Human Epilepsy Project 2: Resistant Focal Seizures Study

NCT03531008 | Completed

• 1 other identifier: HEP2/TM0020
• Study Type: observational
• Target: 157
• Locations: 1 country
• Sites: 10

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.

Conditions

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

Keywords

focal; partial

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Medication changes

  24 months

• Healthcare utilization

  24 months

Other Outcomes (1)

• Biomarkers

  24 months

Study Arms (1)

Treatment-resistant focal epilepsy

Individuals with treatment-resistant focal epilepsy

Eligibility Criteria

• Age: 16 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• Age ≥ 16 years and ≤ 65 years at time of enrollment
• 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
• Failure of adequate trials of 4 AEDs, with at least 2 due to failure of seizure control, including current AEDs
• 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
• Able to keep a daily seizure diary, either independently or with assistance from a caregiver
• Able to retrospectively report number of seizures/month for 3 months prior to enrollment
• Receiving ≥ 1 AED for treatment of seizures

You may not qualify if:

• 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
• Progressive medical or neurological disorder (brain tumor, AD, PME, etc.)
• Proven autoimmune etiology
• Planning pregnancy in the next 12 months
• Has completed a pre-surgical evaluation and intends to pursue surgery in the near term
• Resective surgery and/or RNS/VNS in place less than 12 months prior to enrollment
• 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)
• History of chronic drug or alcohol abuse (misuse or excessive use that interferes with activities of daily living) within the last 2 years
• Medical, psychiatric or psychosocial condition that would be expected to interfere with the conduct of the study
• Enrolled in any interventional study that required a blinded portion or involves a non-FDA approved drug or device

Sponsors & Collaborators

• Epilepsy Foundation of America: lead
• The Epilepsy Study Consortium: collaborator
• UCB Biopharma S.P.R.L.: collaborator

Study Sites (10)

University of California San Francisco

San Francisco, California, 94143, United States

Location

Yale Comprehensive Epilepsy Center

New Haven, Connecticut, 06520, United States

Location

University of Miami

Miami, Florida, 33136, United States

Location

Idaho Comprehensive Epilepsy Center

Boise, Idaho, 83702, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Minnesota Epilepsy Group

Saint Paul, Minnesota, 55102, United States

Location

New York University

New York, New York, 10016, United States

Location

Northwell Health Comprehensive Epilepsy Center

New York, New York, 10075, United States

Location

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Vanderbilt Epilepsy Center

Nashville, Tennessee, 37232, United States

Location

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.

MeSH Terms

Conditions

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

Study Officials

• 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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 2 Years
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 25, 2018
• First Posted: May 21, 2018
• Study Start: May 7, 2018
• Primary Completion: July 30, 2022
• Study Completion: September 30, 2022
• Last Updated: September 18, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, 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.

Locations

US (10)