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.
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
Eisai Inc. has launched a Phase 3 clinical trial called Momentum I to study the efficacy of lorcaserin as an adjuctive (add-on) treatment in Dravet syndrome to reduce seizure frequency. This is a quadruple-blinded, placebo-control study is seeking to enroll 58 participants in a 14-week core study, with the potential for participants to enter an additional 12-week open-label extension phase following completion of the core study. Patients must be 2 years of age or older, have a diagnosis of Dravet syndrome, and have at least 4 convulsive seizures during the 4-week baseline period.
For more information on the Momentum I study for lorcaserin visit Momentum1Study.org or download this informational brochure.
Additional details can also be found in the study listing at clinicaltrials.gov (NCT04572243)
In a Phase II, multicenter, randomized, double-blind, placebo-controlled study, Epygenix will test the investigational drug, EPX-100 (Clemizole Hydrochloride, oral solution), as an adjunctive therapy for children and adults with Dravet syndrome (2 years and older) who suffer from uncontrolled seizures. The trial will take place at investigational sites in the US.
The study goal is to compare EPX-100 efficacy versus placebo (measured by percent change in seizure frequency).
To participate, your child would need to meet eligibility criteria. At a minimum, these 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
Your child’s personal data will remain private and confidential. Any medical data collected in the study will be used exclusively for therapy development. The aim is to develop transformative therapies to improve the lives of patients and families living with Dravet syndrome.
- More information about the ARGUS trial and locations can be found at ARGUStrial.com or at Clinicaltrials.gov (NCT04462770)
- You can download slides of an overview of the ARGUS study here.
- Watch a presentation from Epygenix at the 2022 DSF Family and Professional Conference.
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.
Neurelis is sponsoring a clinical trial, called the Stellina study, to investigate diazepam nasal spray (VALTOCO) as a rescue medication in children aged 2-5 years with epilepsy. This is a Phase 1/2a study to understand the pharmacokinetics (or how the body processes the medication) as well as to evaluate the safety of this medication in this patient group. There are several study sites across the US.
For more information:
- Visit the study website: neurelisclinicaltrials.com
- Download this informational brochure
- Visit clinicaltrials.gov (NCT05076838)
The MONARCH Study, conducted by Stoke Therapeutics, is currently enrolling for a Phase1/2a trial of STK-001. This new therapeutic medicine acts to specifically increase the number of healthy sodium channels (Nav1.1) that are impacted by mutations in SCN1A gene, the most common cause of Dravet syndrome. The study will focus on safety and tolerability, as well as assessing the efficacy of STK-001 to reduce seizure activity and patient health. It should take 7-9 months to complete, including 1 dose of the medication, a total of 8 visits to the study site, and 3 phone calls.
- Clinical trial details can be found on ClinicalTrials.gov.
- A quick information sheet can be found here.
- For more detailed information, please visit this link or email MONARCH@iqvia.com.
STK-001 is part of a new technology called TANGO that Stoke Therapeutics is leveraging to treat haploinsufficiencies like Dravet syndrome. If you would like to find out more about the science behind this approach, you can read this TANGO explainer provided by Stoke Therapeutics.
You can also hear Stoke Therapeutics discuss their novel approach and their clinical trials in this recorded session from the 2022 DSF Family & Professional Conference.
Takeda is sponsoring the Skyline Study, a Phase 3 clinical research study of an investigational medication for children, teens, and young adults, ages 2 to 21, diagnosed with Dravet syndrome. The investigational medication, soticlestat, works differently than currently approved medications and is being tested as an add-on therapy in the clinical research study. All participants will continue with their standard anti-seizure medication during the study.
Throughout participation in the 25-week study, all study-related visits, tests, and the investigational medication will be provided at no cost. Reimbursement for expenses related to study participation such as travel, meals, etc., may also be available.
Study staff will determine eligibility based on additional study criteria.
To learn more about the clinical research study:
- Visit the Skyline Study website at skyline-study.com
- Download the Study Brochure
- Watch this informational video about the Skyline Trial
- Watch this recorded session from Takeda at the 2022 DSF Family and Professional Conference
- See the trial listing at clinical trials.gov (NCT04940624)
El estudio Skyline es un estudio de investigación clínica de fase 3 de un medicamento en investigación para niños, adolescentes y adultos jóvenes, de 2 a 21 años, diagnosticados con síndrome de Dravet. El medicamento en investigación, soticlestat, funciona de manera diferente a los medicamentos aprobados actualmente y se está probando como terapia complementaria en el estudio de investigación clínica. Todos los participantes continuarán con su medicación anticonvulsiva estándar durante el estudio.
Durante toda la participación en el estudio de 25 semanas, todas las visitas, las pruebas y los medicamentos relacionados con el estudio se proporcionarán sin costo alguno. Es posible que también se reembolsen ciertos gastos relacionados con la participación en el estudio, como traslados, comidas, etc.
El personal del estudio determinará la elegibilidad según criterios adicionales del estudio.
Para obtener más información sobre el estudio de investigación clínica y comprobar si su hijo/a, o un ser querido, califica, visite el sitio web del estudio Skyline [skyline-study.com].
The STARS Study is testing an inhaler containing an investigational drug to see whether it could help people who experience prolonged epileptic seizures (i.e. lasting more than 3 minutes).
This is a Phase 3, double-blind, randomized, placebo-controlled study to assess the efficacy and safety of Staccato Alprazolam in study participants age 12 years and older with stereotypical prolonged seizures. The primary and secondary outcome measures will focus on treatment success for the treated seizure and recurrence of seizures in the hours following treatment, as well as gathering information on safety and any adverse events.
- Learn more and find study sites at the STARS trial website.
- Download the STARS Participant Brochure
- You can find detailed information on the trial at clinical trials.gov
- Additionally, you can watch this recorded session from the 2022 DSF Family and Professional Conference
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
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 for Dravet syndrome
Last Updated September 2022
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.