NBI-921352 for Developmental and Epileptic Encephalopathy Syndrome

Recruiting at 3 trial locations

Overseen By Neurocrine Medical Information Call Center

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Neurocrine Biosciences

Must be taking: Antiseizure medications

Must not be taking: Cannabinoids, Systemic steroids

Disqualifiers: Dravet syndrome, Head trauma, 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)

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

Compensation: Varies

Number of Visits: Unknown

Duration: 164 weeks

52 Participants Needed

Trial Summary

What is the purpose of this trial?

Extension study to evaluate how safe and tolerable the drug NBI-921352 is when used as adjunctive therapy in participants with SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE).

Will I have to stop taking my current medications?

The trial does not require you to stop taking your current antiseizure medications, but you cannot be on more than four. If you are taking systemic steroids or certain cannabinoids, you may need to stop them before joining the study.

What data supports the effectiveness of the drug NBI-921352 for treating Developmental and Epileptic Encephalopathy Syndrome?

Research on similar drugs, like quinidine, shows that targeting specific genetic mutations in epilepsy can reduce seizures significantly, although results can vary. This suggests that NBI-921352, which may work in a similar way, could potentially be effective for certain genetic forms of epilepsy. ^{1 2 3 4 5}

Research Team

Clinical Development Lead

Principal Investigator

Neurocrine Biosciences

Eligibility Criteria

This trial is for individuals with SCN8A-DEE, a severe form of epilepsy. Participants can be those who completed the prior NBI-921352 study or new patients aged 2 to 21, averaging at least one seizure per week and not seizure-free for over 20 days. They must be on 1-4 other antiseizure meds and have failed two others.

Inclusion Criteria

I, or my guardian, have given consent for me to participate.

I, or my guardian, have given consent for me to participate.

I still have seizures despite trying at least 2 seizure medications.

Exclusion Criteria

My epilepsy symptoms don't match those of Dravet syndrome.

I am using medical marijuana or cannabinoids, not including Epidiolex/Epidyolex, with sponsor approval.

Have a history of moderate or severe head trauma or other neurological disorders or systemic medical diseases that are, in the investigator's opinion, likely to affect nervous system functioning.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive NBI-921352 as adjunctive therapy

164 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• NBI-921352

Trial Overview The safety and tolerability of NBI-921352 as an additional treatment are being tested in this extension study for people with SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE).

Participant Groups

Group I: NBI-921352 Experimental Treatment 1 Intervention

NBI-921352 administered for up to 164 weeks.

Who Is Running the Clinical Trial?

Neurocrine Biosciences

Lead Sponsor

Trials

78

Recruited

6,600+

Kyle W. Gano

Neurocrine Biosciences

Chief Executive Officer since 2024

PhD in Pharmacology

Dr. Sanjay Keswani

Neurocrine Biosciences

Chief Medical Officer

MD

References

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

De novo gain-of-function variants in KCNT2 as a novel cause of developmental and epileptic encephalopathy. [2019]

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

Early Treatment with Quinidine in 2 Patients with Epilepsy of Infancy with Migrating Focal Seizures (EIMFS) Due to Gain-of-Function KCNT1 Mutations: Functional Studies, Clinical Responses, and Critical Issues for Personalized Therapy. [2019]

3. Netherlands pubmed.ncbi.nlm.nih.gov

Efficacy of sodium channel blockers in SCN2A early infantile epileptic encephalopathy. [2022]

4. Switzerland pubmed.ncbi.nlm.nih.gov

Emerging evidence of genotype-phenotype associations of developmental and epileptic encephalopathy due to KCNC2 mutation: Identification of novel R405G. [2022]

5. England pubmed.ncbi.nlm.nih.gov

Intolerance to quinidine in a n-of-1 trial for KCNT1 associated epilepsy of infancy with migrating focal seizures. [2022]