Are you measuring what matters in pediatric epilepsy trials?
Many clinical trials in pediatric epilepsies quantify seizures as an endpoint. However, change, in some patients, is more subtle than can be measured by a seizure diary. This webinar will bring together seasoned experts in clinical trial operations and strategy, alongside patient advocacy and public health professionals, to delve into the evolving landscape of clinical trial endpoints for pediatric epilepsies.
Traditional clinical trials for pediatric epilepsy often rely on seizure frequency as the primary endpoint. However, this approach does not always capture the full spectrum of patient experiences, especially when changes are subtler than what seizure diaries can document. This webinar is set to address this pivotal issue, focusing on the need for more comprehensive and patient-centered endpoints.
Participants will gain valuable insights into the current trends in endpoint development, particularly for developmental and epileptic encephalopathies (DEE). These insights will not only reflect the latest scientific advancements but also incorporate the nuanced perspectives of those living with these conditions. The key learning points for the audience will include:
- Trends in endpoints for DEE
- Broadening the scope of clinical trial endpoints: Seizure diaries are commonly used as endpoints in pediatric epilepsy trials but these measures may not capture subtle and meaningful changes in some patients, and this webinar explores trends in developing more nuanced and patient-centric endpoints
- Transforming challenges into actionable research: The webinar will emphasize how families, advocacy groups and researchers can collaborate to identify measurable, impactful outcomes that reflect the lived experiences of children with rare epilepsies
This webinar can push the boundaries of research to accommodate the diverse and real-world needs of young patients and their families.
Join this webinar that promises to be as informative as it is impactful, providing attendees with the knowledge and strategies to enhance the effectiveness and sensitivity of clinical trials in pediatric epilepsy.
Speakers
Juliane Mills, MPH, Senior Director Therapeutic Strategy, Worldwide Clinical Trials
Juliane Mills is a seasoned scientist in the world of clinical research with over 20 years of experience. Juliane’s mission is simple but powerful: to make a real difference for people battling rare diseases. At Worldwide, she is the go-to person for creating smart, streamlined plans that help bring new treatments to those in need, without forgetting the real people behind the diagnoses – the patients, their loved ones and their caregivers.
Gabi Conecker, MPH, Executive Director & Co-Director, Decoding Developmental Epilepsies
Gabi Conecker is Executive Director and Co-founder of Decoding Developmental Epilepsies (DDE) – home to the International SCN8A Alliance, DEE-P Connections and The Inchstone Project. Gabi has a nearly 20 years of experience in advocating for and advancing public health. Gabi earned her Master of Public Health (MPH) from Columbia University’s Mailman School of Public Health. Gabi is mom to 12-year-old Elliott, who is profoundly impacted by SCN8A Developmental and Epileptic Encephalopathy. She founded DDE to bring the community together to fight for better care, treatments and outcomes for those profoundly impacted by rare epilepsy disorders.
JayEtta Hecker, MS, Executive Director & Co-Director, Decoding Developmental Epilepsies
JayEtta Hecker is the Board Chair and Co-founder of the International SCN8A Alliance, DEE-P Connections and The Inchstone Project. JayEtta is an economist who specialized in independent public policy analyses and held several positions with the National Academy of Sciences.
JayEtta trained and served in multiple federal agencies, including 25 years with the congressional research arm, and the government accountability office. She is a formidable advocate in the fight against SCN8A and developmental and epileptic encephalopathies.
JayEtta partnered with Gabi, her daughter, to form Wishes for Elliott to advance SCN8A research and create a lasting legacy for Elliott, who continues to serve as an inspiration with his pure love, connection, resilience and joy.
Jessica Duis, MD, MS, Senior Medical Director in Clinical Development, Neurocrine Biosciences
Jessica Duis is Senior Medical Director in Clinical Development for the Neurology pipeline at Neurocrine Biosciences. She is a geneticist and pediatrician and did her medical training at Johns Hopkins. In academia, she developed several Centers of Excellence including translational, clinical research and drug development. She has been working in the field of biologics, in particular gene therapy for about 10 years, and ran several investigator driven clinical trials before coming to industry.
She is passionate about patient advocacy, early patient advocacy organization engagement, and endpoint selection to capture sensitive predictors of efficacy in the natural environment. She has been involved in endpoint validation and discussions with regulators around clinical endpoints for severe neurogenetic conditions and developmental epileptic encephalopathies. Her work on digital health technologies have led to regulatory qualification. She has worked on several gene therapies that are now approved or progressing through the accelerated approval pathway.
Who Should Attend?
This webinar will benefit Vice Presidents, Directors, Managers, Department Heads, Scientists and Researchers working within:
- Clinical Affairs
- Clinical R&D
- Clinical Research
- Clinical Pharmacology
- Clinical Operations
- Project Management
- Regulatory Affairs
- Medical Affairs
What You Will Learn
Attendees will learn about:
- Trends in endpoints for developmental and epileptic encephalopathies (DEE)
- Broadening the scope of clinical trial endpoints
- Transforming challenges into actionable research
Xtalks Partner
Worldwide Clinical Trials
Worldwide Clinical Trials (Worldwide) is a leading global contract research organization (CRO) that works in partnership with biotechnology and pharmaceutical companies to create customized solutions that advance new medications, from discovery to reality. Our full-service clinical development capabilities include bioanalytical laboratory services and Phase I-IV clinical trials, as well as post-approval and real-world evidence studies – all powered by our talented and accessible team of clinicians, scientists, and researchers who bring their first-hand expertise to each clinical program.
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Our team of 3,000+ professionals spans 60+ countries, and believes that through collaboration, and a culture that embraces diversity, equity, inclusion, and belonging (DEI&B), everyone plays an important role in making a world of difference for patients and their caregivers. We are united in cause with our customers to improve the lives of patients through new, innovative therapies.
For more information, please visit www.worldwide.com or connect with us on LinkedIn.
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