Clinical Research and Trials

CLINICAL TRIALS for CDKL5

Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome

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 od4@nyu.edu or Latoya King at Latoya.King@nyumc.org for more information or questions on this study.


Ganaxolone Trial for Pediatric Epilepsy and Other Rare Genetic Epilepsies

Marinus Pharmaceuticals is conducting a clinical trial in children with CDKL5 to evaluate a compound it is developing for epilepsy, called ganaxolone. If you are interested in participating in the clinical trial, please speak with your doctor and visit Clinical Trials website to learn more about the trial as well as inclusion and exclusion criteria and who to contact to participate or if you have any questions.
This trial was initially for PCDH19, another rare epilepsy, but has expanded to include CDKL5.

Current enrolling sites are:

  • Sacramento, CA
  • San Francisco, CA
  • Atlanta, GA
  • Indianapolis, IN
  • Hackensack, NJ
  • Livingston, NJ
  • Rome, Italy

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