Why Clinical Trials Matter

If your child has a rare condition like KCNT1-related epilepsy, you may hear about clinical trials as a possible option. Clinical trials are how new treatments are tested to see if they are safe and if they actually help. Without families like yours to participate in the trials, no new medications could ever be approved.

By joining a trial, you are not only trying something new for your child—you are contributing to research that could change the future for others like them. It’s a deeply personal choice, and this guide is here to help you understand all aspects of that decision.

Download our handout.

Video Title	Speakers	Summary	Watch / Download
What Are Clinical Trials	Gitesh Dubal, CEO & Founder, Nova Spark Ventures	What are clinical trials, and why do they matter for rare diseases like KCNT1 epilepsy? 13 min	Watch Video
Small Molecules & ASOs	Dr David Bearden, 2022	How small molecules and ASOs can target KCNT1.	Watch Video / Slides
Gene Therapy Basics	Emma Jones – VP Clinical and Medical Affairs, Encoded Therapeutics	Explains how gene therapy works and different delivery methods.	Watch Video (19 min)/ Slides
From Lab to Patient: Preclinical & Clinical Research Explained	Dianne Mitchell – COO, CAMK2 Foundation & Scientific Director, Global Dare	What happens before a drug gets tested in people.	Watch Video
Current and future treatments for KCNT1	Mark Fitzgerald, MD – Children’s Hospital of Philadelphia	There are no antiseizure medications focused on potassium channels today.	Watch Video / Slides
Logistical and Financial Considerations of Trials	Juliana Mills, Worldwide Clinical Trials, Sophia Cacciatore, Lundbeck Pharmaceuticals, Kim Hindery, Child Neurology Foundation	A panel discussion discusses the financial and logistical considerations of participating in a clinical trial.	Watch Video
What Are Clinical Trials	Derek Ansel, Worldwide Clinical Trials	This overview explains how participants are identified, eligibility is determined, and what to expect during participation, while addressing common concerns.	Watch Video
Expectations and Goals of Trials	Seth Rotberg, Heather Bentley, and Dr. Amy Raymond	The panel explores what families can expect when joining a clinical trial—how studies are designed, what participation involves, and why every family’s data matters.	Watch Video
Deciding to Participate in Clinical Trials	U.S. Department of Health and Human Services	This video discusses types of human research with a focus on clinical trials, and explains common terms that potential participants should know.	Watch Video

Next Steps

Ready to Explore Active Studies?

Once you feel confident about the clinical trial process, visit the Trials & Studies Portal to see KCNT1-specific opportunities and broader epilepsy/DEE studies.

Glossary of Clinical Research Terms

Term	Definition
Placebo	A pill, liquid, or procedure that looks like the treatment being tested but has no active ingredient. Placebos help researchers see if the investigational treatment works better than no treatment.
Randomized Controlled Trial (RCT)	A study where participants are randomly assigned (like flipping a coin) to different groups, such as the treatment group or the placebo/standard care group, to prevent bias.
Open-Label Trial	A study where both the researchers and participants know which treatment is being given.
Double-Blind Trial	A study where neither participants nor researchers know who is receiving the investigational treatment versus the placebo/standard care until the trial ends, to avoid bias.
Crossover Trial	A study where participants receive more than one treatment during the trial, switching (“crossing over”) from placebo to investigational treatment (or vice versa) after a set period.
Biomarker	A measurable sign of a disease or its progression, such as EEG patterns, blood test results, or levels of certain molecules in the body. Biomarkers can also indicate if a treatment is working.
Informed Consent	The process of learning about a study before joining. Families receive a document that explains the purpose, procedures, risks, benefits, and rights as a participant.
Inclusion & Exclusion Criteria	The rules about who can or cannot join a study, usually based on age, diagnosis, health conditions, or medications.