have IGE that was diagnosed within the prior year. We will follow these participants for a minimum of two years.
Cohort 2: Longstanding Treatment Responsive
Cohort 2 will consist of subjects with established IGE who have been responsive to treatment.
Cohort 3: Longstanding IGE, Treatment Resistant
Cohort 3 will consist of patients with established treatment-resistant IGE.
结局指标
主要结局
Average heart rate
时间窗: Baseline
Participants will be provided a Fitbit Inspire HR watch and will be trained to open an account on fitbit.com and synchronize their Fitbit watch data into their Fitbit cloud account. Daily steps is synchronized and collected for this study.
Columbia Suicide Severity Rating Scale (C-SSRS) Score
时间窗: Month 24
C-SSRS systematically tracks suicidal ideation and behavior. The total score range is 0 (no ideation is present) - 5 (active suicidal ideation with specific plan and intent). The higher the score, the greater one's suicidal ideation. Any score greater than 0 is important and may indicate the need for mental health intervention
Cogstate Neuropsychological Assessment Score
时间窗: Month 24
The Cogstate is a battery of online computerized tests that assesses functions such as attention, memory, and processing speed. Scores are normalized to the range of 0-100th percentile based on speed of test completion and task accuracy. A higher percentile score indicates higher levels of cognitive functioning within the tested domain as compared to controls.
Average daily steps
时间窗: Baseline
Participants will be provided a Fitbit Inspire HR watch and will be trained to open an account on fitbit.com and synchronize their Fitbit watch data into their Fitbit cloud account. Daily steps is synchronized and collected for this study.
Change in Quality of Life in Epilepsy Inventory-10 (QOLIE-10) Score
时间窗: Month 12, Month 24
QOLIE-10 assesses the patient's quality of life. There are 11 total items. The total score range is 10-51; with higher scores indicating greater impairment. The total score is the sum of scores for all questions divided by the number of items answered. Thus, if a patient skipped an item, it is not reflected in the total score.
Number of episodes of non-adherence
时间窗: Baseline
Self-reported via the Seer Seizure Diary App (https://app.seermedical.com)
Average daily wake duration
时间窗: Baseline
Participants will be provided a Fitbit Inspire HR watch and will be trained to open an account on fitbit.com and synchronize their Fitbit watch data into their Fitbit cloud account. Daily steps is synchronized and collected for this study. Using the Embleema Patient Web App, participants will be able to consult their activity and sleep data in interactive graphs.
Change in General Anxiety Disorder-7 Screener (GAD-7) Score
时间窗: Month 12, Month 24
This will only be reported for adults. GAD-7 consists of 7 problems. Participants report how often they have been bothered by the 7 problems over the last 2 weeks on a Likert Scale from 0 (not at all) to 3 (nearly every day). The total score range is 0-21; the higher the score, the more severe the anxiety. 0-4=minimal anxiety, 5-9=mild, 10-14=moderate, 15-21=severe anxiety.
Number of Individual Seizures
时间窗: Baseline
Self-reported via the Seer Seizure Diary App (https://app.seermedical.com)
Number of Cluster Seizures
时间窗: Baseline
Self-reported via the Seer Seizure Diary App (https://app.seermedical.com)
Average distance walked
时间窗: Baseline
Participants will be provided a Fitbit Inspire HR watch and will be trained to open an account on fitbit.com and synchronize their Fitbit watch data into their Fitbit cloud account. Daily steps is synchronized and collected for this study.
Average daily sleep duration
时间窗: Baseline
Participants will be provided a Fitbit Inspire HR watch and will be trained to open an account on fitbit.com and synchronize their Fitbit watch data into their Fitbit cloud account. Daily steps is synchronized and collected for this study. Using the Embleema Patient Web App, participants will be able to consult their activity and sleep data in interactive graphs.
Wide Range Achievement Test (WRAT-4) Score
时间窗: Baseline
WRAT4 is an academic skills assessment which measures reading skills, math skills, spelling, and comprehension. Only the Word Reading portion of the assessment will be administered. The total raw score range is from 0-70, with a normalized score range of 55-145. The higher the score, the more advanced the participant's reading comprehension skills for their age range.
Change in Quality of Life in Epilepsy Inventory-10 (QOLIE-10) Score
时间窗: Baseline, Month 12
QOLIE-10 assesses the patient's quality of life. There are 11 total items. The total score range is 10-51; with higher scores indicating greater impairment. The total score is the sum of scores for all questions divided by the number of items answered. Thus, if a patient skipped an item, it is not reflected in the total score.
Change in Quality of Life in Epilepsy Inventory-10 (QOLIE-10) Score
时间窗: Baseline, Month 24
QOLIE-10 assesses the patient's quality of life. There are 11 total items. The total score range is 10-51; with higher scores indicating greater impairment. The total score is the sum of scores for all questions divided by the number of items answered. Thus, if a patient skipped an item, it is not reflected in the total score.
Change in General Anxiety Disorder-7 Screener (GAD-7) Score
时间窗: Baseline, Month 12
This will only be reported for adults. GAD-7 consists of 7 problems. Participants report how often they have been bothered by the 7 problems over the last 2 weeks on a Likert Scale from 0 (not at all) to 3 (nearly every day). The total score range is 0-21; the higher the score, the more severe the anxiety. 0-4=minimal anxiety, 5-9=mild, 10-14=moderate, 15-21=severe anxiety.
Change in General Anxiety Disorder-7 Screener (GAD-7) Score
时间窗: Baseline, Month 24
This will only be reported for adults. GAD-7 consists of 7 problems. Participants report how often they have been bothered by the 7 problems over the last 2 weeks on a Likert Scale from 0 (not at all) to 3 (nearly every day). The total score range is 0-21; the higher the score, the more severe the anxiety. 0-4=minimal anxiety, 5-9=mild, 10-14=moderate, 15-21=severe anxiety.
Columbia Suicide Severity Rating Scale (C-SSRS) Score
时间窗: Baseline
C-SSRS systematically tracks suicidal ideation and behavior. The total score range is 0 (no ideation is present) - 5 (active suicidal ideation with specific plan and intent). The higher the score, the greater one's suicidal ideation. Any score greater than 0 is important and may indicate the need for mental health intervention
Columbia Suicide Severity Rating Scale (C-SSRS) Score
时间窗: Month 12
C-SSRS systematically tracks suicidal ideation and behavior. The total score range is 0 (no ideation is present) - 5 (active suicidal ideation with specific plan and intent). The higher the score, the greater one's suicidal ideation. Any score greater than 0 is important and may indicate the need for mental health intervention
Cogstate Neuropsychological Assessment Score
时间窗: Baseline
The Cogstate is a battery of online computerized tests that assesses functions such as attention, memory, and processing speed. Scores are normalized to the range of 0-100th percentile based on speed of test completion and task accuracy. A higher percentile score indicates higher levels of cognitive functioning within the tested domain as compared to controls.
Cogstate Neuropsychological Assessment Score
时间窗: Month 12
The Cogstate is a battery of online computerized tests that assesses functions such as attention, memory, and processing speed. Scores are normalized to the range of 0-100th percentile based on speed of test completion and task accuracy. A higher percentile score indicates higher levels of cognitive functioning within the tested domain as compared to controls.
次要结局
未报告次要终点