Ganaxolone for TSC-Related Epilepsy

Recruiting at 110 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

132 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a Phase 3, global, open-label extension (OLE) study of adjunctive GNX treatment in children and adults with TSC who previously participated in either Study 1042-TSC-3001 or Study 1042-TSC-2001

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 Ganaxolone generally safe for human use?

Ganaxolone has shown good safety in treating status epilepticus (a severe type of seizure) and could be important in future treatments, indicating it is generally safe for human use. ^{1 2 3 4 5}

How does the drug Ganaxolone differ from other treatments for TSC-related epilepsy?

Ganaxolone is unique because it is a synthetic neuroactive steroid that modulates the GABA(A) receptor in a different way than traditional drugs like benzodiazepines, offering a novel approach to treating epilepsy. It has shown effectiveness in reducing seizures in various epilepsy models and is being studied for its potential in treating TSC-related epilepsy, with a favorable safety profile. ^{6 7 8 9 10}

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

References

1. Argentina pubmed.ncbi.nlm.nih.gov

[New antiepileptic drugs]. [2020]

2. United States pubmed.ncbi.nlm.nih.gov

Human safety of lamotrigine. [2019]

3. United States pubmed.ncbi.nlm.nih.gov

Efficacy and safety of antiepileptic drugs: a review of controlled trials. [2019]

4. Netherlands pubmed.ncbi.nlm.nih.gov

Tolerability of adjunctive eslicarbazepine acetate according to concomitant lamotrigine or carbamazepine use: A subgroup analysis of three phase III trials in adults with focal (partial-onset) seizures. [2019]

5. United States pubmed.ncbi.nlm.nih.gov

Adverse reactions to antiepileptic drugs: a multicenter survey of clinical practice. Collaborative Group for Epidemiology of Epilepsy. [2019]

6. United States pubmed.ncbi.nlm.nih.gov

Intravenously Administered Ganaxolone Blocks Diazepam-Resistant Lithium-Pilocarpine-Induced Status Epilepticus in Rats: Comparison with Allopregnanolone. [2019]

7. United States pubmed.ncbi.nlm.nih.gov

Ganaxolone. [2020]

8. United States pubmed.ncbi.nlm.nih.gov

Intravenous ganaxolone in pediatric super-refractory status epilepticus: A single hospital experience. [2022]

9. New Zealand pubmed.ncbi.nlm.nih.gov

Ganaxolone: First Approval. [2022]

10. England pubmed.ncbi.nlm.nih.gov

Ganaxolone: a novel positive allosteric modulator of the GABA(A) receptor complex for the treatment of epilepsy. [2019]