A Phase 3 Randomized Double-Blind Placebo-Controlled Fixed-Dose Multicenter Study to Examine the Efficacy and Safety of ZX008 in Subjects with CDKL5 Deficiency Disorder Followed by an Open-Label Extension

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions:
    Cdkl5 Deficiency Disorder
  • Age: Between 1 years - 35 years
  • Gender: Male or Female
  • Other Inclusion Criteria:
    1. Participant has a confirmed pathogenic or likely pathogenic mutation in the CDKL5 gene and a clinical diagnosis of CDD with epilepsy onset in the first year of life, plus motor and developmental delays
    2. Participant must have failed to achieve seizure control despite previous or current use of 2 or more antiepileptic treatments (AETs)
    3. Participant is currently receiving at least 1 concomitant antiseizure treatment: antiseizure medication (ASM), vagus nerve stimulation (VNS), responsive neurostimulation (RNS), or ketogenic diet (KD)
    4. 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.
    5. At the Screening Visit, parent/caregiver reports that participant has =4 countable motor seizures (CMS) per week
    6. Participant (and/or participant's parent[s]/legal guardian[s]) has provided written informed consent (and assent if applicable)
    7. Participant (and/or participant's parent/caregiver) is willing and able to comply with study requirements (including diary completion, visit schedule, and study drug accountability)

You may not be eligible for this study if the following are true:

    1. Participant has a known hypersensitivity to fenfluramine or any of the excipients in the study drug
    2. Participant has a diagnosis of pulmonary arterial hypertension
    3. Participant 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 Participant
    4. Participant 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
    5. Participant has current eating disorder that suggests anorexia nervosa or bulimia
    6. Participant has a current or past history of glaucoma
    7. Participant is taking >4 concomitant ASMs
    8. Participant 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
    9. Participant has moderate to severe hepatic impairment, assessed based on the Child-Pugh system
    10. Participant has moderate to severe renal impairment
    11. Participant is receiving concomitant therapy with any of the following: centrally-acting anorectic agents; monoamine-oxidase inhibitors; any centrally-acting compound with clinically appreciable amount of serotonin agonist or antagonist properties, including serotonin reuptake inhibition; other centrally-acting noradrenergic agonists, including atomoxetine; or cyproheptadine
    12. Participant has participated in another interventional clinical trial within 30 days of the Screening Visit or is currently receiving an investigational product.
    13. Female subjects of childbearing potential who are pregnant or breastfeeding.
    14. Participant is known to be human immunodeficiency virus positive
    15. Participant is known to have active viral hepatitis B or C
    16. Participant is institutionalized in a facility that does not provide skilled epilepsy care
    17. Participant has previously been treated with Fintepla® (fenfluramine) prior to the Screening Visit

If you are registered as a volunteer, please log in to contact the study team/express interest in this study.