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

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.

Timeline

Start

2018-06-30

Primary completion

2020-07-31

Completion

2021-05-28

Outcome

Met primary endpoint

paper Ganaxolone significantly reduced the frequency of CDD-associated seizures compared with placebo PMID 35429480 ↗

paper in the ganaxolone group and of -6·9% (-24·1 to 39·7) in the placebo group (p=0·0036). PMID 35429480 ↗

Publications

• Yasmen N, Sluter MN, Yu Y, Jiang J. Ganaxolone for management of seizures associated with CDKL5 deficiency disorder. Trends Pharmacol Sci. 2023 Feb;44(2):128-129. doi: 10.1016/j.tips.2022.11.007. Epub 2022 Dec 12. No abstract available.
• Knight EMP, Amin S, Bahi-Buisson N, Benke TA, Cross JH, Demarest ST, Olson HE, Specchio N, Fleming TR, Aimetti AA, Gasior M, Devinsky O; Marigold Trial Group. Safety and efficacy of ganaxolone in patients with CDKL5 deficiency disorder: results from the double-blind phase of a randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2022 May;21(5):417-427. doi: 10.1016/S1474-4422(22)00077-1.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Ganaxolone	Small molecule	63 mg/kg	Other
Subject	Ganaxolone	Small molecule	1800 mg	Other

Indications