Clinical Research and Trials



This study will be enrolling ten patients, ages 2-35 years old, with a confirmed genetic/clinical diagnosis of CDKL5 Deficiency Disorder (CDD) in an open-label trial of fenfluramine for seizure control. Patients will be titrated over 14 days to a dose of ZX008 0.8 mg/kg/day (maximum dose of 30 mg/d).

Visit CDKL5 Fenfluramine Study for more information.

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

Ovid Therapeutics is sponsoring a clinical research study called the ARCADE study. ARCADE will assess the effect of OV935, an investigational medicine, on the frequency of seizures in participants with CDKL5 deficiency disorder (CDD) or duplication 15q syndrome (Dup15q). The study will also examine the safety and tolerability of OV935 and how it works within the body.

A list of participating sites and their contact information can be found at this link. To learn more about participating in the ARCADE clinical trial, please contact a location near you. You can also view the study FAQs and TAK935 fact sheet.

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.

Visit for more information.

*Please note that you do not need to take the pre-screener – contacting the site is the best way to get the most up to date study information.

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 or 617-355-5230.

DTI letter

DTI Details

Marigold Study – CDKL5 Deficiency Disorder

*This study is no longer recruiting

Marinus is committed to developing ganaxolone to improve the lives of children suffering
from CDKL5 deficiency disorder (CDD). Marinus’ pivotal Phase 3 Trial, #TheMarigoldStudy is no longer enrolling patients. Visit:

Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome

*This study is no longer recruiting

PTC Therapeutics is conducting a clinical trial in children with CDKL5 and Dravet Syndrome to evaluate the drug, Ataluren.  If you are interested in participating in the clinical trial, please speak with your doctor and visit the Clinical Trial website to learn more about the trial as well as inclusion and exclusion criteria.


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