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Ganaxolone Expanded Access Program Compassionate Use

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

Status
NO_LONGER_AVAILABLE
Phases
Unknown
Study type
Expanded Access
Source
ClinicalTrials.gov
Registry ID
NCT04678479
Acronym
CDD
Enrollment
Unknown
Registered
2020-12-22
Start date
Unknown
Completion date
Unknown
Last updated
2024-01-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

CDKL5 Disorder

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.

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.

Interventions

DRUGGanaxolone

ganaxolone

Sponsors

Marinus Pharmaceuticals
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
ALL
Age
2 Years to No maximum

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

\-

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026