Clinical Research and Trials

CLINICAL TRIALS for CDKL5

Introducing FAiRE the ZX008-2103 CDKL5 FENFLURAMINE STUDY

Zogenix is pleased to share information about a clinical trial opportunity for patients with CDKL5 related epilepsy. The goal of the ZX008-2103 Study is to learn more about patients with CDKL5 deficiency disorder (CDD) and determine if ZX008 will reduce the seizures in patients who have disease-causing variations on the cyclin-dependent kinase-like5 gene. This study aims to enroll 100 patients aged 1-35 years in this Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study.

Check out this link for a quick introduction to the study: Fenfluramine Study Flyer.

Visit the CDKL5 Fenfluramine Study info at clinicaltrials.gov for the most up-to-date information on sites actively enrolling patients in the study. Check back for locations near you; as they become available to enroll patients, Zogenix will add them to this site list near the bottom of the page!


The PACIFIC Study

In this study, researchers want to test the safety of the investigational drug, LP352, and find out how the drug works when given in addition to anti-seizure medication in adults with developmental and epileptic encephalopathy. The study is now enrolling participants who are at least 18 years old. Visit the study website.


Marigold Study – CDKL5 Deficiency Disorder

*This study received FDA approval on March 18, 2022

Marinus Pharmaceuticals Announces FDA Approval of ZTALMY® (ganaxolone) for CDKL5 Deficiency Disorder

First and only FDA-approved treatment for seizures associated with CDKL5 deficiency disorder (CDD) in patients two years of age and older1 ZTALMY significantly reduced major motor seizure frequency in CDD patients in the pivotal Marigold trial.

Evaluating anatomic connectivity in the brain of patients with CDKL5 Deficiency Disorder

Using diffusion tensor imaging (DTI), data that is often collected but not processed during routine clinical MRI scans of the brain to evaluate functional and anatomic connectivity for a parallel study in CDD patients and mice.

If you would like to share your child’s MRI(s) for this study, we will ask you to enroll in Boston Children’s Hospital CDKL5 Clinic Study so that they are able to collect identifiable information including MRI CDs and other clinical records that allow them to identify your child’s CDKL5 genotype and phenotype. If you are interested, you can contact their CDKL5 research team at cdkl5@childrens.harvard.edu or 617-355-5230.

DTI letter

DTI Details


The ARCADE study adds two rare disorders, CDKL5 deficiency disorder and Duplication 15q syndrome, to TAK-935/OV935 clinical development.

*This study is no longer recruiting

We are eagerly awaiting communication from Takeda on how the CDD community might be involved in their global development for Soticlestat recently announced.

Takeda Pharmaceutical Company Limited and Ovid Therapeutics Inc. provided an overview of their TAK-935/OV935 broad clinical development programs and an extension trial for patients with developmental and epileptic encephalopathies (DEEs) who participated in a previous TAK-935/OV935 clinical study. Click here to read the full announcement.


Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome – completed

*Study results

Ataluren was not effective in reducing seizure frequency or improving cognitive, motor, or behavioral function or quality of life in subjects with either DS or CDD due to nonsense variants.

 

*IFCR is passing this information along to you for information purposes; please check with your doctor first to see if your child should participate.