Ganaxolone for TSC-Related Epilepsy
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.12345Why 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.13467
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
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Open-label Extension
Participants receive adjunctive GNX treatment in an open-label extension study
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Ganaxolone
- GNX oral suspension
Find a Clinic Near You
Who Is Running the Clinical Trial?
Marinus Pharmaceuticals
Lead Sponsor