Active — Not RecruitingPhase 3

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

United States, Austria, Belgium87 participantsStarted 2022-03-08

Plain-language summary

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).

Who can participate

Age range

1 Year – 35 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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 participat…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Percentage Change from Baseline in Countable Motor Seizure Frequency (CMSF) (Part 1)

Timeframe: Baseline (28 days), Combined Titration and Maintenance (T+M) Periods (14 weeks)

Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) (Part 2)

Timeframe: Up to OLE1 Treatment Period Post-Dose Safety Follow-up Visit 17 (up to 70 weeks)

Percentage of Participants With Abnormal Physical Examination Findings (Part 2)

Timeframe: Up to End of Treatment (EoT)/Early Termination (ET) Visit 16 of OLE1 Treatment Period (up to 68 weeks)

Percentage of Participants With Abnormal Neurological Examination Findings (Part 2)

Timeframe: Up to EoT/ET Visit 16 of OLE1 Treatment Period (up to 68 weeks)

Percentage of Participants with Positive Response to Self-harm Question (Part 2)

Timeframe: OLE1 Treatment Period Day 1 to OLE1 Treatment Period Post-Dose Safety Follow-up Visit 17 (up to 70 weeks)

Percentage of Participants with Increase in Valvular Regurgitation from Baseline (except absent to trace) (Part 2)

Timeframe: Baseline (28 days), Cardiac Follow-up Visit 18 (up to 96 weeks)

Trial details

NCT IDNCT05064878

SponsorZogenix, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-11-08

View on ClinicalTrials.gov More CDKL5 Deficiency Disorder trials