Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Study)

Phase 2 Completed 29 enrolled Marinus Pharmaceuticals

RandomizedParallel-groupQuadruple-blindTreatment

Summary

A clinical study to evaluate the efficacy, safety, and tolerability of adjunctive ganaxolone therapy compared to placebo for the treatment of seizures in female children and young adults with genetically confirmed PCDH19 gene mutation.

Timeline

Start: 2019-05-17
Primary completion: 2021-01-19
Completion: 2022-06-20

Outcome

Missed primary endpoint

paper Wilcoxon rank-sum test, p = 0.17 PMID 36870093 ↗

paper however, the trend did not reach statistical significance. PMID 36870093 ↗

Publications

Sullivan J, Gunning B, Zafar M, Guerrini R, Gecz J, Kolc KL, Zhao Y, Gasior M, Aimetti AA, Samanta D. Phase 2, placebo-controlled clinical study of oral ganaxolone in PCDH19-clustering epilepsy. Epilepsy Res. 2023 Mar;191:107112. doi: 10.1016/j.eplepsyres.2023.107112. Epub 2023 Feb 22.

Drugs

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