Current Clinical Trials &
Treatment Pipeline
Learn about opportunities to participate in research studies and what treatments are being developed for Dravet syndrome
Participate in Research
Currently Enrolling Clinical Studies in Dravet Syndrome
Research is our best hope for advancing treatments and eventually finding a cure for Dravet syndrome 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, including: 1) sharing their information so researchers can understand how Dravet syndrome affects individuals; and 2) volunteering for clinical studies or trials to help doctors understand how treatments affect patients. In rare diseases such as Dravet syndrome, 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 2018, thanks to community engagement and study participation, three new medications have received approval from the FDA for the treatment of Dravet syndrome, and there are many more in the development pipeline.
Looking for an easy way to contribute to Dravet syndrome research?
Consider taking one of the currently open research surveys at this page.
A pilot study of artificial intelligence/machine learning behavioral assessment in Dravet syndrome
Researchers are recruiting submissions of videos of children with DS between the ages of 2-12 years going about normal activities (such as playing or eating) to see if their AI/ML approach can distinguish information about medication responses, side effects, and disease symptoms.
If you are interested in contributing a video of your child to help teach the MoSeq program or if you have any questions, please contact: moseq-study@umich.edu.
Brief Description: A major challenge in the management of Dravet syndrome (DS) is determining whether or not a specific antiseizure medication will work for an individual patient. It can take many months before knowing if the medication will work and for some individuals it takes years (if ever) to find a medication that can adequately control their seizures.
Artificial intelligence (AI) now offers the potential to remedy this problem. Dr. Ivan Soltesz’s laboratory at Stanford has developed an AI/machine learning method they call Motion Sequencing (MoSeq). They used their MoSeq program to analyze videos of mice both with and without epilepsy. MoSeq was able to learn to distinguish the two types of mice apart just based upon differences in their movements and behaviors, and in the mice with epilepsy, MoSeq was able to determine the effects of an antiseizure medication without waiting for a seizure to occur.
Drs. Jack Parent and Ivan Soltesz now seek to apply this AI approach to children with DS. In order to “teach” the MoSeq program, we will need to have it analyze videos of children (2-12 years old) with DS going about their normal activities (e.g., playing or eating). Ideal videos for analysis should include primarily only the child with DS, have good lighting, and minimal background objects or patterns (ideally a solid color wall as background).
Once they are able to “teach” the MoSeq program, they hope to do a larger scale study in the future to determine whether or not MoSeq can predict whether or not a child with DS will respond to an antiseizure medication based on how the medication affects their movements and behavior.
If you are interested in contributing a video of your child to help teach the MoSeq program or if you have any questions, please contact: moseq-study@umich.edu.
This work is based upon the following published mouse epilepsy model study by the laboratory of Dr. Ivan Soltesz:
Gschwind et al, Hidden behavioral fingerprints in epilepsy. Neuron. 2023 May 3;111(9):1440-1452.e5. doi: 10.1016/j.neuron.2023.02.003. Epub 2023 Feb 24.
The Dravet Genome Study is a health research project launched by Children’s Hospital of Philadelphia (CHOP) and funded by DSF. The Dravet Genome Study, funded by DSF, will explore the diverse spectrum of outcomes that occur within Dravet syndrome and other SCN1A-related epilepsies using genetic analyses and medical histories. A very frequent topic of discussion in the community is how patients may differ in response to medications, severity of symptoms, and long-term outcomes despite having similar or even identical mutations in the SCN1A gene. This project aims to shed more light on other genetic factors that may impact this spectrum of outcomes. Learn more and sign up here.
Download flyer in English
Descargar este folleto en Español
For more information email: scn1a@chop.edu
ENDEAVOR: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Infants and Children With SCN1A-Positive Dravet Syndrome
ENDEAVOR is a Phase 1/2, 2-part, multi-center study in the United States to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged 6 months to less than 36 months.
Part 1 follows an open-label, dose-escalation design, and Part 2 is planned following demonstration of safety and efficacy in Part 1.
Additional details can also be found in the study listing at clinicaltrials.gov (NCT05419492).
Study Contact:
Name: Encoded Patient Advocacy
Phone Number: (650) 398-4301
Email: patientadvocacy@encoded.com
Study Locations and Contacts:
Location: UCSF Benioff Children’s Hospital: San Francisco, California
Contact: Joseph Sullivan, MD; joseph.sullivan@ucsf.edu
Location: Cook Children’s Medical Center; Fort Worth, Texas
Contact: Dianna Grado, RN; Dianna.Grado@cookchildrens.org; (682)885-2844
For more information on ETX101 and current trials, including information on studies in the UK (EXPEDITION) and Australia (WAYFINDER), visit: https://encoded.com/programs/clinical-studies/
A new study titled “GABA Biomarkers in Dravet Syndrome” is taking place at and sponsored by Cook Children’s Medical Center in Fort Worth, Texas in collaboration with Encoded Therapeutics.
The study is seeking participants aged up to 18 years with a diagnosis of SCN1A+ Dravet syndrome. This is a non-interventional study, which means study participants will not receive any experimental treatments.
Previous research has indicated that a chemical messenger in the brain, called GABA, is impacted in Dravet syndrome. The researchers in this study will use techniques to non-invasively measure the electric and magnetic activity in the human brain to try to develop biomarkers for levels of GABA in the human brain. The study will also involve blood draws to measure levels of GABA in the blood. Identifying a biomarker like this would be useful for understanding GABA mechanism in children with Dravet syndrome and could potentially guide the development of future treatments.
The study is posted on ClinicalTrials.gov (ID: NCT05651204)
If you are interested to learn more about participation, you can contact the study coordinators:
Sabrina Shandley, PhD
Phone: 682-715-5026
Email: NeuroResearch@cookchildrens.org
Study Name: ARGUS Trial
Study Sponsor: Harmony Biosciences (previously Epygenix Therapeutics)
Investigational Medicine: EPX-100 (Clemizole Hydrochloride, oral solution)
Study Phase: Phase 2
Study Goal: The study goal is to compare EPX-100 efficacy versus placebo (measured by percent change in seizure frequency).
Who can participate?
Children and adults with Dravet syndrome (2 years and older) who suffer from uncontrolled seizures.
Eligibility criteria include:
- Clinical and genetic diagnosis of Dravet syndrome
- Seizures not adequately controlled by current anti-epileptic drugs
- Experience 4 or more convulsive seizures during a 4-week baseline period
Additional criteria apply and will be discussed with you by the study team.
Will there be a placebo arm?
Yes, this is a placebo-controlled study, participants will have a 1 in 2 (50% chance) of receiving placebo (a liquid that looks like clemizole/EPX-100 but contains no active medication). The study is considered double-blind, so neither the participant/caregiver nor study doctor will know which group participants are in during the study.
Study Length: 4 week screening period + 16 week double-blind study
Open-Label Extension: All eligible participants will have the opportunity to enroll in a long-term open label extension study where participants will receive the investigational medicine.
Study Location(s): Global sites, including in the United States, Canada, Europe and Asia. Visit the links below to find individual site listings.
More information on the study:
- Study Website: ARGUStrial.com
- clinicaltrials.gov/study/NCT04462770
Study Name: DEEp SEA Study
Study Sponsor: Longboard Pharmaceuticals (now acquired by Lundbeck)
Investigational Medicine: bexicaserin (LP352)
Study Phase: Phase 3
Study Goal: To assess the safety of the investigational drug and its potential to reduce the number of seizures in children and adults with Dravet syndrome.
Who can participate?
- patient with a clinical diagnosis of Dravet syndrome
- patient is 2 to 65 years of age
- patient experiences countable motor seizures
Additional criteria apply and will be discussed with you by the study team.
Will there be a placebo arm?
Yes, this is a randomized placebo-controlled study, participants will randomly be assigned to receive the experimental therapy (bexicaserin/LP352) or a placebo medication.
Open-label Extension: Patients will have the opportunity to enroll in an open label extension study to receive access to the study medication if they meet eligibility criteria after completing the Phase 3 study.
Study Location(s): Sites are global, including in the United States, Canada, Brazil, Mexico, Australia, United Kingdom, Europe, and China. Site locations can be found at the links below.
More information on the study:
- Study website: www.DEEpDSStudy.com
- clinicaltrials.gov/study/NCT06660394
There is also a DEEp OCEAN Study that includes patients with other Developmental and Epileptic Encephalopathies (DEEs), including SCN1A Gain-of-Function DEE. More information at www.DEEpDEEStudy.com or clinicaltrials.gov/study/NCT06719141
Dr. Danielle Andrade at the University of Toronto is leading a study to better understand Dravet syndrome in adult patients. While they would like to know about seizure frequency, they also want to understand other aspects of daily life for adults with Dravet syndrome including behavior, gait, posture, and mood. Participants will be sent links to several questionnaires and asked to provide some video recordings (a tutorial on the video recordings will be provided to participants to explain more).
The first 100 participants will have the opportunity to be compensated for participation.
For more information, to ask questions, or to join the study, contact Arunan Selvarajah: arunan.selvarajah@uhnresearch.ca.
Study Name: EMPEROR Study
Study Sponsor: Stoke Therapeutics
Investigational Medicine: zorevunersen (STK-001)
Study Phase: Phase 3
Study Goal: To assess the effectiveness, safety, and tolerability of zorevunersen in patients with Dravet syndrome. The study will evaluate the potential of zorevunersen for disease modification; measuring changes in major motor seizure frequency as well as in behavior and cognition, clinical status, and health-related quality of life.
Who can participate?
- patient with a clinical diagnosis of Dravet syndrome and a documented mutation in the SCN1A gene (gain-of-function variants in SCN1A are not eligible)
- patient is 2 to 17 years of age
- patient has used of at least 2 prior or current seizure interventions (including anti-seizure medications, ketogenic diet, and/or vagus nerve stimulation)
- patient experiences a set number of major motor seizures during the baseline period
Additional criteria apply and will be discussed with you by the study team.
Will there be a placebo arm?
Yes, this is a sham-controlled study, participants will have a 1 in 2 (50% chance) of receiving a sham treatment (a lumbar puncture without administration of the experimental medicine). The study is considered double-blind, so neither the participant/caregiver nor study doctor will know which group participants are in during the study.
Study Length: 8-week baseline period and 52-week study period
Open-label Extension: Patients will have the opportunity to enroll in an open label extension study and receive zorevunersen if they meet eligibility criteria after completing the Phase 3 study.
Study Location(s): Approximately 60 sites globally in the United States, United Kingdom, Europe, and Japan. Specific study site information will be updated at the links below as sites become activated.
More information on the study:
- Study website, coming soon: https://www.emperorstudy.com/
- clinicaltrials.gov/study/NCT06872125
Study site information anticipated soon as sites become activated.
Open-Label, Single-Arm, Phase 3 Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome
The ORCHID study is evaluating an investigational oral medication called fenfluramine hydrochloride in young children 1 to <2 years of age with Dravet syndrome.
Your child may be eligible to participate if they:
- Are 1 to less than 2 years of age
- Have a documented or likely diagnosis of Dravet syndrome
- Are currently receiving at least 1 anti-seizure medication at a stable dose for at least 4 weeks prior to joining the study
- Are not currently receiving more than 4 anti-seizure medications
- Have had 4 or more motor seizures in the 28 days prior to joining the study
- There are additional criteria that need to be met, which the study team at the study center will discuss with you
To learn more and see if your child may be able to take part in this study, visit the study website at orchidstudy.com
For additional details, find the study listing on clinicaltrials.gov (NCT06118255)
The STARS Study is aDouble-Blind, Randomized, Placebo-Controlled, Multicenter, Outpatient, Parallel-Group Study to Assess the Efficacy and Safety of Staccato Alprazolam in Study Participants 12 Years of Age and Older With Stereotypical Prolonged Seizures
The STARS Study is testing if an inhaler containing an investigational drug is able to quickly stop a prolonged seizures (e.g.laster longer than 3 minutes) once they have begun.
- During the initial study, the inhaler will be sued to treat one seizure during the 12-week period, and participants will also be able to use rescue medications should their seizure persist after using the inhaler.
- Two versions of the inhaler will be tested in this initial study and there is a 50% chance of receiving each inhaler:
- One version will contain the active investigational drug
- One version will contain a placebo (no active medication)
- The STARS Study will last up to approximately 19 weeks and includes a 6-week Screening Period, a 12-week Study Treatment Period, and a Follow-Up Visit 5-12 days after the study treatment period ends.
- Participants may have the option of joining an extension study after completing the initial study, where all participants will receive the inhaler with the active investigational drug – there will be no placebo in the extension study.
There are over 100 clinical study sites internationally.
For more information, including where to find a study site near you, visit StarsEpilepsyStudy.com
Download the study flyer in English
Descargar este folleto en Español
Find the study listing at ClinicalTrials.gov (NCT05077904)
What is in the pipeline for Dravet?
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.
DSF places a high priority on funding research that has a clear path to genetic understanding, clinical application, and/or therapeutic development. Click here to view DSF Funded Research.
Therapeutic Pipeline for Dravet Syndrome
Genetic-Based Therapies
- Denotes US FDA Orphan Drug Designation or Rare Pediatric Disease Designation for Dravet syndrome
Last Updated March 2025
Why should you be cautious about sharing personal experiences in an active clinical trial?
With so many clinical trials for Dravet syndrome actively enrolling, it is an important time to consider the impacts that talking about experiences in an ongoing trial can have on the outcomes. Social media has changed the way we exchange information, and now it has the potential to influence clinical trial outcomes. Broad sharing of clinical trial details could have significant consequences and could affect your child’s participation in the trial. Read More
Clinical Trial Phases
Sometimes clinical trial phases are combined, particularly in rare diseases where the study population is small but has large unmet medical needs. You may see trials that are Phase 1/2a for example, where primary safety studies also evaluate if the medication is effective at the same time.
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 patients 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 patients to confirm its effectiveness while still monitoring for safety.
Purpose: Efficacy and Adverse Reactions
FDA Approval
FDA approval of a drug means that after reviewing the information from the clinical trials, the drug is determined to provide benefits that outweigh its known and potential risks for the intended population.
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: Long-term Safety and Efficacy
*More than one Phase III study may be required before a New Drug Application (NDA) may be submitted to the FDA.
Clinical Trials Glossary
Clinical research studies come in two main forms: interventional and observational. Observational studies, including Natural History Studies, gather information about patients with a specific disorder or diagnosis to better understand how the disorder presents in terms of symptoms, severity, and progression over time. Observational studies do not provide any type of treatment or therapy, but occur in addition to a patient’s normal medical care coordination. Interventional studies provide at least some of the participants in the study with an investigational intervention (such as a medication, procedure, or therapy) to determine if the intervention has an effect on measured symptoms.
A placebo is a “sham” treatment or substance without any therapeutic effect (such as a sugar pill) designed to be used in a study in place of the experimental treatment. In a placebo-controlled trial, a
certain proportion of study participants are given a placebo in place of a study drug so that group of study participants can be used as a comparison to determine if the study drug provided any benefit. Importantly, most participants, and often the study research team, are unaware of who received the placebo and who received the study drug until after the data is collected (see below section on “blinded
studies”).
Double-blinded studies mean that participants and researchers are not aware of who is in each study group (for example: placebo group versus investigational drug). This helps to prevent any bias, or unintentional effects of people’s emotions affecting the results of the trial. Because often everyone involved in a trial is hopeful that the experimental intervention will improve patient’s symptoms, this can create an unintentional bias towards results that support that conclusion. Sometime studies may be triple or quadruple blind, meaning even the researchers analyzing the data are unaware of the groups until the very end of the study. If no one is aware of who is receiving the study drug and who is receiving the placebo until after the results are analyzed then there is much less chance for bias to impact the results.
In a randomized trial, volunteers are assigned randomly to different groups (for example, the placebo group or investigational drug groups of varying doses). Randomly means participants are chosen by chance, and not by selection based on their characteristics, disease severity, or medical history. This helps to prevent bias, allowing for more reliable answers.
Outcome measures, also sometimes called “endpoints,” are the specific symptoms or characteristics that are being measured in participants in an interventional trial. Outcome measures are clearly outlined prior to the initiation of a clinical trial. Primary outcomes measures will be the symptom or characteristic measured which researchers consider to be the most important and if a significant change is measured during the trial in this outcome, the intervention is considered effective. In Dravet syndrome, changes in the frequency of convulsive seizures are often the primary outcome measure. Secondary outcome measures are other symptoms and characteristics that the researchers think will be important to measure, but not as important as the primary outcome measures.
Inclusion and exclusion criteria (or “eligibility criteria”) establish the characteristics that will allow an individual to participate (or be excluded from) a clinical study. These characteristics can include things such as age, gender, previous or current treatment history, and presence of other medical conditions. The goal of a clinical trial is to answer a specific research question, such as how well an investigational drug treats specific symptoms. Researchers specify inclusion and exclusion criteria to help them assess their specific question in the safest and most accurate way possible. Inclusion and exclusion criteria usually are different for every trial.
A study sponsor is responsible for overseeing, paying, and collecting the data for a clinical trial. The sponsor can be an individual, company, institution, group, or organization; most often, you will notice a pharmaceutical or biotechnology company is the sponsor.
Once a patient is approved for a clinical trial, but before any treatment begins, the study organizers will give them an informed consent document. Informed consent is the process of agreeing to participate in a clinical trial after learning all the details of the study. The informed consent document should contain details of the purpose of the study, how the study is conducted, how any personal medical information will be shared, specific requirements of participation, risks and benefits of participation, how long the study will last, any costs or payments to the participant, and important study contact information.
After reviewing the informed consent document, participants will sign the form if they wish to proceed. Taking part in a trial is voluntary. Participants can decide not to sign the informed consent and discontinue the trial. Signing the informed consent is not a contract to participate; participants can leave a trial at any time, for any reason.
Clinical trials monitor safety of an intervention in every phase. Adverse events are any unfavorable change in the health of a study participant while in the trial or within a set time after a trial has ended. An adverse event is not always caused by the intervention or investigational treatment that is being studied. Keeping track of any adverse event that occurs within participants in a trial can help researchers to determine if any of the adverse events are directly related to the study drug and/or if additional safety precautions are warranted.
