New hope for rare epilepsy: drug shows promise in phase 3 trial

NCT ID NCT05064878

Ongoing Disease control Sponsor: Zogenix, Inc. Source: ClinicalTrials.gov ↗

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

CDKL5 disorder developmental and epileptic encephalopathy, 2 epilepsy with generalized tonic-clonic seizures infantile spasms Spasms, Infantile visual epilepsy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CDKL5 DEFICIENCY DISORDER EPILEPTIC SPASM GENERALIZED TONIC CLONIC SEIZURE REFRACTORY SEIZURES

Contacts and locations

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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