LGS Treatment Pipeline & Clinical Trials

What is a Clinical Trial?

Clinical Trial Phases

  • Phase I: Researchers test a new drug or treatment in a small group of healthy volunteers and/or people with the disease for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. Purpose: Safety and Dosage
  • Phase II: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety. Purpose: Efficacy and Side Effects
  • Phase III: 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. Purpose: Efficacy and Adverse Reactions
  • FDA Approval
  • Phase IV: Studies are done after the drug or treatment has been marketed to gather information on the drug’s effect in various populations and any side effects associated with long-term use. Purpose: Safety and Efficacy

The following companies have indicated that they are working on a medication or treatment that might be effective for patients living with LGS.

FDA-Approved for Use in LGS:

  • Epidiolex (CBD) Approved 2018
  • Sympazan (Clobazam Oral Film) Approved 2018
  • Topamax (Topiramate) Approved 2012
  • Onfi (Clobazam) Approved 2011
  • Felbatol (Felbamate) Approved 2008
  • Banzel (Rufinamide) Approved 2008
  • Lamictal (Lamotrigine) Approved 1998

FDA-Approved for Use in Acute Repetitive Seizures/Seizure Clusters:

  • Valtoco (Nasal Diazepam) Approved 2020
  • Nayzilam (Nasal Midazolam) Approved 2019
  • Diastat (Rectal Diazepam) Approved 1997

Treatments in Clinical Trials for LGS:

  • Zogenix – Fintepla – FDA-Approved for Dravet Syndrome. Pending Approval for LGS.
  • Eisai – Perampanel – Phase 3
  • Takeda – Soticlestat – Phase 2 Complete
  • SK Life Sciences – Carisbamate – Phase 1

Find Clinical Trials

   For more information about clinical trials, visit NIH, ClinicalTrials.gov.