Clinical Trials & Emerging Treatments
Research is the most critical component for advancing treatments and getting closer to cures and is not just the responsibility of scientists and doctors. It is important that patients participate to whatever extent they are able, including: 1) sharing their information so researchers can understand how various forms of SCN8A affect individuals such as through our International SCN8A Registry; and 2) participating in clinical studies or trials to help us understand how treatments affect patients.
In rare diseases such as SCN8A, it can be difficult for potential treatments to recruit the required number of participants to be able to test the treatment and hopefully be granted FDA approval. Since we are not a large community, one of the most valuable resources we do have, which includes our strong patient community and our ability to contribute to the understanding of this disorder through participation in research.
Clinical research trials aim to answer questions such as:
- Does this investigational drug work?
- Does it work better than another medicine already available?
- Does it cause any side effects?
- Are there any other benefits that could improve patient quality of life?
Thank you for considering getting involved in research. If you have any questions, please reach out to us at [email protected]
Below are updates on clinical trials that SCN8A families may be eligible to participate in.
Praxis Precision Medicine
Praxis currently has a trial underway to investigate the safety and efficacy of Relutrigine (PRAX-562), a medication designed to help regulate sodium flow into the brain more precisely. The hope is that this drug will help regulate the flow of sodium as it is meant to and lead to reduced seizures. The study has been designed to allow participants the option of a fully remote trial from home, attend clinic visits at a study site or a combination of both.
Neurocrine Biosciences
Neurocrine has closed the recent Kayak Study (“352”) which was evaluating the efficacy, safety, and tolerability of an investigational study drug compared to a placebo (inactive drug) as add-on therapy in young people 2 to 21 years of age with SCN8A developmental and epileptic encephalopathy (SCN8A-DEE).
As per the video and letter below from Neurocrine that has been shared with the community, there was not sufficient efficacy to continue the trial but they plan to open a new clinical trial in the early part of 2025 to test a medication they believe can help those with SCN8A-DEE and other related epilepsy disorders – “355”.
Longboard Pharmaceuticals
Recruitment Closed – The PACIFIC study
The PACIFIC Study is a clinical trial evaluating participants with DEE. The primary objectives of the study are to assess the safety & tolerability of an investigational study drug. The PACIFIC study is also designed to examine change in seizure frequency over the 90-day treatment period. The study plans to enroll approximately 50 participants with a variety of treatment resistant seizures that fall into the category of DEEs. Approximately 35 study sites in the United States and Australia will participate.
How Do Clinical Trials Work?
