Ganaxolone Expanded Access Program Compassionate Use (NCT04678479) | Clinical Trial Compass
NO_LONGER_AVAILABLENot Applicable
Ganaxolone Expanded Access Program Compassionate Use
Plain-language 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.
Who can participate
Age range
2 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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:
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Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this ganaxolone expanded access program is no longer available, can you tell me whether ganaxolone has since been approved or if there are other active trials studying it for CDKL5 disorder that we could look into instead?
2Given that this was a compassionate use program rather than a standard clinical trial, what does that tell us about how much safety and effectiveness data currently exists for ganaxolone in CDKL5 disorder, and how should that shape our expectations?
3Are there any currently recruiting clinical trials specifically for CDKL5 disorder that you would recommend we explore, now that this particular program is closed?
4What standard or approved treatment options exist for managing seizures related to CDKL5 disorder right now, and how do those compare to what ganaxolone was being studied for?
5If ganaxolone seems like a promising direction based on what you know, is there any pathway — such as another expanded access request or a new trial — through which we might still be able to access it?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.