Clinical Trials in LGS
What is a Clinical Trial?
Clinical trials are research studies performed in those with a medical condition like LGS. They scientifically evaluate a medical or surgical intervention. They are the primary way that researchers find out if a new treatment, like a new drug, diet, or medical device is safe and effective in reducing seizures in those with LGS.
Without volunteers, clinical trials cannot exist and we would not be able to find new treatments for LGS and seizures. Families who participate in clinical trials are on the cutting edge of helping us to find better treatments and cures. The reason we have treatments for LGS today is due to the fact that LGS families participated in past trials. We are forever grateful for their contribution.
What are the Phases of a Clinical Trial?
Research is our best hope for advancing treatments and eventually finding a cure for LGS and associated epilepsies. Research is not just the responsibility of scientists and doctors. It is important that patients participate to whatever extent they are able.
Patients can participate in research in several ways, including 1) sharing their information with scientists through patient registries so researchers can understand how LGS affects individuals; and 2) volunteering for clinical studies or trials to help doctors understand how treatments affect patients.
Clinical studies seek to answer questions such as:
- Does this investigational drug or treatment work?
- Does it work better than another treatment already available?
- Does it cause any side effects?
- Are there any other benefits that could improve patient quality of life?
Since 2011, thanks to community engagement and study participation, 7 new medications have received approval from the FDA for the treatment of LGS.
Should I participate in a Clinical trial?
Learn more here on things to consider before you participate in a trial
What Treatments are currently in clinical trials for LGS?
RNS System Clinical Trial
This study examines the safety and effectiveness of Responsive Neurostimulation (RNS) for reducing seizures in individuals 12 years of age or older with LGS.
SKYWAY Phase 3 Soticlestat (TAK-935) Study
This anti-seizure medicine slows down the activation of the enzyme cholesterol 24-hydroxylase (CH24H). It has the potential to reduce seizure susceptibility and improve seizure control. It is currently in phase 3 studies for those with LGS.
The PACIFIC Study
The PACIFIC Study is a Phase 1b/2a clinical study for adult participants with DEEs. This study will assess the safety & tolerability of LP352. Importantly, the PACIFIC study is also designed to examine changes in seizure frequency over the treatment period.
STARS Phase 3 Staccato Alprazolam Study
The STARS Phase 3 Staccato Alprazolam Study is designed for people who experience prolonged epileptic seizures lasting more than 3 minutes. It tests an inhaler containing an investigational drug to see whether it could help people who experience prolonged epileptic seizures.
Stellina Study for VALTOCO® (diazepam nasal spray) in 2 to 5-Year-Old Children With Epilepsy
The Stellina Study is a Phase 1/2a clinical study evaluating a single dose of diazepam nasal spray (VALTOCO) as a rescue medication in children aged 2-5 who have been diagnosed with epilepsy. The objectives of the study are to assess the pharmacokinetics (PK) of diazepam after one intranasal dose of VALTOCO as well as assess the long-term safety and tolerability in the six-month open-label safety period.
Learn More about Clinical Trials
For more information about clinical trials, visit NIH, ClinicalTrials.gov.