Ganaxolone Expanded Access Program Compassionate Use

NCT ID: NCT04678479

Last Updated: 2024-01-22

Basic Information

• Recruitment Status: NO_LONGER_AVAILABLE
• Study Classification: EXPANDED_ACCESS

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 enrollment.

Conditions

CDKL5 Disorder

Interventions

Ganaxolone

ganaxolone

Intervention Type: DRUG

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

-

• Minimum Eligible Age: 2 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Marinus Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Paula Bokesch, MD, FAAP

Role: STUDY_DIRECTOR

Marinus Pharmaceuticals, Inc.

Igor Grachev, MD, PhD

Role: STUDY_DIRECTOR

Marinus Pharmaceuticals, Inc.

Other Identifiers

1042-CDD-EAP-3005

Identifier Type: -

Identifier Source: org_study_id