Ganaxolone Expanded Access Program Compassionate Use

CDKL5 Disorder Clinical Trial

— CDD  

Official title:

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

Clinical Trial Details — Status: No longer available

Administrative data

• NCT number: NCT04678479
• Other study ID #: 1042-CDD-EAP-3005
• Status: No longer available

Study information

• Verified date: January 2024
• Source: Marinus Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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.

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 information / eligibility

• Status: No longer available
• Enrollment: 0
• Gender: All
• Age group: 2 Years and older

Eligibility

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:

-

Related Conditions & MeSH terms

• CDKL5 Disorder

Intervention

Drug:
• Ganaxolone
ganaxolone

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Marinus Pharmaceuticals