CDD: Ganaxolone Expanded Access Program Compassionate Use
Sponsor
Marinus Pharmaceuticals (Industry)
Overall Status
Available
CT.gov ID
NCT04678479
Collaborator
(none)
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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 Design
Study Type:
Expanded Access
Official Title:
Expanded Access With Adjunctive Ganaxolone Treatment in Children and Young Adults With Cyclin Dependent Kinase-like 5 Deficiency Disorder
Outcome Measures
Primary Outcome Measures
Eligibility Criteria
Criteria
Ages Eligible for Study:
2 Years
and Older
Sexes Eligible for Study:
All
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:
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Marinus Pharmaceuticals
Investigators
- Study Director: Paula Bokesch, MD, FAAP, Marinus Pharmaceuticals, Inc.
- Study Director: Igor Grachev, MD, PhD, Marinus Pharmaceuticals, Inc.
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.Responsible Party:
Marinus Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT04678479
Other Study ID Numbers:
- 1042-CDD-EAP-3005
First Posted:
Dec 22, 2020
Last Update Posted:
Apr 4, 2022
Last Verified:
Apr 1, 2022
Additional relevant MeSH terms: