The study will also look at:
- If there are any safety concerns for participants.
- How fast the study drug is processed by the body.
Up to 150 participants with Dravet syndrome will take part in this global study.
About The Argus Trial
The Argus Trial is being conducted to see if the investigational medicine, Clemizole is able to lower the number of seizures in participants with Dravet syndrome who are 2 years and older. The study will evaluate how often and what types of seizures are decreased in participants with Dravet syndrome.
This is the first time that the investigational medicine, Clemizole will be given to participants with Dravet syndrome, and your child will be carefully monitored for safety during the study. Participants will have a 1 in 2 (50%) chance of receiving either the investigational medication Clemizole, or a placebo (a liquid that looks like the investigational medication but contains no active medication). Neither you nor the study staff will know which one the participant is receiving. Participants who complete the first 4 months of the study may have the ability to continue to receive Clemizole in the optional extension period at no cost to them. Participants must remain on the antiseizure medications they are currently taking during the study.
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 a genetic mutation. However, the cause in other cases remains unknown. The disease usually appears during the first year and not only results in persistent drug-resistant seizures but also causes intellectual and developmental delays. Additionally, children with DS typically experience hyperactivity, sleep difficulties, growth and balance issues, and difficulty relating to others.
Argus Trial Quick Facts
The Argus clinical study may last up to about 3½ years, and includes 4 parts: Screening, Double-Blind Period, Long-term Extension, and Follow-up. Participants who complete the first 4 months of the study may have the ability to receive the investigational medication (Clemizole) for an additional 3 years. The study doctors will share the details with you.
| Screening | Double-Blind Period | Long-Term Extension | Follow-Up |
|---|---|---|---|
| Tests and procedures are done to see if a potential participant qualifies for the study | Participants will receive the investigational medication, Clemizole, or a placebo | All eligible participants will receive the investigational medication | The study team will check on the participant’s Dravet symptoms and overall health approximately 2 weeks after receiving the last dose of the investigational medication |
| About 1 visit | Clinic visits and phone calls | Clinic visits and phone calls | One phone call |
| 4 weeks | About 4 months | Up to 3 years | About 2 weeks |
Seizure and medication diary: You will fill out a seizure and medication diary at home to keep track of the participant’s seizures and when they take the investigational medication. The seizure diary is important to help researchers understand the effects of the investigational medication on the participant’s seizures.
Study Drug
The investigational medication, Clemizole, is an oral solution that will be administered twice a day during the study.
How To Qualify
People living with Dravet syndrome may be able to participate in this study if they*:
- Are at least 2 years old
- Genetically proven diagnosis of Dravet syndrome
- Onset of seizures prior to 18 months of age.
- Lack of seizure control despite taking 1 or more antiseizure medications.
- Have frequent seizures (average 4 seizures per month within last 28 days)
- Are not enrolled in another clinical study
*This is not a complete list of study requirements. The study doctor will review all the requirements with you and/or the potential participant.
Why Participate
If the person living with Dravet qualifies and decides to participate, they will receive:
- All study-related care and the investigational medication at no charge
- Assistance with study-related travel expenses (including food and lodging), as needed
- Close monitoring by a study doctor and study team who specialize in Dravet
- The opportunity to help researchers learn more about Dravet
Safety while participating in the Argus clinical study is our highest priority. If any questions or concerns arise, a study team member is available to help.
Participation in any clinical study is always voluntary. Participants are free to leave the study at any time and for any reason. Privacy will be maintained throughout the study.
Interested Parents and Caregivers
If your loved one has been diagnosed with Dravet syndrome and you are interested in learning if they may be eligible for participation in the Argus Trial, please reach out to clinicaltrials@harmonybiosciences.com and a representative from Harmony Biosciences will be in contact with you.
The study brochure provides information similar to this website to help you discuss participation in the Argus clinical study with the potential participant's doctor. You can also share it with someone that may want to learn about the study.
Study Locations
To find a clinical site near you, select a region below the
search box.
Please contact the study coordinator at the
nearest site listed or find contact information at https://clinicaltrials.gov/ct2/show/NCT04462770
Children’s Nebraska
8200 Dodge St Omaha, NE 68114
-
Sookyong Koh
-
402-559-2977
The Nemours Foundation
1600 Rockland Rd Wilmington, DE 19803
-
Stephen Falcheck
-
302-344-1528
University of Utah
201 South Presidents Circle, Rm 210 Salt Lake City, Utah 84112, United States
-
Angela Peters
-
801-585-9266
Seattle Children's Hospital
4800 Sand Point Way NE, M.B.7.420 Seattle, Washington 98105
-
Julie Ziobro
-
206-987-0058
Cincinnati Children’s Hospital Medical Center
3333 Burnet Avenue Suite ML 2015 Cincinnati, Ohio 45229
-
Gewalin Aungaroon
-
513-803-3177
Child Neurology Consultants of Austin
7940 Shoal Creek Boulevard Suite 100 Austin, Texas 78757
-
Karen Keough
-
210-416-4163
Wake Forest Baptist Medical Center
1 Medical Center Boulevard Winston-Salem, North Carolina 27157
-
Gautam S. Popli
-
336-716-2151
University of California, San Francisco Medical Center
550 16th Street 5th Floor San Francisco, California 94158
-
Ernesto Gonzalez Giraldo
-
415-353-8440
Cleveland Clinic
9500 Euclid Avenue Cleveland, Ohio 44195
-
Elia Pestana-Knight
-
216-636-5860
The Children’s Hospital of Philadelphia
The Hub for Clinical Collaboration 3550 Civic Center Blvd. 10th Floor Philadelphia, Pennsylvania 19104
-
Eric Marsh
-
215-419-3212
Weill Cornell Medical Center
505 East 70th Street Helmsley Tower, 3rd Floor New York, NY 10021
-
Zachary Grinspan
-
646-962-3023