Ganaxolone Expanded Access Program Compassionate Use

Last updated: January 18, 2024
Sponsor: Marinus Pharmaceuticals
Overall Status: Trial Not Available

Phase

N/A

Condition

N/A

Treatment

Ganaxolone

Clinical Study ID

NCT04678479
1042-CDD-EAP-3005
  • Ages > 2
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Molecular confirmation of a pathogenic CDKL5 variant, early onset, difficult tocontrol seizures, and neurodevelopmental impairment are required. Male or femalepatients aged ≥ 2 years. In the opinion of the investigator, the patient hasinadequate seizure control on current anti-seizure medications at therapeutic doses.

Exclusion

Exclusion Criteria:

Study Design

Treatment Group(s): 1
Primary Treatment: Ganaxolone
Phase:
Study Start date:
Estimated Completion Date:

Study 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 enrollment.