DRAVET SYNDROME
Clinical trials for DRAVET SYNDROME explained in plain language.
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New hope for infants with severe epilepsy: fenfluramine trial launches
Disease control OngoingThis phase 3 trial is testing the safety and tolerability of fenfluramine (Fintepla) in 25 infants aged 1 to 2 years with Dravet syndrome, a severe form of epilepsy. The drug is given as an oral solution twice daily alongside other seizure medications. Researchers will monitor he…
Matched conditions: DRAVET SYNDROME
Phase: PHASE3 • Sponsor: UCB BIOSCIENCES, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:00 UTC
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Gene therapy trial aims to tame seizures in kids with rare epilepsy
Disease control OngoingThis early-stage study tests a gene therapy called ETX101 in 5 children with Dravet syndrome, a severe form of epilepsy. The therapy delivers a gene that helps calm overactive brain cells. Researchers will check if it safely reduces seizure frequency and improves thinking and dai…
Matched conditions: DRAVET SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Encoded Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 13:04 UTC
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CBD epilepsy drug under Real-World watch for rare seizure disorders
Disease control OngoingThis study follows about 111 people with Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex who are taking Epidyolex (a CBD oral solution) for seizures. Researchers track how many stay on the drug for a year and how many seizure-free days they have. It's an o…
Matched conditions: DRAVET SYNDROME
Sponsor: Jazz Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 13:01 UTC
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New hope for rare epilepsy: fenfluramine made available for dravet patients
Disease control APPROVED_FOR_MARKETINGThis study offers fenfluramine to people with Dravet syndrome whose seizures have not improved with other medications. The goal is to provide access to the drug and monitor its safety. Participants must have a genetic diagnosis of Dravet syndrome and have tried all available seiz…
Matched conditions: DRAVET SYNDROME
Sponsor: University of California, Los Angeles • Aim: Disease control
Last updated Jun 27, 2026 12:09 UTC
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Hope for dravet syndrome: Long-Term safety trial of STK-001 underway
Disease control OngoingThis study looks at the long-term safety of an experimental drug called zorevunersen (STK-001) in 60 people with Dravet syndrome, a severe form of epilepsy. Participants must have completed an earlier STK-001 study. Researchers will monitor side effects, seizure frequency, and qu…
Matched conditions: DRAVET SYNDROME
Phase: PHASE2 • Sponsor: Stoke Therapeutics, Inc • Aim: Disease control
Last updated Jun 27, 2026 09:01 UTC
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New hope for dravet syndrome: expanded access to seizure drug stiripentol
Disease control APPROVED_FOR_MARKETINGThis program provides expanded access to the drug stiripentol for people aged 6 months and older with Dravet syndrome, a severe form of epilepsy. The goal is to help control seizures that do not respond to other treatments. Participants must have a confirmed diagnosis and meet sa…
Matched conditions: DRAVET SYNDROME
Sponsor: Children's Hospital Medical Center, Cincinnati • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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New drug LP352 aims to control seizures in severe epilepsy over the long term
Disease control ENROLLING_BY_INVITATIONThis phase 3 study is testing the long-term safety and effectiveness of LP352 (Bexicaserin) in 324 children and adults with developmental and epileptic encephalopathy (DEE), including Dravet and Lennox-Gastaut syndromes. Participants who completed earlier studies will receive LP3…
Matched conditions: DRAVET SYNDROME
Phase: PHASE3 • Sponsor: Longboard Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 08:08 UTC
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Gene therapy trial aims to tame severe seizures in kids with dravet syndrome
Disease control OngoingThis study tests a gene therapy called ETX101 in 4 children with Dravet syndrome, a severe seizure disorder caused by a gene change. The therapy uses a harmless virus to deliver a working copy of the gene to brain cells. The main goals are to see if it is safe and if it reduces s…
Matched conditions: DRAVET SYNDROME
Phase: PHASE1, PHASE2 • Sponsor: Encoded Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:57 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Matched conditions: DRAVET SYNDROME
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 02, 2026 01:00 UTC
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Epidiolex liver check: is Long-Term use safe?
Knowledge-focused OngoingThis study follows 154 people taking Epidiolex (cannabidiol) for seizures caused by Lennox-Gastaut syndrome, Dravet syndrome, or tuberous sclerosis complex. Researchers will monitor for signs of chronic liver injury and fibrosis over time using blood tests and liver scans. The go…
Matched conditions: DRAVET SYNDROME
Phase: PHASE4 • Sponsor: Jazz Pharmaceuticals • Aim: Knowledge-focused
Last updated Jul 01, 2026 00:00 UTC
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New study aims to predict sudden death in severe epilepsy
Knowledge-focused OngoingThis study looks at how repeated seizures affect breathing and heart function in people with Dravet syndrome, a severe form of epilepsy. Researchers will monitor 92 children and adults for 24-48 hours using video, brain wave, heart, and breathing recordings. The goal is to find c…
Matched conditions: DRAVET SYNDROME
Phase: NA • Sponsor: Hospices Civils de Lyon • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:11 UTC