Adults 2 to 21, any sex, with CDKL5 Deficiency Disorder. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Summary of 28-day Seizure Frequency for Major Motor Seizure TypesPrimary· End of the double-blind 17 week treatment period
Summary of 28-day seizure frequency for Major Motor Seizure Types during the double-blind treatment period relative to the 6-week prospective baseline period
Note: Summaries are based on the sum of the individual seizures, the countable seizures, and the clusters with uncountable seizures (each cluster with uncountable seizures counts as 1 seizure). Within the baseline and post baseline intervals, 28-day seizure frequency was calculated as the total number of seizures in the interval divided by the number of days with available seizure data in the interval, multiplied by 28.
Baseline (Median)
Group
Value
95% CI
Placebo
49.17
32.20 – 60.67
Ganaxolone
54.00
38.24 – 106.67
17 week-post baseline phase (Median)
Group
Value
95% CI
Placebo
55.50
35.75 – 80.14
Ganaxolone
45.03
31.83 – 76.03
Caregiver Global Impression of Change in AttentionSecondary· End of the double-blind 17 week treatment period
Caregiver global impression of change in attention during the double-blind treatment period of ganaxolone compared to placebo. Investigators and caregivers reported improvements in attention, mood, behavior and sleep via investigator narratives
Very Much Improved - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
1
Ganaxolone
1
Much Improved - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
7
Ganaxolone
2
Minimally Improved - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
14
Ganaxolone
21
No Change - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
23
Ganaxolone
18
Minimally Worse - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
1
Ganaxolone
1
Much Worse - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
1
Ganaxolone
1
Very Much Worse - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
0
Ganaxolone
1
Caregiver Global Impression of Change in Target BehaviorSecondary· End of the double-blind 17 week treatment period
Caregiver global impression of change in target behavior during the double-blind treatment period of ganaxolone compared to placebo. Investigators and caregivers reported improvements in attention, mood, behavior and sleep via investigator narratives.
Very Much Improved - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
0
Ganaxolone
0
Much Improved - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
6
Ganaxolone
4
Minimally Improved - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
14
Ganaxolone
20
No Change - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
22
Ganaxolone
19
Minimally Worse - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
1
Ganaxolone
2
Much Worse - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
2
Ganaxolone
0
Very Much Worse - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
1
Ganaxolone
0
Clinical Global Impression of Improvement - Parent/CaregiverSecondary· End of the double-blind 17 week treatment period
Clinical global impression of improvement during the double-blind treatment period of ganaxolone compared to placebo. The CGI is rated on a 7-point scale, with the severity of illness scale using a range of responses.
Very Much Improved - Visit 5 (End of Week 17) - Parent/Caregiver
Group
Value
95% CI
Placebo
1
Ganaxolone
0
Much Improved - Visit 5 (End of Week 17) - Parent/Caregiver
Very Much Worse - Visit 5 (End of Week 17) - Clinician
Group
Value
95% CI
Placebo
0
Ganaxolone
1
Percentage of Seizure-free Days for Major Motor Seizure TypesSecondary· End of the double-blind 17 week treatment period
Percentage of Seizure-free Days for Major Motor Seizure types during the double-blind treatment period of ganaxolone compared to placebo. The major motor seizure types include bilateral tonic (sustained motor activity = 3 seconds), generalized tonic-clonic, atonic/drop, bilateral clonic, and focal to bilateral tonic-clonic.
Baseline
Group
Value
95% CI
Placebo
30.32
± 27.070
Ganaxolone
22.57
± 25.761
17-week-Post-Baseline Phase
Group
Value
95% CI
Placebo
36.17
± 30.932
Ganaxolone
32.29
± 30.615
Arithmetic Change from Baseline
Group
Value
95% CI
Placebo
5.86
± 15.350
Ganaxolone
9.62
± 21.364
Arithmetic Change in Longest Seizure Free Interval, Based on Primary Seizure TypesSecondary· End of the double-blind 17 week treatment period
Arithmetic change in longest seizure free interval, based on primary seizure types during the double-blind treatment period of ganaxolone compared to placebo
Group
Value
95% CI
Placebo
-4.63
± 14.867
Ganaxolone
-0.02
± 9.376
Caregiver Global Impression of Change in Seizure Intensity and DurationSecondary· End of the double-blind 17 week treatment period
Caregiver global impression of change in seizure intensity and duration during the double-blind treatment period of ganaxolone compared to placebo. CGI-C is a 7 point scale that requires the clinician to assess how much the patient's illness has improved or worsened relative to a baseline state at the beginning of the intervention.
Very Much Improved - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
1
Ganaxolone
2
Much Improved - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
5
Ganaxolone
15
Minimally Improved - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
11
Ganaxolone
11
No Change - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
21
Ganaxolone
10
4Minimally Worse - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
5
Ganaxolone
3
Much Worse - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
4
Ganaxolone
2
Very Much Worse - Visit 5 (End of Week 17)
Group
Value
95% CI
Placebo
0
Ganaxolone
2
Adverse events — posted to ClinicalTrials.gov
Time frame: Screening through 17-week Double-blind Phase.
Reporting threshold: 3%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
A clinical study to evaluate the efficacy, safety, and tolerability of adjunctive ganaxolone therapy compared to placebo for the treatment of seizures in children and young adults with genetically confirmed CDKL5 gene mutation.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
Other recruiting trials for CDKL5 Deficiency Disorder
Currently open trials in the same condition.
NCT05064878 — A Study to Investigate the Efficacy and Safety of ZX008 in Subjects With CDKL5 Deficiency Disorder
· Phase 3
· active not recruiting
NCT05558371 — International CDKL5 Clinical Research Network
· recruiting
Other Marinus Pharmaceuticals trials
Trials by the same sponsor.
NCT05604170 — Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy
· Phase 3
· terminated
NCT05323734 — Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy
· Phase 3
· completed
NCT04391569 — Randomized Therapy In Status Epilepticus
· Phase 3
· completed
NCT04285346 — Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)
· Phase 2
· completed
NCT03865732 — Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Stud
· Phase 2
· completed
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Marinus Pharmaceuticals
Last refreshed: 14 April 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03572933.