Human Epilepsy Project 2: Resistant Focal Seizures Study (HEP2)

Biomarkers, Health Outcomes and Healthcare Utilization in People With Resistant Focal Epilepsy

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.

Study Overview

• Status: Completed
• Conditions: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epilepsy; Biomarkers

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.

• Study Type: Observational
• Enrollment (Actual): 157

Contacts and Locations

Study Locations

• United States
  • California
    • San Francisco, California, United States, 94143
      • University of California San Francisco
  • Connecticut
    • New Haven, Connecticut, United States, 06520
      • Yale Comprehensive Epilepsy Center
  • Florida
    • Miami, Florida, United States, 33136
      • University of Miami
  • Idaho
    • Boise, Idaho, United States, 83702
      • Idaho Comprehensive Epilepsy Center
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic
    • Saint Paul, Minnesota, United States, 55102
      • Minnesota Epilepsy Group
  • New York
    • New York, New York, United States, 10016
      • New York University
    • New York, New York, United States, 10075
      • Northwell Health Comprehensive Epilepsy Center
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Hospital of the University of Pennsylvania
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Vanderbilt Epilepsy Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 65 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• 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.

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Treatment-resistant focal epilepsy; Individuals with treatment-resistant focal epilepsy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Seizure freedom rates, seizure frequency and degree of disability	To prospectively quantify seizure frequency over a 24 month period in a cohort of adults with treatment resistant focal epilepsy.	24 Months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Medication changes	To prospectively quantify medication changes over a 24 month period in a cohort of adults with treatment resistant focal epilepsy.	24 months
Healthcare utilization	To prospectively quantify healthcare utilization	24 months

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Biomarkers	Although we are not doing these analyses as part of this study, we plan future studies that would include genomics, proteomics, and metabolomics analyses on these samples. We will also make the sample available to other approved researchers upon request and review.	24 months

Collaborators and Investigators

• Sponsor: Epilepsy Foundation of America
• Collaborators: UCB Biopharma S.P.R.L.; The Epilepsy Study Consortium
• Investigators: Principal Investigator: Brandy Fureman, PhD, Epilepsy Foundation; Principal Investigator: Jacqueline French, MD, New York University; Principal Investigator: Ruben Kuzniecky, MD, Northwell Health; Principal Investigator: Daniel Lowenstein, MD, University of California, San Francisco

Study record dates

Study Major Dates

• Study Start (Actual): May 7, 2018
• Primary Completion (Actual): July 30, 2022
• Study Completion (Actual): September 30, 2022

Study Registration Dates

• First Submitted: April 25, 2018
• First Submitted That Met QC Criteria: May 8, 2018
• First Posted (Actual): May 21, 2018

Study Record Updates

• Last Update Posted (Actual): September 18, 2023
• Last Update Submitted That Met QC Criteria: September 15, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Keywords: focal; partial
• Additional Relevant MeSH Terms: Epilepsy; Nervous System Diseases; Brain Diseases; Central Nervous System Diseases
• Other Study ID Numbers: HEP2/TM0020

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: No protected health information is stored in the HEP2 database, and participants are tracked using their unique Participant Identifier only. All requests for data and specimen utilization (via collaboration with HEP2 investigators) will be submitted using a standardized form that will be available on the HEP2 website, and will be modeled on those used in the Gene Discovery in Epilepsy project (epi4k.org). These requests will be reviewed by the HEP2 PIs, and agreement to pursue collaborations and sharing of specimens will be based on: 1) strong scientific justification for the proposed collaborative project; 2) attestation by collaborators that all safeguards related to patient confidentiality and distribution of specimens will be upheld; and 3) track record of collaborators.
• IPD Sharing Time Frame: The data will become available for request one year after the database is locked, and will remain available indefinitely.
• IPD Sharing Access Criteria: All requests for data and specimen utilization (via collaboration with HEP2 investigators) will be submitted using a standardized form that will be available on the HEP2 website, and will be modeled on those used in the Gene Discovery in Epilepsy project (epi4k.org). These requests will be reviewed by the HEP2 PIs, and agreement to pursue collaborations and sharing of specimens will be based on: 1) strong scientific justification for the proposed collaborative project; 2) attestation by collaborators that all safeguards related to patient confidentiality and distribution of specimens will be upheld; and 3) track record of collaborators.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; ICF

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No