Marinus Pharmaceuticals has commenced a pivotal Phase lll clinical trial called Marigold Study to investigate the use of oral ganaxolone in children and young adults with CDKL5 Deficiency Disorder (CDD).
The global, double-blind, placebo-controlled trial will enrol about 70 patients aged between two and 21 with a confirmed disease-related CDKL5 gene variant.
As part of the Marigold Study, the patients will undergo a baseline period before being randomised to be treated with either ganaxolone or placebo for 17 weeks, in addition to their existing anti-seizure treatment.
After the treatment period, the patients meeting certain eligibility criteria will be given the opportunity to receive ganaxolone in the open-label phase of the trial.
Marigold Study’s primary efficacy objective is percentage reduction in seizures.
Its secondary outcome measures comprise non-seizure-related endpoints to capture certain changes in behavioural and sleep disturbances that were seen as improvements in earlier clinical studies with ganaxolone.
Marinus Pharmaceuticals CEO Christopher Cashman said: “After seeing the long-term results from our Phase ll trial showing that ganaxolone provides a meaningful reduction in the number of seizures for children with CDD, as well as an impressive increase in seizure-free days, we focused our efforts to expedite the initiation of this registration study.
“CDKL5 patients are in need of new treatment options and we look forward to working with the entire community in the conduct of this study.”
CDD is result of a mutation of the cyclin-dependent kinase-like 5 (CDKL5) gene, which is located on the X chromosome and encodes proteins essential for normal brain function.
Most of the children affected by CDD cannot walk, talk, or feed themselves. There are currently no approved therapies available for CDD.