Journal
NEUROTHERAPEUTICS
Volume 11, Issue 2, Pages 401-411Publisher
SPRINGER
DOI: 10.1007/s13311-013-0252-z
Keywords
Anti-epileptogenic drugs; Disease-reversal; Disease-modification; Antiepileptogenic drug trial design; Disease-modifying drug trial design; Disease-reversing drug trial design; Feasibility of trial design; Human biomarkers; Novel epilepsy trial design; Epilepsy
Funding
- NIH from the National Center for the Advancement of Translational Science (NCATS), American Epilepsy Society, and faces [UL1 TR000038]
- Epilepsy Study Consortium
- Eisai Medical Research
- GlaxoSmithKline
- Impax
- Johnson Johnson
- Mapp Pharmaceuticals
- Novartis
- Lundbeck
- Pfizer
- Sepracor
- Sunovion
- SK Life Science
- Supernus Pharmaceuticals
- UCB Inc/Schwarz Pharma
- Upsher Smith
- Vertex
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Although trials with anti-seizure drugs have not shown anti-epileptogenic or disease-modifying activity in humans, new compounds are on the horizon that may require novel trial designs. We briefly discuss the unique challenges and the available options to identify innovative clinical trial designs that differentiate novel anti-epileptogenic and disease-modifying compounds, preferably early in phase II, from current anti-seizure drugs. The most important challenges of clinical testing of agents for epilepsy prevention include having sufficient preclinical evidence for a suitable agent to proceed with a human trial of an anti-epileptogenic drug, and to demonstrate the feasibility of doing such a trial. Major challenges in trial design to assess agents for disease modification include the choice of suitable study parameters, the identification of a high-risk study population, the type of control, the time and duration of treatment, and a feasible follow-up period.
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