Ganaxolone Expanded Access Program Compassionate Use (CDD)

January 18, 2024 updated by: Marinus Pharmaceuticals

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

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

Study Overview

Status

No longer available

Conditions

Intervention / Treatment

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 current anti seizure medications at therapeutic doses will be eligible for enrollment.

Study Type

Expanded Access

Expanded Access Type

  • Individual Patients
  • Treatment IND/Protocol

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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:

-

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Marinus Pharmaceuticals

Investigators

  • Study Director: Paula Bokesch, MD, FAAP, Marinus Pharmaceuticals, Inc.
  • Study Director: Igor Grachev, MD, PhD, Marinus Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 7, 2022

Primary Completion

December 7, 2022

Study Completion

December 7, 2022

Study Registration Dates

First Submitted

December 16, 2020

First Submitted That Met QC Criteria

December 16, 2020

First Posted (Actual)

December 22, 2020

Study Record Updates

Last Update Posted (Actual)

January 22, 2024

Last Update Submitted That Met QC Criteria

January 18, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 1042-CDD-EAP-3005

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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