CLINICAL TRIALS for CDKL5
CDKL5 FENFLURAMINE STUDY
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.
Marigold Study – CDKL5 Deficiency Disorder
*This study is no longer recruiting but has an Expanded Access Program (EAP)
Expanded access, sometimes called “compassionate use,” is the use of an investigational medicine (i.e., one that has not been approved by the Food and Drug Administration (FDA)) by a patient who cannot be enrolled in a clinical trial. Marinus Pharmaceuticals supports Expanded Access Programs (EAPs) as an important way of allowing patients access to investigational medicines prior to FDA approval. Learn more here.
Last year, Marinus reported positive topline data from the Phase 3 Marigold Study, the first double-blind placebo-controlled trial to provide evidence of efficacy in CDD and the first Phase 3 trial to examine three times a day dosing of ganaxolone in pediatric patients. You can read more at this link.
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 firstname.lastname@example.org or 617-355-5230.
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
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.