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

Primary Purpose

SCN8A Developmental and Epileptic Encephalopathy Syndrome

Status

Enrolling by invitation

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

NBI-921352

About this trial

This is an interventional treatment trial for SCN8A Developmental and Epileptic Encephalopathy Syndrome focused on measuring Epilepsy, Sodium channel, voltage-gated, type VIII, alpha subunit (SCN8A), NaV1.6 inhibitor

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept 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

1. 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.

Sites / Locations

UCSF Medical Center
Children's National Hospital
University of Rochester
Cook Children's Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

NBI-921352

Arm Description

106 week treatment Period

Outcomes

Primary Outcome Measures

The subject incidence of serious treatment-emergent adverse events (TEAEs), TEAEs leading to discontinuation of study treatment, and fatal TEAEs.

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.

Full Information

NCT ID

NCT05226780

First Posted

January 26, 2022

Last Updated

February 7, 2023

Sponsor

Neurocrine Biosciences

1. Study Identification

Unique Protocol Identification Number

NCT05226780

Brief Title

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

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)

Study Type

Interventional

2. Study Status

Record Verification Date

February 2023

Overall Recruitment Status

Enrolling by invitation

Study Start Date

July 12, 2022 (Actual)

Primary Completion Date

March 2026 (Anticipated)

Study Completion Date

May 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Neurocrine Biosciences

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. 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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

SCN8A Developmental and Epileptic Encephalopathy Syndrome

Keywords

Epilepsy, Sodium channel, voltage-gated, type VIII, alpha subunit (SCN8A), NaV1.6 inhibitor

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

52 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

NBI-921352

Arm Type

Experimental

Arm Description

106 week treatment Period

Intervention Type

Drug

Intervention Name(s)

NBI-921352

Intervention Description

Administered orally

Primary Outcome Measure Information:

Title

The subject incidence of serious treatment-emergent adverse events (TEAEs), TEAEs leading to discontinuation of study treatment, and fatal TEAEs.

Time Frame

Day 1 to Week 106

Secondary Outcome Measure Information:

Title

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.

Time Frame

(End of) Weeks 5, 30, 54, 78, and 104

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Accepts Healthy Volunteers

Eligibility Criteria

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 1. 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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Clinical Development Lead

Organizational Affiliation

Neurocrine Biosciences

Official's Role

Study Director

Facility Information:

Facility Name

UCSF Medical Center

City

San Francisco

State/Province

California

ZIP/Postal Code

94143

Country

United States

Facility Name

Children's National Hospital

City

Washington

State/Province

District of Columbia

ZIP/Postal Code

20010

Country

United States

Facility Name

University of Rochester

City

Rochester

State/Province

New York

ZIP/Postal Code

14642

Country

United States

Facility Name

Cook Children's Medical Center

City

Fort Worth

State/Province

Texas

ZIP/Postal Code

76104

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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