NCT04678479

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.

Trial Health

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 16, 2020

Completed
6 days until next milestone

First Posted

Study publicly available on registry

December 22, 2020

Completed
Last Updated

January 22, 2024

Status Verified

January 1, 2024

First QC Date

December 16, 2020

Last Update Submit

January 18, 2024

Conditions

CDKL5 Disorder

Interventions

GanaxoloneDRUG

ganaxolone

Eligibility Criteria

Age2 Years+
Sexall
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

CDKL5 deficiency disorder

Interventions

ganaxolone

Study Officials

  • Paula Bokesch, MD, FAAP

    Marinus Pharmaceuticals, Inc.

    STUDY DIRECTOR

  • Igor Grachev, MD, PhD

    Marinus Pharmaceuticals, Inc.

    STUDY DIRECTOR

Study Design

Study Type
expanded access
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 16, 2020

First Posted

December 22, 2020

Last Updated

January 22, 2024

Record last verified: 2024-01