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.
Drug: Ganaxolone
ganaxolone
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:
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Juby Philip
302-559-4320
jphilip@marinuspharma.com
Kevin Cintron
305-956-8271
kcintron@marinuspharma.com
Paula Bokesch, MD, FAAP
Study Director
Marinus Pharmaceuticals, Inc.
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Igor Grachev, MD, PhD
Study Director
Marinus Pharmaceuticals, Inc.