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
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 Dravet syndrome. We have added links to each website for more information. Additional resource information on clinical trials can be found at http://clinicaltrials.gov/info/resources.