A Phase 3 Study to Examine the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder.

Running, not enrolling · Phase 3 · Has a placebo group

Conditions studied: CDKL5 Deficiency Disorder, Generalized Tonic Clonic Seizure, Epileptic Spasm, Refractory Seizures

In brief

This is a Phase 3 Study to examine the efficacy and safety of ZX008 in children and adults with cyclin-dependent kinase like-5 (CDKL5) deficiency disorder (CDD).

Key facts

Study ID

NCT05064878

Run by

Zogenix, Inc.

People needed

87

Starts

2022-03-08

Expected to finish

2027-11-08

Last updated by the study team

2026-04-29

Who can join

Age: 1 and older, up to 35. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Ep0216 154 — Birmingham, Alabama, United States
• Ep0216 144 — Los Angeles, California, United States
• Ep0216 101 — San Francisco, California, United States
• Ep0216 173 — Aurora, Colorado, United States
• Ep0216 149 — Washington D.C., District of Columbia, United States
• Ep0216 157 — Atlanta, Georgia, United States
• Ep0216 113 — Brookline, Massachusetts, United States
• Ep0216 134 — Detroit, Michigan, United States
• Ep0216 166 — Chapel Hill, North Carolina, United States
• Ep0216 164 — Cleveland, Ohio, United States
• Ep0216 120 — Philadelphia, Pennsylvania, United States
• Ep0216 124 — Memphis, Tennessee, United States
• Ep0216 171 — Austin, Texas, United States
• Ep0216 2505 — Linz, Austria
• Ep0216 804 — Brussels, Belgium
• Ep0216 801 — Edegem, Belgium
• Ep0216 2802 — Tbilisi, Georgia
• Ep0216 902 — Bielefeld, Germany
• Ep0216 909 — Kehl-kork, Germany
• Ep0216 908 — Kiel, Germany
• Ep0216 901 — Vogtareuth, Germany
• Ep0216 1803 — Dublin, Ireland
• Ep0216 1909 — Petah Tikva, Israel
• Ep0216 1906 — Ramat Gan, Israel
• Ep0216 1904 — Tel Aviv, Israel

Full record on ClinicalTrials.gov

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