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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05374928




Registration number
NCT05374928
Ethics application status
Date submitted
11/05/2022
Date registered
16/05/2022
Date last updated
15/08/2024

Titles & IDs
Public title
Human Epilepsy Project 3
Scientific title
Human Epilepsy Project 3: Newly Diagnosed Idiopathic Generalized Epilepsy
Secondary ID [1] 0 0
19-01030
Universal Trial Number (UTN)
Trial acronym
HEP3
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Idiopathic Generalized Epilepsy 0 0
Condition category
Condition code
Neurological 0 0 0 0
Epilepsy

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
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.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Individual Seizures
Timepoint [1] 0 0
Baseline
Primary outcome [2] 0 0
Number of Cluster Seizures
Timepoint [2] 0 0
Baseline
Primary outcome [3] 0 0
Number of episodes of non-adherence
Timepoint [3] 0 0
Baseline
Primary outcome [4] 0 0
Average daily steps
Timepoint [4] 0 0
Baseline
Primary outcome [5] 0 0
Average distance walked
Timepoint [5] 0 0
Baseline
Primary outcome [6] 0 0
Average heart rate
Timepoint [6] 0 0
Baseline
Primary outcome [7] 0 0
Average daily sleep duration
Timepoint [7] 0 0
Baseline
Primary outcome [8] 0 0
Average daily wake duration
Timepoint [8] 0 0
Baseline
Primary outcome [9] 0 0
Change in Quality of Life in Epilepsy Inventory-10 (QOLIE-10) Score
Timepoint [9] 0 0
Baseline, Month 12
Primary outcome [10] 0 0
Change in Quality of Life in Epilepsy Inventory-10 (QOLIE-10) Score
Timepoint [10] 0 0
Baseline, Month 24
Primary outcome [11] 0 0
Change in Quality of Life in Epilepsy Inventory-10 (QOLIE-10) Score
Timepoint [11] 0 0
Month 12, Month 24
Primary outcome [12] 0 0
Change in General Anxiety Disorder-7 Screener (GAD-7) Score
Timepoint [12] 0 0
Baseline, Month 12
Primary outcome [13] 0 0
Change in General Anxiety Disorder-7 Screener (GAD-7) Score
Timepoint [13] 0 0
Baseline, Month 24
Primary outcome [14] 0 0
Change in General Anxiety Disorder-7 Screener (GAD-7) Score
Timepoint [14] 0 0
Month 12, Month 24
Primary outcome [15] 0 0
Columbia Suicide Severity Rating Scale (C-SSRS) Score
Timepoint [15] 0 0
Baseline
Primary outcome [16] 0 0
Columbia Suicide Severity Rating Scale (C-SSRS) Score
Timepoint [16] 0 0
Month 12
Primary outcome [17] 0 0
Columbia Suicide Severity Rating Scale (C-SSRS) Score
Timepoint [17] 0 0
Month 24
Primary outcome [18] 0 0
Cogstate Neuropsychological Assessment Score
Timepoint [18] 0 0
Baseline
Primary outcome [19] 0 0
Cogstate Neuropsychological Assessment Score
Timepoint [19] 0 0
Month 12
Primary outcome [20] 0 0
Cogstate Neuropsychological Assessment Score
Timepoint [20] 0 0
Month 24
Primary outcome [21] 0 0
Wide Range Achievement Test (WRAT-4) Score
Timepoint [21] 0 0
Baseline

Eligibility
Key inclusion criteria
Cohort 1: Newly Diagnosed IGE

Inclusion criteria:

1. Age =13 years at time of enrollment
2. Age =8 years at time of seizure onset
3. Clinical seizure(s) and history consistent with IGE. The only permitted seizure types are absence, myoclonus or generalized tonic-clonic convulsions
4. Occurrence of at least 1 seizure of any type in the 6 months prior to treatment
5. 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
6. Availability of a complete medication history since initiation of treatment, including doses and date of initiation
7. No competing cause of epilepsy (e.g. traumatic brain injury)
8. AED treatment (for seizures) instituted not more than 12 months before enrollment
Minimum age
13 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion Criteria

1. Focal epilepsy
2. Generalized/focal epilepsy mixed syndromes
3. Progressive neurological disorder (brain tumor, Alzheimer's disease, progressive myoclonic epilepsy, etc.)
4. Epileptic or developmental encephalopathy
5. Major medical co-morbidities (e.g. renal failure requiring dialysis, metastatic cancer, HIV, or significant liver or renal disease)
6. Autism Spectrum Disorder
7. History of chronic drug or alcohol abuse (misuse or excessive use that interferes with activities of daily living) within the last 2 years
8. Seizures only during pregnancy
9. History of previous or current significant psychiatric disorder that would interfere with study requirements

Cohort 2: Longstanding Treatment Responsive

Inclusion Criteria:

1. Age =13 years at time of enrollment
2. Age =8 years at time of seizure onset
3. Clinical seizure(s) and history consistent with IGE. The only permitted seizure types are absence, myoclonus or generalized tonic-clonic convulsions
4. Patients must have had one of the following:

1. GTCSs alone accompanied by generalized spike-wave consistent with IGE per adjudication review
2. GTCSs with a history of absence and/or myoclonus, accompanied by generalized spike-wave consistent with IGE per adjudication review
3. GTCSs associated with a history of absence and/or myoclonus, not accompanied by generalized spike-wave consistent with IGE per adjudication review
4. A clear history of absence and/or myoclonus, accompanied by generalized spike-wave consistent with IGE per adjudication review
5. Availability of a complete medication history since initiation of treatment, including doses and date of initiation
6. No competing cause of epilepsy (e.g. traumatic brain injury)
7. Two years of well-controlled seizures.

1. No convulsive seizures in the last two years
2. Myoclonic or absence seizures must be rare (<2 per year) and non-disabling
3. Ongoing therapy with > 1 antiseizure medication



1. Focal epilepsy
2. Paroxysmal nonepileptic seizures
3. Generalized/focal epilepsy mixed syndromes
4. Progressive neurological disorder (brain tumor, Alzheimer's disease, progressive myoclonic epilepsy, etc.)
5. Epileptic or developmental encephalopathy
6. Major medical co-morbidities (e.g. renal failure requiring dialysis, metastatic cancer, HIV, or significant liver or renal disease)
7. Autism Spectrum Disorder
8. History of chronic drug or alcohol abuse (misuse or excessive use that interferes with activities of daily living) within the last 2 years
9. Seizures only during pregnancy
10. History of previous or current significant psychiatric disorder that would interfere with study requirements

Cohort 3: Longstanding IGE, Treatment Resistant

Inclusion Criteria:

1. Age =13 years at time of enrollment
2. Age =8 years at time of seizure onset
3. Clinical seizure(s) and history consistent with IGE. The only permitted seizure types are absence, myoclonus or generalized tonic-clonic convulsions
4. Occurrence of at least 1 seizure of any type in the 6 months prior to treatment onset
5. Patients must have had one of the following:

1. GTCSs alone accompanied by generalized spike-wave consistent with IGE per adjudication review
2. GTCSs with a history of absence and/or myoclonus, accompanied by generalized spike-wave consistent with IGE per adjudication review
3. GTCSs associated with a history of absence and/or myoclonus, not accompanied by generalized spike-wave consistent with IGE per adjudication review
4. A clear history of absence and/or myoclonus, accompanied by generalized spike-wave consistent with IGE per adjudication review
6. 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.
7. No competing cause of epilepsy (e.g. traumatic brain injury)
8. Treatment resistant IGE

1. Initiation of treatment at least 2 years prior to enrollment
2. Treatment resistance, as defined by failure of adequate trials of two tolerated and appropriately chosen and used AED schedules (whether as monotherapies or in combination to achieve seizure freedom). ASMs taken at enrollment would count towards this minimum.
3. Either or both of the following two:

i. One GTCC per year over the last 2 years. ii. Any type of seizure at least every 3 months which can consist of either: disabling myoclonus or absence (in the opinion of the subject and investigator) or GTCC d. Such seizures were not primarily due to significant illness (e.g, URI is not significant unless temperature >101oF (38.3oC), nonadherence to antiseizure medications or >2 alcoholic beverages within 48 hrs of seizure e. Ongoing therapy with > 1 antiseizure medication



1. Focal epilepsy
2. Paroxysmal nonepileptic seizures
3. Generalized/focal epilepsy mixed syndromes
4. Progressive neurological disorder (brain tumor, Alzheimer's disease, progressive myoclonic epilepsy, etc.)
5. Epileptic or developmental encephalopathy
6. Major medical co-morbidities (e.g. renal failure requiring dialysis, metastatic cancer, HIV, or significant liver or renal disease)
7. Autism Spectrum Disorder
8. History of chronic drug or alcohol abuse (misuse or excessive use that interferes with activities of daily living) within the last 2 years
9. Seizures only during pregnancy
10. History of previous or current significant psychiatric disorder that would interfere with study requirements
11. History of/suspicion of provoked seizures accounting for 25% or more of seizures over the prior 2 years (eg alcohol, non-adherence, significant sleep deprivation).

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
9/03/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/08/2025
Actual
Sample size
Target
Accrual to date
Final
320
Recruitment in Australia
Recruitment state(s)
Clayton VI
Recruitment hospital [1] 0 0
Monash University - Melbourne
Recruitment postcode(s) [1] 0 0
3800 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Connecticut
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Maine
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
Missouri
Country [9] 0 0
United States of America
State/province [9] 0 0
New Jersey
Country [10] 0 0
United States of America
State/province [10] 0 0
New York
Country [11] 0 0
United States of America
State/province [11] 0 0
Pennsylvania
Country [12] 0 0
United States of America
State/province [12] 0 0
Texas
Country [13] 0 0
United States of America
State/province [13] 0 0
Utah

Funding & Sponsors
Primary sponsor type
Other
Name
NYU Langone Health
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/study/NCT05374928
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Jacqueline French, MD
Address 0 0
Country 0 0
Phone 0 0
646-558-0839
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05374928