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.

Takeda Pharmaceutical Company Limited[TSE: 4502] (“Takeda”) and Ovid Therapeutics Inc. (NASDAQ: OVID) today provided an overview of their TAK-935/OV935 broad clinical development program. The companies plan to initiate
three clinical trials: in pediatric patients with Dravet syndrome and Lennox-Gastaut syndrome, in pediatric patients with CDKL5 deficiency disorder (CDD) and Duplication 15q (Dup15q) syndrome, 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 and view the study FAQs and fact sheet.

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 or visit for more information.

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 now enrolling. If your child is between the ages of 2-21 w/ a confirmed #CDKL5 diagnosis, they might be a fit. Visit:

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 our CDKL5 Clinic Study so that we can collect identifiable information. This will include MRI CDs and other clinical records that allow us to identify your child’s CDKL5 genotype and phenotype. If you are interested, you can contact our CDKL5 research team at or 617-355-5230.

DTI letter

DTI Details

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.

You may also contact Dr. Orrin Devinsky at or Latoya King at for more information or questions on this study.



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