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 range6 Months – 2 Years

SexALL

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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.
✓. 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:

What they're measuring

Percent Change from baseline in 28-day frequency of countable seizures

Timeframe: Baseline through 12 weeks

Trial details

NCT IDNCT05249556

SponsorImmedica Pharma AB

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-09

Contact for this trial

Clinical Project Manager

+46 8 533 39 500 clinical@immedica.com

View on ClinicalTrials.gov More CDKL5 Deficiency Disorder trials

Plain-language summary

Who can participate

Age range6 Months – 2 Years

SexALL

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.
✓. 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:

Double-blind, Randomized, Placebo-controlled Trial of Ganaxolone in CDKL5 Deficiency Patients 6 Months to Less Than 2 Years Old

Plain-language summary

Who can participate

Inclusion criteria

What they're measuring

Trial details

Contact for this trial

Double-blind, Randomized, Placebo-controlled Trial of Ganaxolone in CDKL5 Deficiency Patients 6 Months to Less Than 2 Years Old

Plain-language summary

Who can participate

Inclusion criteria

What they're measuring

Trial details

Contact for this trial

Exclusion criteria