This is the first time that the study drug will be given to a patient with Dravet syndrome, and your child will be carefully monitored for safety and efficacy during the study. Half of the participants will be given a placebo (drug-free oral liquid solution), but your child will not know which treatment has been administered.
The study will also look at:
- If the study drug affects the general well-being of patients with Dravet syndrome.
- If there are any safety concerns for patients.
- How fast the study drug is removed from the body.
Up to 100 patients with Dravet syndrome from US, Canada, UK and Spain will take part in the study.
About The Argus Trial
The Argus trial is being pursued to see if EPX-100 (Clemizole hydrochloride) is able to decrease the number of convulsive seizures in patients with Dravet syndrome who are 2 years and older. The study will evaluate how often and what types of seizures are decreased in patients with Dravet syndrome as well as provide standard questionnaires to determine how the quality of life of patients is improving.
About Dravet Syndrome
Dravet syndrome is one of the most drug-resistant forms of epilepsy. Eighty percent (80%) of cases of Dravet syndrome are caused by SCN1A mutation. However, the cause in other cases remains unknown. The disease usually appears during the first year and not only results in persistent seizures but causes very serious development symptoms such as sleep disturbances, delayed language, and motor development, among other concerns.
Argus Trial Quick Facts
The Argus Study will have 3 phases: Screening, Titration, and Maintenance. Your child will participate in all 3 phases sequentially. The study doctors will share the details with you. In the screening phase, your child will need to visit the study center one time to receive a seizure calendar and instructions for the next 28 days. Your child will not receive any study drug at this stage, because this is just a way for the doctor to see how your child is doing with current medications.
In the Titration phase, your child will need to visit the study centerfor two visits and will receive 2 safety phone calls . Your child will be givenincreasing doses every week up to 80 mg twice a day This phase will last for 4 weeks.
In the Maintenance phase, your child will need to visit the study center three times and will receive 2 safety phone calls.Your child will maintain the same treatment and you will need to fill in a seizure calendar during these 84 days of treatment. You will also be asked to answer questions as to how your child is doing.
In the Titration phase, your child will need to visit the study center for two visits and will receive two safety phone calls. Your child will be given increasing doses every week up to eighty mg twice a day This phase will last for four weeks.
In the Maintenance phase, your child will need to visit the study center three times and will receive two safety phone calls. Your child will maintain the same treatment and you will need to fill in a seizure calendar during these eighty four days of treatment. You will also be asked to answer questions as to how your child is doing.
The study drug is an oral solution form of EPX-100 (Clemizole HCl). Clemizole HCl was previously approved by the FDA as an antihistamine for the treatment of allergies. Studies with the Dravet syndrome zebrafish model have shown that EPX-100 was able to reduce seizures; this predictable model provides hope that EPX-100 will be helpful to reduce seizures in patients with Dravet syndrome.
Want to know more? Please contact either the study coordinator at the nearest site or the site through https://clinicaltrials.gov/ct2/show/NCT04462770