DRAVET SYNDROME
Clinical trials for DRAVET SYNDROME explained in plain language.
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Hope for seizure control: dravet syndrome drug enters Long-Term testing
Disease control OngoingThis study is checking the long-term safety of repeated doses of an investigational drug called STK-001 for people with Dravet syndrome, a severe form of epilepsy. It is open to about 60 patients who have already safely received the drug in earlier studies. Researchers will also …
Matched conditions: DRAVET SYNDROME
Phase: PHASE2 • Sponsor: Stoke Therapeutics, Inc • Aim: Disease control
Last updated Mar 30, 2026 14:29 UTC
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Safety check: does a seizure drug harm the liver over time?
Disease control OngoingThis study aims to monitor the long-term safety of Epidiolex (cannabidiol), a prescription medication for severe seizure disorders. It will check if long-term use causes chronic liver injury or scarring in 154 participants who are already taking or starting the drug. The main goa…
Matched conditions: DRAVET SYNDROME
Phase: PHASE4 • Sponsor: Jazz Pharmaceuticals • Aim: Disease control
Last updated Mar 23, 2026 15:14 UTC
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Hope for tots: testing seizure drug in infants with rare, severe epilepsy
Disease control OngoingThis study is testing the safety and effects of a medication called fenfluramine in very young children (1 to under 2 years old) who have Dravet syndrome, a severe form of epilepsy. The main goal is to see if the drug is safe for their hearts and bodies, while also checking if it…
Matched conditions: DRAVET SYNDROME
Phase: PHASE3 • Sponsor: UCB BIOSCIENCES, Inc. • Aim: Disease control
Last updated Mar 20, 2026 14:48 UTC
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New hope for controlling severe seizures in rare childhood epilepsy
Disease control APPROVED_FOR_MARKETINGThis program provides expanded access to the medication stiripentol for patients with Dravet syndrome, a rare and severe form of childhood epilepsy. It allows patients aged 6 months and older who have hard-to-control seizures to receive this treatment outside of regular clinical …
Matched conditions: DRAVET SYNDROME
Sponsor: Children's Hospital Medical Center, Cincinnati • Aim: Disease control
Last updated Mar 18, 2026 14:41 UTC
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Gene therapy trial offers hope for kids with severe epilepsy
Disease control OngoingThis early-stage study is testing a one-time gene therapy called ETX101 in young children with Dravet syndrome, a severe form of genetic epilepsy. The main goals are to see if the treatment is safe and if it can reduce the number and severity of seizures over one year. The study …
Matched conditions: DRAVET SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Encoded Therapeutics • Aim: Disease control
Last updated Mar 17, 2026 13:09 UTC
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Gene therapy trial offers hope for children with devastating seizure disorder
Disease control OngoingThis early-stage study is testing a one-time gene therapy called ETX101 in young children (6 months to 4 years old) with a severe form of genetic epilepsy called Dravet Syndrome. The goal is to see if delivering a corrected gene directly to the brain is safe and can help reduce s…
Matched conditions: DRAVET SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Encoded Therapeutics • Aim: Disease control
Last updated Mar 04, 2026 15:29 UTC
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Scientists probe deadly link between seizures and sudden death in rare disorder
Knowledge-focused OngoingThis study aims to understand how repeated seizures in people with Dravet Syndrome affect the body's automatic functions like breathing and heart rate. Researchers will monitor 92 children and adults with this severe form of epilepsy during sleep and around the time of their seiz…
Matched conditions: DRAVET SYNDROME
Phase: NA • Sponsor: Hospices Civils de Lyon • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:28 UTC