Status:

NO_LONGER_AVAILABLE

Ganaxolone Expanded Access Program Compassionate Use

Lead Sponsor:

Marinus Pharmaceuticals

Conditions:

CDKL5 Disorder

Eligibility:

All Genders

2+ years

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 cur...

Eligibility Criteria

Inclusion

  • 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

  • \-

Key Trial Info

Start Date :

Trial Type :

EXPANDED_ACCESS

End Date :

Estimated Enrollment :

Patients enrolled

Trial Details

Trial ID

NCT04678479

Last Update

January 22 2024

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