Official Title
Expanded Access With Adjunctive Ganaxolone Treatment in Children and Young Adults With Cyclin Dependent Kinase-like 5 Deficiency Disorder
Brief Summary

The primary objective is to provide GNX to patients ≥ 2 years with CDD-related seizures who are refractory to, or intolerant of, standard therapy.

Detailed Description

This is a multi-center, long-term, open-label, expanded access protocol of adjunctive GNX

treatment in children, adolescents, and adults with CDD. Patients with inadequate seizure

control on their current anti seizure medications at therapeutic doses will be eligible for


Individual Patients
Treatment IND/Protocol
CDKL5 Disorder

Drug: Ganaxolone

Eligibility Criteria

Inclusion Criteria: - Molecular confirmation of a pathogenic CDKL5 variant, early onset, difficult to control seizures, and neurodevelopmental impairment are required. Male or female patients aged ≥ 2 years. In the opinion of the investigator, the patient has inadequate seizure control on current anti-seizure medications at therapeutic doses.

Exclusion Criteria:


Eligibility Gender
Eligibility Age
Minimum: 2 Years

Juby Philip

Kevin Cintron

Paula Bokesch, MD, FAAP
Study Director
Marinus Pharmaceuticals, Inc.


Igor Grachev, MD, PhD
Study Director
Marinus Pharmaceuticals, Inc.

Marinus Pharmaceuticals
NCT Number
MeSH Terms