Ganaxolone Expanded Access Program Compassionate Use (CDD)

Marinus Pharmaceuticals

Status

Conditions: CDKL5 Disorder

Treatments: Drug: Ganaxolone

Study type: Expanded Access

Funder types: Industry

Identifiers

NCT04678479

1042-CDD-EAP-3005

Details and patient eligibility

About

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

Full 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.

Sex: All

Ages: 2+ years old

Volunteers: No Healthy Volunteers

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