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Ganaxolone for TSC-Related Epilepsy

Phase 3
Waitlist Available
Research Sponsored by Marinus Pharmaceuticals
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline (day 1) and week 1 through week 156
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial studies an adjunctive treatment in children & adults with Tuberous Sclerosis Complex (TSC) who participated in previous studies.

Who is the study for?
This trial is for children and adults with TSC-related epilepsy who have been part of previous studies (1042-TSC-3001 or 1042-TSC-2001). Participants must be able to maintain a seizure diary, take the study drug with food three times daily, use effective contraception if applicable, and not be pregnant or breastfeeding. They should not have other health conditions that could affect the study.
What is being tested?
The trial is testing Ganaxolone as an additional treatment in those with Tuberous Sclerosis Complex (TSC) related epilepsy. It's an open-label extension meaning everyone knows they're getting Ganaxolone, which was previously studied in two earlier trials.
What are the potential side effects?
While specific side effects for this trial are not listed, Ganaxolone may cause dizziness, tiredness, headache and potentially impact hormonal balance due to its nature as a steroid compound.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline (day 1) and week 1 through week 156
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline (day 1) and week 1 through week 156 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of participants with abnormal Columbia-Suicide Severity Rating Scale (C-SSRS)
Secondary study objectives
Change from Baseline in Caregiver Global Impression of Change in Seizure Intensity/Duration (CGI-CSID)
Change from Baseline in quality-of-life scale Short Form-36 (SF-36)
Number of Participants with Clinical Global Impression of Improvement (CGI-I)
+2 more

Side effects data

From 2014 Phase 2 trial • 112 Patients • NCT01339689
19%
Somnolence
14%
Headache
8%
Fatigue
8%
Sedation
7%
Dizziness
7%
Nightmare
7%
Vision blurred
7%
Myalgia
5%
Confusional state
5%
Dysarthria
3%
Pain in extremity
3%
Vertigo
3%
Gastrointestinal disorder
3%
Tremor
3%
Neck pain
3%
Nasopharyngitis
3%
Increased appetite
3%
Ataxia
3%
Balance disorder
3%
Cough
3%
Insomnia
3%
Diarrhoea
3%
Lethargy
2%
Confusion
2%
Tandem galt test abnormal
2%
Dry mouth
2%
Splenomegaly
2%
Road traffic accident
2%
Urinary tract infection
2%
Arthralgia
2%
Balance Issues
2%
Pulmonary congestion
2%
Upper respiratory tract infection
2%
Rhinorrhoea
2%
Protein urine present
2%
Vomiting
2%
Chest pain
2%
Decreased appetite
2%
Abnormal dreams
2%
Nausea
2%
Chest discomfort
2%
Feeling drunk
2%
Irritability
2%
Pharyngitis streptococcal
2%
Blood urine present
2%
Weight increased
2%
Back pain
2%
Muscle spasms
2%
Depressed level of consciousness
2%
Disturbance in attention
2%
Motor dysfunction
2%
Anger
2%
Depressed
2%
Euphoric mood
2%
Initial insomnia
2%
Psychomotor retardation
2%
Sleep disorder
2%
Upper airway resistance syndrome
2%
Anorgasmia
2%
Suicidal ideation
2%
Haematuria
2%
Suicide of companion
2%
Product used for unknown indication
2%
Dry Eye
2%
Abdominal discomfort
2%
Alanine aminotransferase increased
2%
Liver function test abnormal
2%
Tic
2%
Migraine
2%
Suicidal Ideation
2%
Fever
2%
Sinus congestion
2%
Rash
100%
80%
60%
40%
20%
0%
Study treatment Arm
Ganaxolone
Placebo

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups
Experimental Treatment
Group I: Ganaxolone (GNX) oral suspension, 3 times a day (TID)Experimental Treatment1 Intervention
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ganaxolone
2011
Completed Phase 3
~810

Find a Location

Who is running the clinical trial?

Marinus PharmaceuticalsLead Sponsor
22 Previous Clinical Trials
1,641 Total Patients Enrolled
2 Trials studying Tuberous Sclerosis
151 Patients Enrolled for Tuberous Sclerosis

Media Library

Ganaxolone (GNX) oral suspension, 3 times a day (TID) Clinical Trial Eligibility Overview. Trial Name: NCT05604170 — Phase 3
Tuberous Sclerosis Research Study Groups: Ganaxolone (GNX) oral suspension, 3 times a day (TID)
Tuberous Sclerosis Clinical Trial 2023: Ganaxolone (GNX) oral suspension, 3 times a day (TID) Highlights & Side Effects. Trial Name: NCT05604170 — Phase 3
Ganaxolone (GNX) oral suspension, 3 times a day (TID) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05604170 — Phase 3
~66 spots leftby Jun 2027
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