Ganaxolone for TSC-Related Epilepsy

No longer recruiting at 117 trial locations

Age: Any Age

Sex: Any

Trial Phase: Phase 3

Sponsor: Marinus Pharmaceuticals

Disqualifiers: Pregnant, CNS infection, Suicidal, others

Stay on Your Current Meds You can continue your current medications while participating

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a medication called Ganaxolone, taken as an oral suspension, to help manage epilepsy related to Tuberous Sclerosis Complex (TSC). The goal is to evaluate how well Ganaxolone works when added to existing treatments for managing seizures in children and adults who participated in earlier related studies. Those who completed those studies and are willing to take the medication as directed, while maintaining a daily seizure diary, might be suitable candidates. As a Phase 3 trial, this is the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment for epilepsy.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, you should discuss your current medications with the study team to ensure they don't interfere with the trial.

Is there any evidence suggesting that Ganaxolone is likely to be safe for humans?

Research has shown that ganaxolone, the treatment under study for epilepsy related to TSC, has safety data from earlier trials. In one study, the most common side effect was drowsiness, occurring in 28.1% of participants taking ganaxolone, compared to 16.9% of those on a placebo. This indicates that while some experienced increased drowsiness, most did not encounter serious issues.

Ganaxolone has been tested previously for treating seizures, and some participants experienced fewer seizures. As this trial is in a later stage, more evidence suggests that the treatment is generally well-tolerated. However, individual experiences may vary. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard of care for TSC-related epilepsy, which often involves medications like vigabatrin or everolimus, Ganaxolone offers a unique approach. It works by modulating GABA-A receptors, which are crucial for calming overactive neurons in the brain. This novel mechanism helps to reduce seizures, potentially providing relief for patients who do not respond well to existing treatments. Researchers are excited because Ganaxolone could offer a more targeted and effective option with fewer side effects.

What evidence suggests that Ganaxolone might be an effective treatment for TSC-related epilepsy?

Research has shown that ganaxolone offers promising results for treating seizures in people with tuberous sclerosis complex (TSC). In earlier studies, ganaxolone reduced the number of seizures by 16% after just 28 days. Notably, about 30% of participants in those studies experienced at least a 50% reduction in seizures. This trial will evaluate ganaxolone, administered as an oral suspension three times a day, to determine its effectiveness in managing epilepsy related to TSC, offering hope for those seeking better seizure control. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I agree to use effective birth control during and for 30 days after the study.

Parent(s)/caregiver(s) willing and able to maintain an accurate and complete daily seizure diary

Willing and able to take Investigational product (IP) as directed with food TID

Exclusion Criteria

I haven't used any experimental drugs or devices recently.

I don't have any health issues that could affect how a drug works in my body.

Active suicidal plan/intent or history of suicidal thoughts or attempts in the past 6 months

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Open-label Extension

Participants receive adjunctive GNX treatment in an open-label extension study

160 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Ganaxolone
• GNX oral suspension

Trial Overview

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.

How Is the Trial Designed?

Group I: Ganaxolone (GNX) oral suspension, 3 times a day (TID) Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Marinus Pharmaceuticals

Lead Sponsor

Trials

23

Recruited

1,800+

Published Research Related to This Trial

Lamotrigine (LTG) was found to have a similar safety profile to placebo in a pooled analysis of four double-blind trials involving 92 patients, with only two patients withdrawing due to adverse effects.

In a larger group of 572 patients from open studies, common side effects included dizziness and headache, but no significant clinical changes or serious adverse events were linked to LTG, indicating it is generally safe when added to existing antiepileptic treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Human safety of lamotrigine. Betts, T., Goodwin, G., Withers, RM., et al.[2019]

Citations

1.

ir.marinuspharma.com

ir.marinuspharma.com/news/news-details/2024/Marinus-Pharmaceuticals-Announces-Topline-Results-From-Phase-3-TrustTSC-Trial-of-Oral-Ganaxolone-in-Tuberous-Sclerosis-Complex-and-Commences-Process-to-Explore-Strategic-Alternatives/default.aspx

News Details - Investor Relations - Marinus Pharmaceuticals

The Phase 3 TrustTSC trial evaluating oral ganaxolone for the treatment of seizures associated with tuberous sclerosis complex (TSC) in children and adults did ...

2.

tuberous-sclerosis.org

tuberous-sclerosis.org/ganalaxone-topline-results/

Ganaxolone shows positive phase 2 results in TSC-related ...

Ganaxolone's phase 2 trials showed a 16% reduction in seizure frequency after 28 days, with 30% of participants achieving at least a 50% seizure reduction.

3.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04285346

Adjunctive Ganaxolone Treatment (Part A) in TSC ...

To assess preliminary safety and efficacy of ganaxolone as adjunctive therapy for the treatment of primary seizure types in patients with genetically- or ...

4.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC10741146/

The Evolving Landscape of Therapeutics for Epilepsy in ...

This trial aims to evaluate the efficacy and safety of adjunctive ganaxolone compared to the placebo for reducing seizure frequency in TSC ...

5.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05323734

Study Details | NCT05323734 | Adjunctive GNX Treatment ...

This is a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive GNX treatment in children and adults with TSC-related epilepsy.

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05323734

Study Details | NCT05323734 | Adjunctive GNX Treatment ...

This is a Phase 3, global, double-blind, randomized, placebo-controlled study of adjunctive GNX treatment in children and adults with TSC-related epilepsy.

7.

tscalliance.org

tscalliance.org/wp-content/uploads/2023/04/TSC-Alliance-TrustTSC-Educational-Webinar-Clean-Slides-042523.pdf

TrustTSC Study: Enrolling children and adults with TSC

Ganaxolone is a positive allosteric GABAA receptor modulator with a well-defined MOA designed to treat patients suffering from seizure disorders ...