A Phase I, Single Dose Study of Ganaxolone Pharmacokinetics in Adult Subjects with Normal and Impaired Hepatic Function
Abstract number :
3.137
Submission category :
2. Translational Research / 2A. Human Studies
Year :
2025
Submission ID :
229
Source :
www.aesnet.org
Presentation date :
12/8/2025 12:00:00 AM
Published date :
Authors :
Presenting Author: Christine Ochoa Escamilla, PhD – Immedica North America
Joseph Hulihan, MD – Paradigm Neuroscience
Richard Preston, MD – Miller School of Medicine University of Miami
Thomas Marbury, MD – Orlando Clinical Research Center
Rationale: Ganaxolone, a neuroactive steroid that modulates both synaptic and extrasynaptic GABAA receptors, is approved in the US, Europe, and China for the treatment of seizures associated with CDKL Deficiency Disorder (CDD) in patients ≥ 2 years old. Given the important role of hepatic metabolism in the pharmacokinetic (PK) disposition of ganaxolone, a study was conducted to understand the impact of hepatic impairment on its exposure.
Methods: This phase I, multicenter, open label study evaluated the PK, safety and tolerability of ganaxolone in subjects with normal versus impaired hepatic function. Participants 18-79 years old were eligible if they had Child-Pugh class A, B or C hepatic impairments or normal hepatic function and body-mass index (BMI) ≥18.5 kg/m2 to < 42.0 kg/m
Translational Research