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

NCT ID NCT05064878

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

87 people

The number who actually took part.

Started

Mar 2022

Expected to finish

Nov 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 year to 35 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Subject has a confirmed pathogenic or likely pathogenic mutation in the CDKL5 gene and a clinical diagnosis of CDKL5 deficiency disorder (CDD) with epilepsy onset in the first year of life, plus motor and developmental delays. * Subject is male or female, aged 1 to 35 years, inclusive, as of the day of the Screening Visit. * Subject must have failed to achieve seizure control despite previous or current use of 2 or more antiepileptic treatments (AETs). * Subject is currently receiving at least 1 concomitant antiseizure treatment: antiseizure medication (ASM), vagus nerve stimulation (VNS), responsive neurostimulation (RNS), or ketogenic diet (KD). * All medications or interventions for epilepsy (including VNS, RNS, and KD) must be stable prior to screening and are expected to remain stable throughout the study. * At the Screening Visit, parent/caregiver reports that subject has ≥ 4 countable motor seizures (CMS) per week. Exclusion Criteria: * Subject has a known hypersensitivity to fenfluramine or any of the excipients in the study drug. * Subject has a diagnosis of pulmonary arterial hypertension. * Subject has a clinically significant medical condition, including chronic obstructive pulmonary disease, interstitial lung disease, or portal hypertension, or has had clinically relevant symptoms or a clinically significant illness currently or in the 4 weeks prior to the Screening Visit, other than epilepsy, that would negatively impact study participation, collection of study data, or pose a risk to the subject. * Subject has current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction or stroke, severe ventricular arrhythmias, or clinically significant structural cardiac abnormality, including but not limited to mitral valve prolapse, atrial or ventricular septal defects, patent ductus arteriosus, and patent foramen ovale with reversal of shunt. (Note: Patent foramen ovale or a bicuspid aortic valve are not considered exclusionary). * Subject has current eating disorder that suggests anorexia nervosa or bulimia. * Subject has a current or past history of glaucoma. * Subject is taking \> 4 concomitant antiseizure medications (ASMs). Rescue medications are not included in the count. * Subject is receiving concomitant treatment with cannabidiol (CBD) other than Epidiolex/Epidyolex or is being actively treated with tetrahydrocannabinol (THC) or any marijuana product for any condition. * Subject has moderate to severe hepatic impairment. * Subject is currently receiving another investigational product(s) or has received another investigational product within 30 days or within \< 5 times the half-lives of the investigational product, whichever is longer, prior to the Screening Visit. * Subject has previously been treated with Fintepla® (fenfluramine) prior to the Screening Visit.

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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

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

More trials for these conditions

Other studies related to the condition(s) this trial covers.