NBI-827104 for Epilepsy

(Steamboat 2 Trial)

No longer recruiting at 13 trial locations

Overseen By Neurocrine Medical Information Call Center

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Neurocrine Biosciences

Must not be taking: Investigational drugs

Disqualifiers: Cardiovascular, Neurological, Surgery, others

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: 26 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called NBI-827104 for children with epileptic encephalopathy with continuous spike-and-wave during sleep (EECSWS), a type of epilepsy affecting brain activity during sleep. The main goal is to determine if the medicine is safe and tolerable over a long period. Children diagnosed with EECSWS who are not receiving treatment for other priority conditions may be suitable for this trial. Participants should not have recently taken other investigational drugs and should not have certain neurological disorders besides EECSWS. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important advancements in epilepsy care.

Do I need to stop my current medications to join the trial?

The trial information does not specify if you need to stop your current medications. However, if you are taking another investigational drug, you must stop it at least 30 days before joining the study.

Is there any evidence suggesting that NBI-827104 is likely to be safe for humans?

Research shows that NBI-827104 is generally safe for people. In studies, participants taking NBI-827104 reported only a few side effects, indicating its safety. No serious adverse reactions have been reported. This treatment is being tested in children with a specific type of epilepsy, making safety a top priority. While the current study examines long-term safety and tolerability, earlier research provides a reassuring safety record. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for epilepsy?

Unlike the standard treatments for epilepsy, which often include anti-seizure medications like valproate, lamotrigine, and levetiracetam that primarily stabilize electrical activity in the brain, NBI-827104 offers a new approach. Researchers are excited about NBI-827104 because it acts on a specific receptor in the brain, potentially offering a more targeted therapy with fewer side effects. This oral medication could provide a new option for patients who do not respond well to existing treatments, promising a more personalized and effective management of epilepsy.

What evidence suggests that NBI-827104 might be an effective treatment for epilepsy?

Research has shown that NBI-827104 can enter the brain and is designed to block specific calcium channels there. These channels, known as T-type calcium channels, play a role in the brain's electrical activity that can lead to seizures. Although NBI-827104 did not achieve its main goal in a previous study, patients generally tolerated it well. The drug aims to reduce unusual brain activity during sleep, a major issue in epileptic encephalopathy with continuous spike-and-wave during sleep (EECSWS). While information on its effectiveness is limited, its mechanism suggests it might help manage this condition. Participants in this trial will receive NBI-827104 to further evaluate its effectiveness and safety. ^{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 children with a specific brain condition called EECSWS. Those who completed a prior study (NBI-827104-CSWS2010) can join, as well as new participants with confirmed EECSWS diagnosis. Kids under 15kg, those with certain heart or lab issues, other neurological disorders not stable for at least a year, planned brain surgery within six weeks of the trial start, or recent use of investigational drugs cannot participate.

Inclusion Criteria

My EECSWS diagnosis has been confirmed by a specialized panel.

If you were in another study before this one, you must have finished the treatment for at least 12 weeks.

Exclusion Criteria

I am scheduled for brain surgery due to structural abnormalities.

Your doctor found important heart or lab test results during your screening.

I weigh less than 15 kg and did not come from study NBI-827104-CSWS2010.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive NBI-827104 orally to evaluate long-term safety and tolerability

26 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants continue to receive NBI-827104 to assess long-term effects

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• NBI-827104

Trial Overview The study tests the long-term safety and effects of NBI-827104 in kids with EECSWS. It's an extension study meaning it continues from previous research to gather more data on how safe and tolerable this medication is over time for these pediatric patients.

How Is the Trial Designed?

Group I: NBI-827104 Experimental Treatment 1 Intervention

NBI-827104 administered orally

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. neurocrine.gcs-web.com neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-provides-update-phase-2-study-nbi-827104

Release Details - Neurocrine Biosciences NBI-827104 is an investigational, potent, selective, and orally active brain-penetrating T-type calcium channel blocker (Cav 3.1, Cav 3.2, Cav ...

2. humanforschung-schweiz.ch humanforschung-schweiz.ch/en/trial-search/study-detail/63021/translate/en/?cHash=aad48a3b4f8daa783e1f0b3d02c6840b

A long-term study to assess the safety and tolerability of ... The study is designed to assess the efficacy, safety, and tolerability of the active ingredient NBI-827104 against placebo in approximately 24 pediatric ...

3. prnewswire.com prnewswire.com/news-releases/neurocrine-biosciences-provides-update-on-phase-2-study-of-nbi-827104-in-pediatric-patients-with-epileptic-encephalopathy-with-continuous-spike-and-wave-during-sleep-301696306.html

Neurocrine Biosciences Provides Update on Phase 2 ... NBI-827104 was generally well tolerated. EE-CSWS is a rare pediatric developmental and/or epileptic encephalopathy that is characterized by a ...

4. mayo.edu mayo.edu/research/clinical-trials/cls-20511386

Clinical Trials The purposes of this research are to assess the effect of NBI-827104, compared with placebo (no active drug), on the overnight epileptiform ...

5. clinicaltrialsarena.com clinicaltrialsarena.com/news/neurocrine-therapy-epileptic-encephalopathy-trial/

Neurocrine therapy fails to meet endpoint in epileptic ... Neurocrine Biosciences has reported that its investigational therapy NBI-827104 failed to meet its primary endpoint in the Phase II STEAMBOAT clinical trial.

6. cdn.clinicaltrials.gov cdn.clinicaltrials.gov/large-docs/01/NCT04625101/Prot_000.pdf

CLINICAL STUDY PROTOCOL Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-827104 in Pediatric Subjects with. Epileptic Encephalopathy with Continuous Spike-and- ...