Active — Not RecruitingPhase 3

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

United States87 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 range1 Year – 35 Years

SexALL

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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…

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