NCT02358538 is a Phase 2 clinical trial of Ganaxolone in Epilepsy, sponsored by Marinus Pharmaceuticals.

Who is sponsoring NCT02358538?

NCT02358538 is sponsored by Marinus Pharmaceuticals.

What drugs are being tested in NCT02358538?

Interventions in NCT02358538: Ganaxolone.

Is NCT02358538 still recruiting?

NCT02358538 is currently completed. Last updated 21 March 2023.

Are results published for NCT02358538?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2023-03-21 · How we verify

NCT02358538

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Open-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies

Completed Phase 2 Results posted Last updated 21 March 2023

What this trial tests

Phase 2 trial testing Ganaxolone in Epilepsy in 30 participants. Completed in 4 January 2019.

Timeline

6 November 2015

Primary endpoint
16 January 2018

4 January 2019

Quick facts

Lead sponsor	Marinus Pharmaceuticals
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	prevention
Enrollment	30
Start date	6 November 2015
Primary completion	16 January 2018
Estimated completion	4 January 2019
Sites	10 locations across Italy, United States

Drugs / interventions tested

Ganaxolone (GANAXOLONE) — full drug profile →

Conditions studied

Epilepsy — all drugs for Epilepsy →

Sponsor

Marinus Pharmaceuticals — full company profile →

Who can join

Adults 2 to 18, any sex, with Epilepsy. 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 Sum of Individual Seizures and Clusters for 52-week OLE Period (Mean Percent Change & Standard Deviation) Primary · Baseline through 52 week open label period

Percentage change from baseline in 28-day seizure frequency at 3 months (day 91), 26 weeks, 52 week OLE (Mean Percent Change \& Standard Deviation)

Percent Change from baseline (at day 91)

Group	Value	95% CI
CDKL5	-31.23	± 41.438
CSWS	NA	± NA
Lennox- Gastaut	122.10	± 321.124
PCDH19	52.83	± 234.084

Percent Change from baseline (at week 26)

Group	Value	95% CI
CDKL5	-20.55	± 60.588
CSWS	NA	± NA
Lennox- Gastaut	125.38	± 319.051
PCDH19	46.36	± 235.661

Percent change from baseline (52 week OLE through month 6)

Group	Value	95% CI
CDKL5	-54.41	± 40.286
CSWS	NA	± NA
Lennox- Gastaut	-38.74	± 9.292
PCDH19	-19.98	± 63.644

Percent change from baseline (52 week OLE period)

Group	Value	95% CI
CDKL5	-49.20	± 50.206
CSWS	NA	± NA
Lennox- Gastaut	-37.75	± 7.891
PCDH19	-19.95	± 63.571

Summary of 28-day Seizure Frequency for Sum of Individual Seizures and Clusters Through 52-week OLE (Median Percent Change) Primary · Baseline through 52-week open- label period

Percentage change from baseline in 28-day seizure frequency at 3 months (day 91), 26 weeks, 52 week OLE (Median Percent Change)

Percent change from baseline (at Day 91)

Group	Value	95% CI
CDKL5	-47.34	-80.9 – 36.8
Lennox-Gastaut	-10.22	-68.1 – 904.3
PCDH19	-25.98	-100.00 – 723.2
CSWS	NA	NA – NA

Percent change from baseline (at week 26)

Group	Value	95% CI
CDKL5	-37.70	-85.3 – 99.9
Lennox-Gastaut	-9.19	-71.2 – 904.3
PCDH19	-24.59	-100.00 – 723.2
CSWS	NA	NA – NA

Percent change from baseline (at week 26) - with 6 duplicate diary entries removed

Group	Value	95% CI
CDKL5	-44.4	-85.3 – 99.9
Lennox-Gastaut	NA	NA – NA
PCDH19	NA	NA – NA
CSWS	NA	NA – NA

Percent change from baseline (52 week OLE through month 6)

Group	Value	95% CI
CDKL5	-58.94	-89.4 – -10.4
Lennox-Gastaut	-38.74	-45.3 – -32.2
PCDH19	-13.48	-100.0 – 59.6
CSWS	NA	NA – NA

Percent change from baseline (52 week OLE period)

Group	Value	95% CI
CDKL5	-61.93	-89.5 – 16.6
Lennox-Gastaut	-37.75	-43.3 – -32.2
PCDH19	-13.48	-99.0 – 59.6
CSWS	NA	NA – NA

Summary of CGII-C Secondary · End of Week 4, End of Week 8, End of Week 17, End of Week 26, Week 44, Week 62, Week 78

Clinician Global Impression of Change score as assessed by questionnaire. \[ Time Frame: 78 Weeks \] CGII-C scale is qualitative values and not quantitative.

Visit 4 (End of Week 4) - Very Much Improved

Group	Value	95% CI
CDKL5	0
Lennox-Gastaut	2
PCDH19	1
CSWS	NA

Visit 4 (End of Week 4) - Much Improved

Group	Value	95% CI
CDKL5	3
Lennox-Gastaut	4
PCDH19	4
CSWS	NA

Visit 4 (End of Week 4) - Minimally Improved

Group	Value	95% CI
CDKL5	2
Lennox-Gastaut	2
PCDH19	3
CSWS	NA

Visit 4 (End of Week 4) - No Change

Group	Value	95% CI
CDKL5	2
Lennox-Gastaut	0
PCDH19	3
CSWS	NA

Visit 4 (End of Week 4) - Minimally Worse

Group	Value	95% CI
CDKL5	0
Lennox-Gastaut	0
PCDH19	0
CSWS	NA

Visit 4 (End of Week 4) - Much Worse

Group	Value	95% CI
CDKL5	0
Lennox-Gastaut	0
PCDH19	0
CSWS	NA

Visit 4 (End of Week 4) - Very Much Worse

Group	Value	95% CI
CDKL5	0
Lennox-Gastaut	0
PCDH19	0
CSWS	NA

Visit 5 (End of Week 8) - Very Much Improved

Group	Value	95% CI
CDKL5	0
Lennox-Gastaut	1
PCDH19	0
CSWS	NA

Summary of CGII-P Secondary · Patient Global Impression of Change score as assessed by questionnaire. [ Time Frame: 78 Weeks ]

Patient Global Impression of Change score as assessed by questionnaire. \[ Time Frame: 78 Weeks \] CGII-P scale is qualitative values and not quantitative.

Visit 4 (End of Week 4) - Very Much Improved

Group	Value	95% CI
CDKL5	0
Lennox-Gastaut	3
PCDH19	1
CSWS	NA

Visit 4 (End of Week 4) - Much Improved

Group	Value	95% CI
CDKL5	1
Lennox-Gastaut	2
PCDH19	2
CSWS	NA

Visit 4 (End of Week 4) - Minimally Improved

Group	Value	95% CI
CDKL5	4
Lennox-Gastaut	3
PCDH19	5
CSWS	NA

Visit 4 (End of Week 4) - No Change

Group	Value	95% CI
CDKL5	2
Lennox-Gastaut	0
PCDH19	3
CSWS	NA

Visit 4 (End of Week 4) - Minimally Worse

Group	Value	95% CI
CDKL5	0
Lennox-Gastaut	0
PCDH19	0
CSWS	NA

Visit 4 (End of Week 4) - Much Worse

Group	Value	95% CI
CDKL5	0
Lennox-Gastaut	0
PCDH19	0
CSWS	NA

Visit 4 (End of Week 4) - Very Much Worse

Group	Value	95% CI
CDKL5	0
Lennox-Gastaut	0
PCDH19	0
CSWS	NA

Visit 5 (End of Week 8) - Very Much Improved

Group	Value	95% CI
CDKL5	0
Lennox-Gastaut	2
PCDH19	0
CSWS	NA

Number of Participants With Responder Rate of Seizure Frequency Secondary · Month 3 and Week 26

Responder Rate in Terms of 28-day Seizure Frequency Based on the Sum of Individual Seizures and Clusters

Post-baseline through Month 3 (Day 91) - 25% Responder

Group	Value	95% CI
CDKL5	4
Lennox-Gastaut	4
PCDH19	6
CSWS	NA

Post-baseline through Month 3 (Day 91) - 50% Responder

Group	Value	95% CI
CDKL5	3
Lennox-Gastaut	2
PCDH19	4
CSWS	NA

Post-baseline through Month 3 (Day 91) - 75% Responder

Group	Value	95% CI
CDKL5	1
Lennox-Gastaut	0
PCDH19	1
CSWS	NA

Post-baseline 26-week open-label Period - 25% Responder

Group	Value	95% CI
CDKL5	4
Lennox-Gastaut	3
PCDH19	5
CSWS	NA

Post-baseline 26-week open-label Period - 50% Responder

Group	Value	95% CI
CDKL5	2
Lennox-Gastaut	1
PCDH19	3
CSWS	NA

Post-baseline 26-week open-label Period - 75% Responder

Group	Value	95% CI
CDKL5	1
Lennox-Gastaut	0
PCDH19	1
CSWS	NA

Mean Percentage Change of Individual Seizure-free Days Secondary · Baseline, Day 91, Week 26, 52-week OLE through month 6, 52-week OLE Period

Mean Percentage Change of Individual Seizure-free days per 28-day period (through 52-week OLE) period relative to baseline

Mean Percentage Change of individual seizure-free days from baseline to Day 91

Group	Value	95% CI
CDKL5	11.84	± 18.472
Lennox-Gastaut	-1.12	± 24.928
PCDH19	7.87	± 17.843
CSWS	NA	± NA

Mean Percentage Change of individual seizure-free days from baseline to Week 26

Group	Value	95% CI
CDKL5	11.80	± 20.968
Lennox-Gastaut	-2.13	± 22.042
PCDH19	7.94	± 18.016
CSWS	NA	± NA

Mean Percentage Change of individual seizure-free days from baseline to 52-week OLE (181 days)

Group	Value	95% CI
CDKL5	21	± 30.85
Lennox-Gastaut	16.35	± 2.916
PCDH19	17.12	± 27.040
CSWS	NA	± NA

Mean Percentage Change of individual seizure-free days from baseline to 52-week OLE Period

Group	Value	95% CI
CDKL5	20.44	± 28.788
Lennox-Gastaut	14.95	± 4.897
PCDH19	17.02	± 26.904
CSWS	NA	± NA

Adverse events — posted to ClinicalTrials.gov

Time frame: Combined 26-week open label period and 52-week open label extension period. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

CDKL5

Serious: 1/7 (14%)

Deaths: 0/7

CSWS

Serious: 1/2 (50%)

Deaths: 0/2

Lennox-Gastaut

Serious: 2/10 (20%)

Deaths: 0/10

PCDH19

Serious: 3/11 (27%)

Deaths: 0/11

Serious adverse events (8 terms)

Reaction	System	CDKL5	CSWS	Lennox-Gastaut	PCDH19
Seizure	Nervous system disorders	—	—	—	—
Hepatic failure	Hepatobiliary disorders	—	—	—	—
Mental status changes	Psychiatric disorders	—	—	—	—
Pneumonia	Infections and infestations	—	—	—	—
Rash	Skin and subcutaneous tissue disorders	—	—	—	—
Somnolence	Nervous system disorders	—	—	—	—
Pneumonia Viral	Infections and infestations	—	—	—	—
Unintentional Medical Device Removal	General disorders	—	—	—	—

Other adverse events (32 terms — click to expand)

Reaction	System	CDKL5	CSWS	Lennox-Gastaut	PCDH19
Pyrexia	General disorders	—	—	—	—
Somnolence	Nervous system disorders	—	—	—	—
Seizure	Nervous system disorders	—	—	—	—
Headache	Nervous system disorders	—	—	—	—
Fatigue	General disorders	—	—	—	—
Upper respiratory tract infection	Infections and infestations	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—
Decreased appetite	Metabolism and nutrition disorders	—	—	—	—
Fall	Injury, poisoning and procedural complications	—	—	—	—
Hypersomnia	Nervous system disorders	—	—	—	—
Sedation	Nervous system disorders	—	—	—	—
Nasopharyngitis	Infections and infestations	—	—	—	—
Influenza	Infections and infestations	—	—	—	—
Otitis media	Infections and infestations	—	—	—	—
Abdominal pain upper	Gastrointestinal disorders	—	—	—	—
Emotional disorder	Psychiatric disorders	—	—	—	—
Restlessness	Psychiatric disorders	—	—	—	—
Ataxia	Nervous system disorders	—	—	—	—
Fungal Infection	Infections and infestations	—	—	—	—
Cough	Respiratory, thoracic and mediastinal disorders	—	—	—	—
Enuresis	Renal and urinary disorders	—	—	—	—
Gastrostomy	Surgical and medical procedures	—	—	—	—
Dizziness	Nervous system disorders	—	—	—	—
Rhinitis	Infections and infestations	—	—	—	—
Abnormal behavior	Psychiatric disorders	—	—	—	—
Weight decreased	Investigations	—	—	—	—
Rash	Skin and subcutaneous tissue disorders	—	—	—	—
Contusion	Injury, poisoning and procedural complications	—	—	—	—
Balance Disorder	Nervous system disorders	—	—	—	—
Pneumonia	Infections and infestations	—	—	—	—
Sinusitis	Infections and infestations	—	—	—	—
Alopecia	Skin and subcutaneous tissue disorders	—	—	—	—

Most-reported serious reactions: Seizure, Hepatic failure, Mental status changes, Pneumonia, Rash, Somnolence, Pneumonia Viral, Unintentional Medical Device Removal.

Data from ClinicalTrials.gov NCT02358538 adverse events section.

Sponsor's own description

To evaluate the efficacy of open-label ganaxolone as adjunctive therapy for uncontrolled seizures in female children with PCDH19 mutation and other rare genetic epilepsies in an open-label proof-of-concept study.

Publications & conference data

7 peer-reviewed publications reference this trial (live from Europe PMC):

Clinical Potential of Neurosteroids for CNS Disorders.
Reddy DS, Estes WA. · · 2016 · cited 146× · PMID 27156439 · DOI 10.1016/j.tips.2016.04.003
CDKL5 deficiency disorder: clinical features, diagnosis, and management.
Leonard H, Downs J, Benke TA, Swanson L, et al · · 2022 · cited 106× · PMID 35483386 · DOI 10.1016/s1474-4422(22)00035-7
Evaluation of the neuroactive steroid ganaxolone on social and repetitive behaviors in the BTBR mouse model of autism.
Kazdoba TM, Hagerman RJ, Zolkowska D, Rogawski MA, et al · · 2016 · cited 44× · PMID 26525567 · DOI 10.1007/s00213-015-4115-7
Ganaxolone: A New Treatment for Neonatal Seizures.
Yawno T, Miller SL, Bennet L, Wong F, et al · · 2017 · cited 31× · PMID 28878622 · DOI 10.3389/fncel.2017.00246
Effects of ganaxolone on non-seizure outcomes in CDKL5 Deficiency Disorder: Double-blind placebo-controlled randomized trial.
Downs J, Jacoby P, Specchio N, Cross H, et al · · 2024 · cited 14× · PMID 38959712 · DOI 10.1016/j.ejpn.2024.06.005
Abstracts of 20th Annual ASENT Meeting.
· 2018 · cited 2× · PMID 29987760 · DOI 10.1007/s13311-018-0641-4
Novel Medication Ganaxolone for Seizure Disorders.
Chowdhury S, Chowdhury S. · · 2025 · cited 1× · PMID 40787618 · DOI 10.15190/d.2025.7

Verify or expand the search:

Other trials of Ganaxolone

Trials testing the same drug.

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

Other recruiting trials for Epilepsy

Currently open trials in the same condition.

NCT07095933 — The Safety and Efficacy Evaluation of Everolimus as an Adjunctive Treatment for Focal Refractory Epilepsy · EARLY_PHASE1 · recruiting
NCT07224191 — Hippocampal Oscillations During Exploration · NA · recruiting
NCT07219407 — A Long-term Study of the Safety and Effectiveness of RAP-219 in Adults With Focal Onset Seizures · Phase 2 · recruiting
NCT07417280 — LIFUS For Neurological Disorders · NA · recruiting
NCT07490769 — Levetiracetam Three Times Daily in Epilepsy · Phase 3 · 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

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT02358538 (US National Library of Medicine, public domain)
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: 21 March 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/NCT02358538.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing