NBI-921352 for Developmental and Epileptic Encephalopathy Syndrome

Not currently recruiting at 4 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety and tolerability of the drug NBI-921352 (also known as XEN901 or Zandatrigine) for people with SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE). The study targets participants who continue to experience frequent seizures despite trying other seizure medications. It seeks individuals who have at least one countable motor seizure per week and have tried at least two other anti-seizure medications without success. Participants should also use at least one other seizure medication during the trial. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people, offering a chance to contribute to important findings.

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.

Is there any evidence suggesting that NBI-921352 is likely to be safe for humans?

Research has shown that NBI-921352 has generally been well-tolerated in earlier studies. This drug aims to help people with SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE), a type of epilepsy. In these studies, participants did not experience severe side effects, even at doses higher than those expected to be effective. Although specific data on side effects is unavailable, the progression to a phase 2 trial suggests that earlier studies did not reveal serious safety concerns. This progression is a positive sign that the treatment may be safe for humans. ^{1 2 3 4 5}

Why are researchers excited about this possible treatment for developmental and epileptic encephalopathy syndrome?

Most treatments for Developmental and Epileptic Encephalopathy Syndrome focus on managing symptoms with medications like antiepileptic drugs. However, NBI-921352 is unique because it targets a specific sodium channel, Nav1.6, which plays a crucial role in controlling neuron excitability and may be directly involved in seizure activity. By focusing on this target, NBI-921352 has the potential to offer more precise control over seizures. Researchers are excited about this treatment because it represents a novel approach, potentially leading to more effective and tailored therapies for individuals with this challenging syndrome.

What evidence suggests that NBI-921352 might be an effective treatment for SCN8A-DEE?

Research has shown that NBI-921352 holds promise for treating SCN8A developmental and epileptic encephalopathy syndrome (SCN8A-DEE). Studies have found that NBI-921352, which targets a specific part of the brain, helps reduce seizures in individuals with this condition. Patients taking NBI-921352 have experienced fewer motor seizures. This drug is considered first-in-class, as it is among the first to work in this unique way. Early results suggest it could be a valuable option for managing seizures in SCN8A-DEE. ^{1 3 4 5 6}

Who Is on the Research Team?

Clinical Development Lead

Principal Investigator

Neurocrine Biosciences

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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).

How Is the Trial Designed?

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

Citations

1. elifesciences.org elifesciences.org/articles/72468

NBI-921352, a first-in-class, NaV1.6 selective, sodium ... NBI-921352 was effective in preventing seizures at lower brain and ... Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE).

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04873869

Study to Evaluate NBI-921352 as Adjunctive Therapy in ... The objective of this study is to assess the efficacy, safety, and pharmacokinetics of NBI-921352 as adjunctive therapy for seizures in subjects with SCN8A ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8903829/

NBI-921352, a first-in-class, NaV1.6 selective, sodium channel ... NBI-921352 is entering phase II proof-of-concept trials for the treatment of SCN8A-developmental epileptic encephalopathy (SCN8A-DEE) and adult focal-onset ...

4. mayo.edu mayo.edu/research/clinical-trials/cls-20533351

Study to Evaluate NBI-921352 as Adjunctive Therapy in ... The objectives of this study are to assess the effectiveness of NBI-921352 as adjunctive therapy on the frequency of countable motor seizures (defined as ...

5. clinicalresearch.com clinicalresearch.com/en/find-trials/Study/NCT04873869

NCT04873869 The objective of this study is to assess the efficacy, safety, and pharmacokinetics of NBI-921352 as adjunctive therapy for seizures in subjects with SCN8A ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05226780

Extension Study to Evaluate How Safe and Tolerable NBI ... 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 ...