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 is 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?
- Phase 1 Studies:
- In phase 1 of a clinical trial, researchers test a new drug or treatment in a small group of healthy volunteers and/or people with the disease for the first time and they evaluate its safety, determine a safe dosage range, and identify side effects
- Phase 2 Studies:
- In phase 2, a drug or treatment is given to a larger group of people to see if it is effective for seizures and to further evaluate its safety
- Phase 3 Studies:
- In this phase, the drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely. More than one Phase III study may be required before a New Drug Application (NDA) may be submitted to the FDA.
- FDA Approval:
- Once the FDA accepts a filing for the approval of a new treatment, the FDA must complete its review process within 10 months in most cases. The date at the end of the review period is referred to as the Prescription Drug User Fee Act (PDUFA) date.
- Phase 4 Studies:
- In this phase, studies are done after the drug or treatment has been FDA-approved and marketed to families. The goal is to gather information on the drug’s effect in various populations and identify any side effects associated with long-term use.
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. In rare diseases such as LGS, it can be difficult for potential treatments to reach the required number of participants to be granted FDA approval. In the absence of large numbers, we must leverage the resources we do have, which include our strong patient community.
Clinical studies seek 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?
Since 2011, thanks to community engagement and study participation, 4 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?
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 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 is testing an inhaler containing an investigational drug to see whether it could help people who experience prolonged epileptic seizures.
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 assessing 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.
Updated May 25, 2022