Ganaxolone Expanded Access Program Compassionate Use

N/ANo Longer AvailableNCT04678479

Marinus Pharmaceuticals

Overview

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

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 enrollment.

Study Type

EXPANDED_ACCESS

Conditions

CDKL5 Disorder

Interventions

GanaxoloneDRUG

ganaxolone

Eligibility

Sex: ALLMin age: 2 Years

Medical Language ↔ Plain English

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: \-

Data from ClinicalTrials.gov

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