Study of Ganaxalone for Children With CDKL5 Deficiency Disorder Currently Enrolled 100 Participants

The Marigold study (NCT03572933) has obtained a 100 participant enrollment as of today. The Marigold study is a pivotal phase 3 study evaluating ganaxolone (Marinus Pharmaceuticals, Radnor, PA) in children and young adults with CDKL5 Deficiency Disorder (CDD), a rare genetic epilepsy. The enrollment will continue through the end of the month to capture the remaining few participants in the screening phase.  

“Enrolling 100 patients in our registrational, pivotal phase 3 trial evaluating ganaxolone in children with CDD is a significant milestone for both Marinus and the CDD community,” said Joe Hulihan, MD, chief medical officer of Marinus. “Our ability to identify and rapidly enroll qualified patients into this study is indicative of the significant need for a new therapy to reduce seizure burden and improve patient outcomes. The study has been well conducted and we are encouraged by the limited adverse events, low dropout rates and the vast majority of patients entering the open-label extension. We are appreciative of the patients and broader CDD community for their participation in the Marigold study and we remain committed to addressing the unmet need of these patients who currently have no approved treatment options.”

Karen Utley, president of the International Foundation for CDKL5 Research, commented, “We are grateful for Marinus’ recognition of the tremendous need of children with CDD and its dedication to advancing the development of ganaxolone as a potential new treatment for these children through the Marigold study. We are proud of our collaborative partnership to lead the way in finding a cure and treatments for children with CDD and are hopeful that ganaxolone may be a novel treatment option that improves outcomes for our children.”

The Marigold study is a global, double-blind, placebo controlled, phase 3 clinical trial designed to enroll approximately 100 participants between the ages of 2 and 21 with a confirmed disease related CDKL5 gene variant. Participants undergo a baseline period before being randomized to receive either ganaxolone, up to 1,800 mg/day, or placebo for 17 weeks in addition to their existing antiseizure treatment. Following the treatment period, all participants that meet certain eligibility requirements will have the opportunity to receive ganaxolone in the open label phase of the study. The study’s primary efficacy endpoint is percent reduction in seizures. Secondary outcome measures include nonseizure-related endpoints to capture certain changes in behavioral and sleep disturbances that were seen as improvements in previous clinical studies with ganaxolone.