Expanded Access With Adjunctive Ganaxolone Treatment in Children and Young Adults With Cyclin Dependent Kinase-like 5 Deficiency Disorder

1042-CDD-EAP-3005

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

-1

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: -

{'studyType': 'EXPANDED_ACCESS', 'expandedAccessTypes': {'individual': True, 'treatment': True}}

2024-01-22