Double-blind, Randomized, Placebo-controlled Trial of Ganaxolone in CDKL5 Deficiency Patients 6 M… (NCT05249556) | Clinical Trial Compass
Not Yet RecruitingPhase 3
Double-blind, Randomized, Placebo-controlled Trial of Ganaxolone in CDKL5 Deficiency Patients 6 Months to Less Than 2 Years Old
20 participantsStarted 2026-06
Plain-language summary
This study will assess the efficacy, safety, and tolerability of ganaxolone (GNX) compared with placebo (PBO) as adjunctive therapy to the participant's standard anti-epileptic medication for the treatment of seizures in pediatric patients from 6 months to less than 2 years old with genetically confirmed CDD during a 12-week, DB phase. Pharmacokinetic (PK) assessments and population PK analyses will also be performed during this time. The DB phase will be followed by an optional long-term OL phase at which time all participants will receive GNX as an adjunct to their standard anti-seizure medication. Efficacy, safety and tolerability, and PK assessments will continue to be performed.
Who can participate
Age range
6 Months – 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
. A diagnosis of CDD, including molecular confirmation of a pathogenic or likely pathogenic CDKL5 variant and refractory seizures (see Inclusion criterion 5).
. The principal investigator (PI) must review the results of the genetic analysis and confirm that gene mutation is likely to be the cause of the epilepsy syndrome.
. If the participant has a de novo variant of unknown significance (VUS) in the kinase domain of the CDKL5 gene, parental testing is negative and meets all other inclusion criteria, then the participant can be included.
. Genetic mutations will be confirmed by the sponsor's chosen central laboratory. In France, genetic mutations may be confirmed by an approved French organization, in compliance with French legislation prior to Screening/Visit 1.
. Male or female participants aged 6 months to less than 2 years.
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.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
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.
What they're measuring
1
Percent Change from baseline in 28-day frequency of countable seizures
. Parent(s) or LAR willing to give written informed consent, after being properly informed of the nature and risks of the study and prior to engaging in any study-related procedures.
. Failure to control seizures despite appropriate trial of 1 or more anti-seizure medications at therapeutic doses.
. Have a history of at least 8 countable seizures during the 28 days prior to screening. Countable seizures will be defined by the following:
Exclusion criteria
. Have an active CNS infection, demyelinating disease, degenerative neurological disease, or CNS disease deemed progressive as evaluated by brain imaging (magnetic resonance imaging \[MRI\]).
. Have any disease or condition (medical or surgical; other than CDKL5 deficiency) at screening that might compromise the hematologic, cardiovascular, pulmonary, renal, gastrointestinal, or hepatic systems; or other conditions that might interfere with the absorption, distribution, metabolism, or excretion of the IP, or would place the participant at increased risk.
. Has a positive result for tetrahydrocannabinol (THC) test (via urine or plasma drug screen) at the screening visit. Concomitant Epidiolex/Epidyolex (cannabidiol derivative \[CBD\]) use will be allowed in the DB phase provided the participant has been on a stable dose for at least 1 month prior to screening and is expected to remain on a stable dose without a foreseeable change for the duration of the DB phase.
. Use of a CBD preparation other than Epidiolex/Epidyolex for 1 month prior to screening.
. An AST (aspartate aminotransferase/serum glutamic-oxaloacetic transaminase \[SGOT\]) or ALT (alanine aminotransferase/serum glutamic-pyruvic transaminase \[SGPT\]) \> 3 times the upper limit of normal (ULN) at study entry. If AST or ALT increases \> 3 X ULN during the study, the participant should be followed with weekly laboratory repeat testing and continue in study if levels trending down. The participant will be discontinued if levels do not decline to \< 3 X ULN.
. Total bilirubin levels greater than the ULN at study entry. In cases of a documented, stable medical condition (ie, Gilbert's Syndrome) resulting in levels of total bilirubin greater than the ULN, the medical monitor can determine if a protocol exception can be made. If total bilirubin increases to 1.5 x the ULN or more during study, the participant will be discontinued.
. Participants with significant renal insufficiency, with an estimated glomerular filtration rate (eGFR) \< 30 mL/min (calculated using the Cockcroft-Gault formula or Pediatric GFR calculator or Bedside Schwartz).