Expanded Access with Adjunctive Ganaxolone (GNX) Treatment in Children and Young Adults with Cyclin-dependent Kinase-like 5 (CDKL5) deficiency disorder (CDD)

  • sponsor
    Marinus Pharmaceuticals
Updated on 30 June 2021


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 Cervical Intraepithelial Neoplasia, Rare Diseases and Disorders
Clinical Study IdentifierTX276149
SponsorMarinus Pharmaceuticals
Last Modified on30 June 2021


Yes No Not Sure

Inclusion Criteria

Molecular confirmation of a pathogenic or likely pathogenic CDKL5 variant, early onset, difficult to control seizures, and neurodevelopmental impairment are required
Male or female patients aged ≥ 2 years
Informed Consent signed by the patient, patient’s parent(s) or LAR indicating that they understand the purpose of and procedures required for expanded access and are willing to participate in the expanded access protocol
Age-appropriate assent, if indicated
In the opinion of the Investigator, the patient has inadequate seizure control on current anti-seizure medications at therapeutic doses

Exclusion Criteria

Pregnant or breastfeeding
Have an active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive as evaluated by brain imaging (magnetic resonance imaging)
Known allergic reaction or sensitivity to GNX or excipients
Previously or actively participating in a clinical trial of GNX
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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