Trial · NCT03572933

Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder

NCT03572933, Phase 3, Completed, Marinus Pharmaceuticals; results first posted 2022-07-26

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder. Trials, record NCT03572933. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03572933

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder
NCT number: NCT03572933
Status: Completed
Phases: Phase 3
Enrollment: 101 participants, actual
Lead sponsor: Marinus Pharmaceuticals
Conditions: CDKL5 Deficiency Disorder
Interventions: ganaxolone, Placebo
First posted: 2018-06-28
Last updated: 2023-04-14
Other registries' IDs: eudract:2018-001180-23
Results first posted: 2022-07-26

Source ClinicalTrials.gov

Registered outcomes8

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Summary of 28-day Seizure Frequency for Major Motor Seizure Types	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.
Secondary	Caregiver Global Impression of Change in Attention	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
Secondary	Caregiver Global Impression of Change in Target Behavior	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.
Secondary	Clinical Global Impression of Improvement - Parent/Caregiver	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.
Secondary	Clinical Global Impression of Improvement - Clinician	[Time Frame: 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
Secondary	Percentage of Seizure-free Days for Major Motor Seizure Types	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.
Secondary	Arithmetic Change in Longest Seizure Free Interval, Based on Primary Seizure Types	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
Secondary	Caregiver Global Impression of Change in Seizure Intensity and Duration	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.

Source ClinicalTrials.gov

Outcomes8

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Summary of 28-day Seizure Frequency for Major Motor Seizure Types	End of the double-blind 17 week treatment period	ITT Population	Seizures per day	MEDIAN
SECONDARY	Caregiver Global Impression of Change in Attention	End of the double-blind 17 week treatment period	Per Protocol Population	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Caregiver Global Impression of Change in Target Behavior	End of the double-blind 17 week treatment period	Intent to Treat Population	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Clinical Global Impression of Improvement - Parent/Caregiver	End of the double-blind 17 week treatment period	Intent to Treat Population	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Clinical Global Impression of Improvement - Clinician	[Time Frame: End of the double-blind 17 week treatment period]	ITT Population	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Percentage of Seizure-free Days for Major Motor Seizure Types	End of the double-blind 17 week treatment period	ITT Population	percent of seizure-free days	MEAN
SECONDARY	Arithmetic Change in Longest Seizure Free Interval, Based on Primary Seizure Types	End of the double-blind 17 week treatment period	Intent to Treat Population	days	MEAN
SECONDARY	Caregiver Global Impression of Change in Seizure Intensity and Duration	End of the double-blind 17 week treatment period	Intent to Treat Population	Participants	COUNT_OF_PARTICIPANTS

Source ClinicalTrials.gov

Results by group82

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Summary of 28-day Seizure Frequency for Major Motor Seizure Types	Placebo	Baseline (Median)	49.17	95% Confidence Interval 32.20 to 60.67	Seizures per day
Summary of 28-day Seizure Frequency for Major Motor Seizure Types	Ganaxolone	Baseline (Median)	54.00	95% Confidence Interval 38.24 to 106.67	Seizures per day
Summary of 28-day Seizure Frequency for Major Motor Seizure Types	Placebo	17 week-post baseline phase (Median)	55.50	95% Confidence Interval 35.75 to 80.14	Seizures per day
Summary of 28-day Seizure Frequency for Major Motor Seizure Types	Ganaxolone	17 week-post baseline phase (Median)	45.03	95% Confidence Interval 31.83 to 76.03	Seizures per day
Caregiver Global Impression of Change in Attention	Placebo	Very Much Improved - Visit 5 (End of Week 17)	1		Participants
Caregiver Global Impression of Change in Attention	Ganaxolone	Very Much Improved - Visit 5 (End of Week 17)	1		Participants
Caregiver Global Impression of Change in Attention	Placebo	Much Improved - Visit 5 (End of Week 17)	7		Participants
Caregiver Global Impression of Change in Attention	Ganaxolone	Much Improved - Visit 5 (End of Week 17)	2		Participants
Caregiver Global Impression of Change in Attention	Placebo	Minimally Improved - Visit 5 (End of Week 17)	14		Participants
Caregiver Global Impression of Change in Attention	Ganaxolone	Minimally Improved - Visit 5 (End of Week 17)	21		Participants

1 to 10 of 82Source ClinicalTrials.gov

See all 82 results by group

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Placebo	51
Overall Study	STARTED	Ganaxolone	50
Overall Study	COMPLETED	Placebo	47
Overall Study	COMPLETED	Ganaxolone	48
Overall Study	NOT COMPLETED	Placebo	4
Overall Study	NOT COMPLETED	Ganaxolone	2

Source ClinicalTrials.gov

Adverse events94

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Somnolence	Nervous system disorders	Placebo	8	51	8
other	Somnolence	Nervous system disorders	Ganaxolone	18	50	20
other	Seizure	Nervous system disorders	Placebo	9	51	12
other	Seizure	Nervous system disorders	Ganaxolone	7	50	8
other	Sedation	Nervous system disorders	Placebo	2	51	2
other	Sedation	Nervous system disorders	Ganaxolone	3	50	3
other	Hypersomnia	Nervous system disorders	Placebo	0	51	0
other	Hypersomnia	Nervous system disorders	Ganaxolone	2	50	2
other	Lethargy	Nervous system disorders	Placebo	2	51	2
other	Lethargy	Nervous system disorders	Ganaxolone	2	50	2

1 to 10 of 94Source ClinicalTrials.gov

See all 94 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Placebo	0	51
Total, all-cause mortality	Ganaxolone	0	50
Total, other adverse events	Placebo	45	51
Total, other adverse events	Ganaxolone	43	50
Total, serious adverse events	Placebo	5	51
Total, serious adverse events	Ganaxolone	6	50

Source ClinicalTrials.gov

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
35429480	DERIVED	PubMed
36517284	DERIVED	PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Ganaxolone vs Placebo: deaths from any cause	Trial comparisons
Ganaxolone vs Placebo: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT03572933

GET https://conductscience.org/api/trials/v1/NCT03572933/results?page=2

API reference For AI assistants