Promising seizure drug for rare genetic disorder enters final testing phase

NCT ID NCT05064878

First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 27 times

Summary

This study tests a medicine called ZX008 (fenfluramine) to see if it can safely reduce seizures in children and adults with CDKL5 deficiency disorder, a rare genetic condition that causes severe epilepsy and developmental delays. About 87 participants aged 1 to 35 who have not had success with at least two other seizure treatments will receive either the drug or a placebo. The goal is to measure how much seizure frequency drops and monitor for side effects.

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Contacts and locations

Locations

  • Ep0216 101

    San Francisco, California, 94158, United States

  • Ep0216 1103

    Barcelona, Spain

  • Ep0216 1117

    Madrid, Spain

  • Ep0216 1118

    Santiago de Compostela, Spain

  • Ep0216 113

    Brookline, Massachusetts, 02115, United States

  • Ep0216 120

    Philadelphia, Pennsylvania, 19104-4318, United States

  • Ep0216 1201

    Florence, Italy

  • Ep0216 1202

    Verona, Italy

  • Ep0216 1204

    Genova, Italy

  • Ep0216 1206

    Roma, Italy

  • Ep0216 1208

    Roma, Italy

  • Ep0216 1212

    Modena, Italy

  • Ep0216 124

    Memphis, Tennessee, 38105, United States

  • Ep0216 134

    Detroit, Michigan, 48201, United States

  • Ep0216 1401

    Zwolle, Netherlands

  • Ep0216 144

    Los Angeles, California, 90095, United States

  • Ep0216 149

    Washington D.C., District of Columbia, 20010, United States

  • Ep0216 1502

    Shizuoka, Japan

  • Ep0216 1505

    Niigata, Japan

  • Ep0216 1512

    Hiroshima, Japan

  • Ep0216 1518

    Omura-shi, Japan

  • Ep0216 154

    Birmingham, Alabama, 35233, United States

  • Ep0216 157

    Atlanta, Georgia, 30329, United States

  • Ep0216 164

    Cleveland, Ohio, 44195, United States

  • Ep0216 166

    Chapel Hill, North Carolina, 27514, United States

  • Ep0216 171

    Austin, Texas, 78731, United States

  • Ep0216 173

    Aurora, Colorado, 80045, United States

  • Ep0216 1803

    Dublin, Ireland

  • Ep0216 1904

    Tel Aviv, Israel

  • Ep0216 1906

    Ramat Gan, Israel

  • Ep0216 1909

    Petah Tikva, Israel

  • Ep0216 2104

    Lisbon, Portugal

  • Ep0216 2105

    Porto, Portugal

  • Ep0216 2505

    Linz, Austria

  • Ep0216 2802

    Tbilisi, Georgia

  • Ep0216 3101

    Dubai, United Arab Emirates

  • Ep0216 602

    London, United Kingdom

  • Ep0216 604

    Sheffield, United Kingdom

  • Ep0216 607

    Bristol, United Kingdom

  • Ep0216 611

    Manchester, United Kingdom

  • Ep0216 801

    Edegem, Belgium

  • Ep0216 804

    Brussels, Belgium

  • Ep0216 901

    Vogtareuth, Germany

  • Ep0216 902

    Bielefeld, Germany

  • Ep0216 908

    Kiel, Germany

  • Ep0216 909

    Kehl-kork, Germany

Conditions

Explore the condition pages connected to this study.