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Genetic developmental and epileptic encephalopathy
MONDO:0100062A complex neurodevelopmental disorder characterized by a range of developmental delays and epileptic encephalopathy phenotypes. Seizure onset is variable and intellectual disability is variable in presence and severity.
Also known as: developmental and epileptic encephalopathy, hereditary developmental and epileptic encephalopathy
93 clinical trials for this condition and its sub-types, 2 tagged with Genetic developmental and epileptic encephalopathy itself.
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Sub-types of Genetic developmental and epileptic encephalopathy
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Dravet syndrome 39 trials
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Lennox-Gastaut syndrome 26 trials · 27 incl. sub-types
3 sub-types
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Neonatal-onset developmental and epileptic encephalopathy 0 trials · 5 incl. sub-types
20 sub-types
- Developmental and epileptic encephalopathy, 13 3 trials
- Developmental and epileptic encephalopathy, 25 1 trial
- Developmental and epileptic encephalopathy, 42 1 trial
- Developmental and epileptic encephalopathy, 12 0 trials
- Developmental and epileptic encephalopathy, 16 0 trials
- Developmental and epileptic encephalopathy, 21 0 trials
- Developmental and epileptic encephalopathy, 24 0 trials
- Developmental and epileptic encephalopathy, 26 0 trials
- Developmental and epileptic encephalopathy, 28 0 trials
- Developmental and epileptic encephalopathy, 29 0 trials
- Developmental and epileptic encephalopathy, 31A 0 trials
- Developmental and epileptic encephalopathy, 32 0 trials
- Developmental and epileptic encephalopathy, 33 0 trials
- Developmental and epileptic encephalopathy, 34 0 trials
- Developmental and epileptic encephalopathy, 39 0 trials
- Developmental and epileptic encephalopathy, 41 0 trials
- Developmental and epileptic encephalopathy, 44 0 trials
- Developmental and epileptic encephalopathy, 45 0 trials
- Developmental and epileptic encephalopathy, 46 0 trials
- Developmental and epileptic encephalopathy, 47 0 trials
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2 sub-types
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New hope for dravet syndrome: phase 3 trial of LP352 aims to cut seizure frequency
Disease control OngoingThis study tests whether the drug LP352 can safely reduce seizures in children and adults with Dravet syndrome, a severe form of epilepsy. About 160 participants will receive either LP352 or a placebo, and researchers will track changes in seizure frequency over several months. T…
Phase 3 • Sponsor: Longboard Pharmaceuticals • Aim: Disease control
Last updated Sep 19, 2026 00:00 UTC
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New hope for Hard-to-Treat seizures: phase 3 trial launches
Disease control OngoingThis study tests an experimental drug called LP352 to see if it can safely reduce seizures in children and adults with developmental and epileptic encephalopathies (DEE), a group of severe epilepsy syndromes. About 320 participants will receive either LP352 or a placebo, and rese…
Phase 3 • Sponsor: Longboard Pharmaceuticals • Aim: Disease control
Last updated Sep 17, 2026 00:00 UTC
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Brain implant shows promise for Hard-to-Treat seizures in rare epilepsy
Disease control OngoingThis study tests a brain-responsive neurostimulation system in 24 people aged 12 and older with Lennox-Gastaut syndrome whose seizures are not controlled by medication. The device detects and responds to seizure activity in the brain to reduce drop seizures. The goal is to see if…
Phase 2 • Sponsor: NeuroPace • Aim: Disease control
Last updated Sep 17, 2026 00:00 UTC
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New drug LP352 aims to control seizures in severe epilepsy over the long term
Disease control By invitation onlyThis 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…
Phase 3 • Sponsor: Longboard Pharmaceuticals • Aim: Disease control
Last updated Sep 17, 2026 00:00 UTC
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New hope for kids with rare epilepsy: drug trial targets seizure control
Disease control OngoingThis study tests an investigational drug called relutrigine in 160 children with a severe form of epilepsy called developmental and epileptic encephalopathy (DEE). The goal is to see if the drug can safely reduce monthly seizure frequency compared to a placebo. After the initial …
Phase 3 • Sponsor: Praxis Precision Medicines • Aim: Disease control
Last updated Aug 06, 2026 00:00 UTC
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Can a new drug tame severe childhood epilepsy?
Disease control OngoingThis trial is testing an experimental drug called PRAX-222 in children aged 2 to 18 who have a rare, severe form of epilepsy caused by changes in the SCN2A gene. The goal is to see if the drug is safe and can reduce how often seizures happen. Participants will receive either PRAX…
Phase 1/2 • Sponsor: Praxis Precision Medicines • Aim: Disease control
Last updated Aug 05, 2026 00:00 UTC
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Real-World study tracks Epidyolex's Long-Term impact on seizures
Disease control OngoingThis study follows 158 people in France who are prescribed Epidyolex (a cannabidiol-based medicine) for seizures as part of their normal care. Researchers will track how long people stay on the treatment, side effects, seizure frequency, and changes in daily functioning and quali…
Sponsor: Jazz Pharmaceuticals • 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…
Phase 1/2 • 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…
Sponsor: Jazz Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 13:01 UTC
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New hope for rare epilepsy: drug shows promise in phase 3 trial
Disease control OngoingThis Phase 3 study tests whether fenfluramine (ZX008) can safely reduce seizures in 87 children and adults with CDKL5 deficiency disorder, a rare genetic condition causing severe epilepsy. Participants receive either the drug or a placebo, followed by an open-label extension wher…
Phase 3 • Sponsor: Zogenix, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:24 UTC
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New hope for rare epilepsy: fenfluramine made available for dravet patients
Disease control ApprovedThis 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…
Sponsor: University of California, Los Angeles • Aim: Disease control
Last updated Jun 27, 2026 12:09 UTC
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Brain pacemaker shows promise for rare childhood epilepsy
Disease control OngoingThis pilot study tests whether deep brain stimulation (DBS) of specific brain pathways can safely reduce seizures in people with Lennox-Gastaut syndrome, a severe form of epilepsy. Five participants receive the electrical stimulation as an add-on to their usual treatment. The goa…
Sponsor: Liankun_Ren • Aim: Disease control
Last updated Jun 27, 2026 12:06 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…
Phase 2 • Sponsor: Stoke Therapeutics, Inc • Aim: Disease control
Last updated Jun 27, 2026 09:01 UTC
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New hope for kids with hard-to-treat genetic seizures
Disease control OngoingThis study tests a new medicine called PRAX-562 in 77 children with rare genetic epilepsies (SCN2A or SCN8A). The goal is to see if it safely reduces motor seizures. The trial has two parts: a double-blind phase where some children get the drug and some get a placebo, followed by…
Phase 2/3 • Sponsor: Praxis Precision Medicines • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC
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New hope for dravet syndrome: expanded access to seizure drug stiripentol
Disease control ApprovedThis 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…
Sponsor: Children's Hospital Medical Center, Cincinnati • Aim: Disease control
Last updated Jun 27, 2026 08:13 UTC
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Could a urea cycle drug help kids with rare epilepsy?
Disease control OngoingThis early-phase trial is testing whether glycerol phenylbutyrate (Ravicti), a drug already approved for a different condition, is safe and tolerable for children with genetic disorders like STXBP1 and SLC6A1 that cause seizures and developmental delays. The study will enroll 50 …
Early phase 1 • Sponsor: Weill Medical College of Cornell University • Aim: Disease control
Last updated Jun 27, 2026 08:12 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…
Phase 1/2 • Sponsor: Encoded Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 07:57 UTC
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Bridge program keeps seizure drug available for eligible patients
Disease control Expanded access (ended)This program provides continued access to the seizure medication perampanel (Fycompa) for people who completed certain earlier studies and are still benefiting from the drug. It runs until the drug is commercially available in their country or no participants remain. Only those a…
Sponsor: Eisai Inc. • Aim: Disease control
Last updated Jun 26, 2026 17:19 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…
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC
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New inhaler aims to stop prolonged seizures fast and safely
Symptom relief By invitation onlyThis study tests the long-term safety of an inhaled medication called Staccato alprazolam for people aged 12 and older who have prolonged seizures. About 300 participants will use the inhaler when a seizure starts to see if it stops the seizure quickly and without serious side ef…
Phase 3 • Sponsor: UCB Biopharma SRL • Aim: Symptom relief
Last updated Aug 16, 2026 00:00 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…
Phase 4 • Sponsor: Jazz Pharmaceuticals • Aim: Knowledge-focused
Last updated Aug 28, 2026 00:00 UTC
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Counting the uncounted: a nationwide look at two rare epilepsies
Knowledge-focused CancelledThis observational study aims to measure how many people in Portugal have Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS), two rare and severe forms of epilepsy. Researchers will review existing medical records from about three public hospitals to estimate the percentage of…
Sponsor: Takeda • Aim: Knowledge-focused
Last updated Aug 19, 2026 00:00 UTC
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EEG clues may reveal who benefits from seizure drug
Knowledge-focused By invitation onlyThis study looks at whether specific brain wave patterns on an EEG can predict how well the drug fenfluramine works for people with Lennox-Gastaut Syndrome (LGS), a severe form of epilepsy. Researchers will follow 20 children and adults aged 2 to 35 who are already taking fenflur…
Sponsor: University of Chicago • Aim: Knowledge-focused
Last updated Aug 08, 2026 00:03 UTC
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Can mapping a rare Gene's effects pave the way to targeted epilepsy therapies?
Knowledge-focused OngoingThis study follows children aged 6 to 21 who have a CNKSR2 gene mutation, which can cause epilepsy, language difficulties, and intellectual disability. Researchers will use brain scans, EEGs, and developmental tests to track how the condition changes over time. The goal is to cre…
Sponsor: University of California, San Francisco • Aim: Knowledge-focused
Last updated Jul 25, 2026 00:00 UTC
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New registry aims to unlock secrets of rare CDKL5 disorder
Knowledge-focused PausedThis study creates a registry for up to 500 people with CDKL5 Deficiency Disorder (CDD), a rare genetic condition that causes seizures and developmental delays. Patients and their caregivers will provide information about symptoms, treatments, and quality of life over several yea…
Sponsor: University of Pennsylvania • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:23 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…
Sponsor: Hospices Civils de Lyon • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:11 UTC