Ganaxolone Expanded Access Program Compassionate Use
- Conditions
- CDKL5 Disorder
- Registration Number
- NCT04678479
- Lead Sponsor
- Marinus Pharmaceuticals
- 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.
Recruitment & Eligibility
- Status
- NO_LONGER_AVAILABLE
- Sex
- All
- Target Recruitment
- Not specified
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.
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Exclusion Criteria
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Study & Design
- Study Type
- EXPANDED_ACCESS
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method