Clinical trial NCT03531008: Human Epilepsy Project 2: Resistant Focal Seizures Study
Official title: Biomarkers, Health Outcomes and Healthcare Utilization in People With Resistant Focal Epilepsy
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Biomarkers
Keywords: focal; partial
Lead sponsor: Epilepsy Foundation of America
Sponsor class: OTHER
Collaborators: name: The Epilepsy Study Consortium
name: UCB Biopharma S.P.R.L.
First posted: 2018-05-21
Enrollment: 157
Sex: ALL
Minimum age (years): 16
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population will include a cohort of 205 individuals with focal epilepsy who meet the ILAE definition of treatment resistance.

Brief summary: The HEP2 study is designed to better understand the challenges of living with focal seizures that do not respond to medication, by following 205 people with medication-resistant focal epilepsy over two years to measure changes in health status, healthcare costs, quality of life, and biomarkers of epilepsy severity and treatment response.

Detailed description: The Epilepsy Foundation is launching a partnership, called the Human Epilepsy Project, in collaboration with the Epilepsy Study Consortium. This study (which is called HEP2 for short) is designed to better understand the challenges of living with focal seizures that do not respond to medication. The HEP2 study will follow 200 people with medication-resistant focal epilepsy (with seizures that occur at least 2 times per month) over two years to measure changes in their seizure frequency, treatments used, adverse events experienced, presence of co-morbidities like depression and anxiety, healthcare costs, and quality of life. Blood samples will also be collected in order to look for biomarkers of epilepsy severity and treatment response.

Participants can join the HEP2 study at any one of nine recruiting study centers. These study centers were selected because they are epilepsy centers with track records of conducting high-quality research in epilepsy and efficiently recruiting participants into studies. The designated sites for the HEP2 study are located in New York, California, Minnesota, Connecticut, Pennsylvania, and Tennessee. A participant may enroll in the HEP2 study but continue to receive their standard epilepsy care with their current physician, as long as the participant is willing to share his or her medical records, and travel to the study center for two or three in person visits at the beginning of the study, after the first year, and a final visit after the second year.

Eligibility criteria: Inclusion Criteria:

1. Age ≥ 16 years and ≤ 65 years at time of enrollment
2. Diagnosed with focal epilepsy with prior vEEG confirmation; if no VEEG confirmation, but in the opinion of the investigator the seizures are definite, the case can be reviewed and adjudicated by the consortium
3. Failure of adequate trials of 4 AEDs, with at least 2 due to failure of seizure control, including current AEDs
4. Have a seizure frequency of ≥ 2 focal seizures/month, including auras, and at least 1 observable seizures/month, that are countable by the subject and/or caregiver for the 3 months prior to enrollment
5. Able to keep a daily seizure diary, either independently or with assistance from a caregiver
6. Able to retrospectively report number of seizures/month for 3 months prior to enrollment
7. Receiving ≥ 1 AED for treatment of seizures

Exclusion Criteria:

1. Patient has a diagnosis of idiopathic ("primary") generalized epilepsy (e.g., juvenile myoclonic epilepsy, absence epilepsy) or mixed focal and generalized (e.g., Lennox-Gastaut syndrome) or non-epileptic seizures within the last 12 months prior to study entry
2. Progressive medical or neurological disorder (brain tumor, AD, PME, etc.)
3. Proven autoimmune etiology
4. Planning pregnancy in the next 12 months
5. Has completed a pre-surgical evaluation and intends to pursue surgery in the near term
6. Resective surgery and/or RNS/VNS in place less than 12 months prior to enrollment
7. Presence of moderate or greater developmental or cognitive delay (e.g., if an adolescent, not in self-contained classroom; if IQ is documented, should be ≥ 70)
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. Medical, psychiatric or psychosocial condition that would be expected to interfere with the conduct of the study
10. Enrolled in any interventional study that required a blinded portion or involves a non-FDA approved drug or device

Primary outcomes:
measure: Seizure freedom rates, seizure frequency and degree of disability
timeFrame: 24 Months
Secondary outcomes:
measure: Medication changes
timeFrame: 24 months
measure: Healthcare utilization
timeFrame: 24 months
Other outcomes:
measure: Biomarkers
timeFrame: 24 months
Locations:
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: New Haven
country: United States
facility: Yale Comprehensive Epilepsy Center
state: Connecticut
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Boise
country: United States
facility: Idaho Comprehensive Epilepsy Center
state: Idaho
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Saint Paul
country: United States
facility: Minnesota Epilepsy Group
state: Minnesota
city: New York
country: United States
facility: New York University
state: New York
city: New York
country: United States
facility: Northwell Health Comprehensive Epilepsy Center
state: New York
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt Epilepsy Center
state: Tennessee
Study officials:
affiliation: Epilepsy Foundation
name: Brandy Fureman, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: New York University
name: Jacqueline French, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Northwell Health
name: Ruben Kuzniecky, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of California, San Francisco
name: Daniel Lowenstein, MD
role: PRINCIPAL_INVESTIGATOR
References: