A Study to Investigate the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder
NCT ID: NCT05064878
Last Updated: 2025-11-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE3
87 participants
INTERVENTIONAL
2022-03-08
2027-11-08
Brief Summary
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Detailed Description
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Part 1 of the study is 20 weeks in duration and will consist of the following stages: Baseline Period (ie, Baseline \[BL\]; 4 weeks including the Screening Visit and baseline observation), Titration Period (ie, Titration; 2 weeks), Maintenance Period (ie, Maintenance; 12 weeks), and a 2-week Transition Period (ie, Transition; 2 weeks) to the open-label starting dose.
Part 2 is a 54-week, open-label, flexible-dose, long-term extension for subjects who complete Part 1. Part 2 includes an Open-Label Extension (OLE) Treatment Period (52 weeks) with a Taper Period (ie, Taper; 2 weeks).
The primary study analysis to evaluate the efficacy and safety of ZX008 in children and adults with CDD will be based on Part 1 data in all randomized subjects.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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ZX008 0.8 mg/kg/day
Part 1: ZX008 0.8 mg/kg/day will be administered twice a day (BID) in equally divided doses; maximum of 30 mg/day, (subjects taking concomitant stiripentol will receive 0.5 mg/kg/day, \[maximum of 20 mg/day\]) with or without food.
ZX008 (Fenfluramine Hydrochloride)
ZX008 is supplied as an oral aqueous solution of Fenfluramine Hydrochloride.
Placebo
Part 1: Matching ZX008 placebo will be administered twice a day (BID) in equally divided doses with or without food.
Matching ZX008 Placebo
Matching ZX008 placebo is supplied as an oral solution.
ZX008
Part 2: Open-label ZX008 will be administered using a flexible dosing regimen, up to ZX008 0.8 mg/kg/day; maximum dose: 30 mg/day (subjects taking concomitant stiripentol will receive 0.5 mg/kg/day, \[maximum of 20 mg/day\]). ZX008 will be administered twice a day (BID) in equally divided doses with or without food.
ZX008 (Fenfluramine Hydrochloride)
ZX008 is supplied as an oral aqueous solution of Fenfluramine Hydrochloride.
Interventions
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ZX008 (Fenfluramine Hydrochloride)
ZX008 is supplied as an oral aqueous solution of Fenfluramine Hydrochloride.
Matching ZX008 Placebo
Matching ZX008 placebo is supplied as an oral solution.
Eligibility Criteria
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Inclusion Criteria
* 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 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 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 moderate to severe hepatic impairment.
* 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 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 participated in another interventional clinical trial within 30 days of the Screening Visit or is currently receiving an investigational product.
* Subject has previously been treated with Fintepla® (fenfluramine) prior to the Screening Visit.
1 Year
35 Years
ALL
No
Sponsors
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Zogenix, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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UCB Cares
Role: STUDY_DIRECTOR
001 844 599 2273
Locations
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Ep0216 154
Birmingham, Alabama, United States
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Los Angeles, California, United States
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San Francisco, California, United States
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Aurora, Colorado, United States
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Washington D.C., District of Columbia, United States
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Atlanta, Georgia, United States
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Boston, Massachusetts, United States
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Detroit, Michigan, United States
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Chapel Hill, North Carolina, United States
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Cleveland, Ohio, United States
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Philadelphia, Pennsylvania, United States
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Memphis, Tennessee, United States
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Austin, Texas, United States
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Linz, , Austria
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Brussels, , Belgium
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Edegem, , Belgium
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Tbilisi, , Georgia
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Bielefeld, , Germany
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Kehl-Kork, , Germany
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Kiel, , Germany
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Vogtareuth, , Germany
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Dublin, , Ireland
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Petah Tikva, , Israel
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Ramat Gan, , Israel
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Tel Aviv, , Israel
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Florence, , Italy
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Genova, , Italy
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Modena, , Italy
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Roma, , Italy
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Roma, , Italy
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Verona, , Italy
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Hiroshima, , Japan
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Niigata, , Japan
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Ōmura, , Japan
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Shizuoka, , Japan
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Zwolle, , Netherlands
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Lisbon, , Portugal
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Porto, , Portugal
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Barcelona, , Spain
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Madrid, , Spain
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Santiago de Compostela, , Spain
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Dubai, , United Arab Emirates
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Bristol, , United Kingdom
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London, , United Kingdom
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Manchester, , United Kingdom
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Sheffield, , United Kingdom
Countries
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Other Identifiers
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2021-003222-76
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
U1111-1303-2043
Identifier Type: OTHER
Identifier Source: secondary_id
ZX008-2103/EP0216
Identifier Type: -
Identifier Source: org_study_id