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
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
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.