DEVELOPMENTAL AND EPILEPTIC ENCEPHALOPATHY
Clinical trials for DEVELOPMENTAL AND EPILEPTIC ENCEPHALOPATHY explained in plain language.
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Experimental gene therapy hopes to quiet seizures in kids with rare STXBP1 brain disease
Disease control TerminatedThis early-stage trial tests a single dose of CAP-002 gene therapy in 12 children aged 18 months to 8 years with STXBP1 encephalopathy, a genetic condition that causes severe seizures and developmental delays. The main goal is to check safety, but researchers will also track whet…
Matched conditions: DEVELOPMENTAL AND EPILEPTIC ENCEPHALOPATHY
Phase: PHASE1, PHASE2 • Sponsor: Capsida Biotherapeutics, Inc. • Aim: Disease control
Last updated May 17, 2026 08:35 UTC
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New hope for rare seizure disorders: Long-Term drug safety trial underway
Disease control ENROLLING_BY_INVITATIONThis study is testing the long-term safety and effectiveness of an investigational drug called LP352 for controlling seizures in children and adults with developmental and epileptic encephalopathy (DEE), a group of severe brain disorders. About 324 people who completed earlier st…
Matched conditions: DEVELOPMENTAL AND EPILEPTIC ENCEPHALOPATHY
Phase: PHASE3 • Sponsor: Longboard Pharmaceuticals • Aim: Disease control
Last updated May 17, 2026 08:31 UTC
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Could a common drug ease rare childhood epilepsy?
Symptom relief OngoingThis study tests whether a medicine called phenylbutyrate is safe and tolerable for children with certain genetic conditions that cause seizures and developmental delays, such as STXBP1 and SLC6A1 disorders. The drug aims to help the body's remaining proteins work better. Researc…
Matched conditions: DEVELOPMENTAL AND EPILEPTIC ENCEPHALOPATHY
Phase: EARLY_PHASE1 • Sponsor: Weill Medical College of Cornell University • Aim: Symptom relief
Last updated May 11, 2026 20:40 UTC