NCT03572933

Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder

A Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone Treatment in Children and Young Adults With Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) Followed by Long-term Open-label Treatment

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 101 (actual)
• Sponsor: Marinus Pharmaceuticals · Industry
• Sex: All
• Age: 2 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

A clinical study to evaluate the efficacy, safety, and tolerability of adjunctive ganaxolone therapy compared to placebo for the treatment of seizures in children and young adults with genetically confirmed CDKL5 gene mutation.

Detailed description

The Marigold Study is a global, double-blind, placebo-controlled, Phase 3 clinical trial that will enroll approximately 70 patients between the ages of 2 and 21 with a confirmed disease-related CDKL5 gene variant. Patients will undergo a baseline period before being randomized to receive, in addition to their existing anti-seizure treatment, either ganaxolone or placebo for 17 weeks. Following the treatment period, all patients 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 will include non-seizure-related endpoints to capture certain behavioral and sleep disturbances that have been seen in previous clinical studies with ganaxolone.

Conditions

• CDKL5 Deficiency Disorder

Interventions

Type	Name	Description
DRUG	ganaxolone	active drug
DRUG	Placebo	inactive

Timeline

• Start date: 2018-06-30
• Primary completion: 2020-07-31
• Completion: 2021-05-28
• First posted: 2018-06-28
• Last updated: 2023-04-14
• Results posted: 2022-07-26

Locations

36 sites across 8 countries: United States, Australia, France, Israel, Italy, Poland, Russia, United Kingdom

Regulatory

• FDA-regulated drug study