Extension Study to Evaluate How Safe and Tolerable NBI-921352 is as an Adjunctive Therapy for Subjects With SCN8A-DEE

Sponsor

Neurocrine Biosciences (Industry)

Overall Status

Enrolling by invitation

CT.gov ID

NCT05226780

Collaborator

(none)

Enrollment

Location

Arm

45.6

Anticipated Duration (Months)

1.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Extension Study to Evaluate how safe and tolerable the drug NBI-921352 is when used as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE).

Condition or Disease	Intervention/Treatment	Phase
SCN8A Developmental and Epileptic Encephalopathy Syndrome	Drug: NBI-921352	Phase 2

Study Design

Study Type:

Interventional

Anticipated Enrollment :

52 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A Prospective, Long-Term, Interventional, Active Extension Study to Evaluate the Safety and Tolerability of NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)

Actual Study Start Date :

Jul 12, 2022

Anticipated Primary Completion Date :

Mar 1, 2026

Anticipated Study Completion Date :

May 1, 2026

Arms and Interventions

Arm	Intervention/Treatment
Experimental: NBI-921352 106 week treatment Period	Drug: NBI-921352 Administered orally

Arm

Intervention/Treatment

Experimental: NBI-921352

106 week treatment Period

Drug: NBI-921352

Administered orally

Outcome Measures

Primary Outcome Measures

The subject incidence of serious treatment-emergent adverse events (TEAEs), TEAEs leading to discontinuation of study treatment, and fatal TEAEs. [Day 1 to Week 106]

Secondary Outcome Measures

Percentage change from baseline in 28-day seizure frequency for countable motor seizures during the Treatment Period of the study at (End of) Weeks 5, 30, 54, 78, and 104. [(End of) Weeks 5, 30, 54, 78, and 104]

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Key Inclusion Criteria:

Written or oral pediatric assent from the subject and written informed consent from the subject's parent(s) or legal guardian(s) for subjects<18 years of age and for subjects ≥18 years of age.
Completed 16 weeks of treatment in Study NBI-921352-DEE2012.
Continue to use a nocturnal alerting system or practice consistent with standards of care for the duration of the study.
Have an adequate rescue medication regimen per the investigator's judgment in place for the duration of the study.

Key Exclusion Criteria

Have developed any other disorder for which the treatment takes priority over treatment of SCN8A-DEE or is likely to interfere with study treatment or impair treatment compliance.