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

• ClinicalTrials.gov Identifier: NCT03531008
• Recruitment Status: Active, not recruiting
• First Posted: May 21, 2018
• Last Update Posted: February 3, 2021
• Sponsor: Epilepsy Foundation of America
• Collaborators: The Epilepsy Study Consortium; UCB Biopharma S.P.R.L.
• Information provided by (Responsible Party): Epilepsy Foundation of America

Study Description

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.

Condition or disease
Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; 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 Design

• Study Type: Observational [Patient Registry]
• Estimated Enrollment: 205 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Target Follow-Up Duration: 2 Years
• Official Title: Biomarkers, Health Outcomes and Healthcare Utilization in People With Resistant Focal Epilepsy
• Actual Study Start Date: May 7, 2018
• Estimated Primary Completion Date: July 2022
• Estimated Study Completion Date: September 2022

Groups and Cohorts

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

Outcome Measures

Primary Outcome Measures :

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

Secondary Outcome Measures :

1. Medication changes [ Time Frame: 24 months ]
   To prospectively quantify medication changes over a 24 month period in a cohort of adults with treatment resistant focal epilepsy.
2. Healthcare utilization [ Time Frame: 24 months ]
   To prospectively quantify healthcare utilization

Other Outcome Measures:

1. Biomarkers [ Time Frame: 24 months ]
   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.

Biospecimen Retention:   Samples With DNA

Blood samples will be collected at the first visit and the final visit. A third sample may be collected from those participants who experience a seizure-free period of three months or more during the study period. All blood samples will be retained indefinitely, for use in future research. The specific details of future studies are unknown at this time, but will include genomic, proteomic, metabolomic and transcriptome analyses.

Eligibility Criteria

• Ages Eligible for Study: 16 Years to 65 Years (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• 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.

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

Contacts and Locations

Locations

United States, California

University of California San Francisco

San Francisco, California, United States, 94143

United States, Connecticut

Yale Comprehensive Epilepsy Center

New Haven, Connecticut, United States, 06520

United States, Florida

University of Miami

Miami, Florida, United States, 33136

United States, Idaho

Idaho Comprehensive Epilepsy Center

Boise, Idaho, United States, 83702

United States, Minnesota

Mayo Clinic

Rochester, Minnesota, United States, 55905

Minnesota Epilepsy Group

Saint Paul, Minnesota, United States, 55102

United States, New York

New York University

New York, New York, United States, 10016

Northwell Health Comprehensive Epilepsy Center

New York, New York, United States, 10075

United States, Pennsylvania

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States, 19104

United States, Tennessee

Vanderbilt Epilepsy Center

Nashville, Tennessee, United States, 37232

Sponsors and Collaborators

Epilepsy Foundation of America

The Epilepsy Study Consortium

UCB Biopharma S.P.R.L.

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

More Information

Additional Information:

• Responsible Party: Epilepsy Foundation of America
• ClinicalTrials.gov Identifier: NCT03531008
• Other Study ID Numbers: HEP2/TM0020
• First Posted: May 21, 2018
• Last Update Posted: February 3, 2021
• Last Verified: February 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• 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.
• Supporting Materials: Study Protocol; Informed Consent Form (ICF)
• Time Frame: The data will become available for request one year after the database is locked, and will remain available indefinitely.
• 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.
• URL: http://www.humanepilepsyproject.org

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Epilepsy Foundation of America:

focal; partial

Additional relevant MeSH terms:

Epilepsy; Nervous System Diseases; Brain Diseases; Central Nervous System Diseases