New hope for rare epilepsy: drug shows promise in phase 3 trial
NCT ID NCT05064878
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This 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 where everyone gets the drug. The main goal is to see if motor seizures drop by at least 50% compared to baseline.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Fenfluramine (also known as FINTEPLA or ZX008), an oral liquid medication
- What this could lead to
- If it works, this could provide a new treatment option to reduce seizures in people with CDKL5 deficiency disorder, a rare and severe epilepsy condition.
- What could go wrong
- This is a Phase 3 trial, but results are not yet final. The drug may not work better than placebo, and there are known risks like heart valve problems and increased pressure in the lungs that require careful monitoring.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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San Francisco, California, 94158, United States
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Barcelona, Spain
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Madrid, Spain
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Santiago de Compostela, Spain
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Brookline, Massachusetts, 02115, United States
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Philadelphia, Pennsylvania, 19104-4318, United States
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Florence, Italy
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Verona, Italy
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Genova, Italy
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Roma, Italy
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Roma, Italy
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Modena, Italy
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Memphis, Tennessee, 38105, United States
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Detroit, Michigan, 48201, United States
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Zwolle, Netherlands
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Los Angeles, California, 90095, United States
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Washington D.C., District of Columbia, 20010, United States
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Shizuoka, Japan
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Niigata, Japan
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Hiroshima, Japan
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Omura-shi, Japan
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Birmingham, Alabama, 35233, United States
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Atlanta, Georgia, 30329, United States
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Cleveland, Ohio, 44195, United States
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Chapel Hill, North Carolina, 27514, United States
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Austin, Texas, 78731, United States
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Aurora, Colorado, 80045, United States
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Dublin, Ireland
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Tel Aviv, Israel
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Ramat Gan, Israel
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Petah Tikva, Israel
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Lisbon, Portugal
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Porto, Portugal
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Linz, Austria
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Tbilisi, Georgia
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Dubai, United Arab Emirates
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London, United Kingdom
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Sheffield, United Kingdom
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Bristol, United Kingdom
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Manchester, United Kingdom
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Edegem, Belgium
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Brussels, Belgium
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Vogtareuth, Germany
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Bielefeld, Germany
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Kiel, Germany
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Kehl-kork, Germany
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Brain device zaps seizures in Tough-to-Treat epilepsy
- Hot baths tested as seizure treatment for rare childhood disorder
- Fever's impact on seizures in rare genetic disorder revealed
- New drug trial aims to tame seizures in rare childhood disorder
- Warm baths tested as seizure treatment for rare childhood disorder
- New drug shows promise for Tough-to-Treat seizures in kids