Study to Evaluate NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)

December 21, 2023 updated by: Neurocrine Biosciences

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-921352 as Adjunctive Therapy in Subjects With SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)

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 Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

52

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • California
      • San Francisco, California, United States, 94158
        • Recruiting
        • UCSF Medical Center
        • Contact:
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
    • Illinois
      • Chicago, Illinois, United States, 60611
    • Minnesota
      • Rochester, Minnesota, United States, 55905
    • New York
    • North Carolina
      • Winston-Salem, North Carolina, United States, 27157
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia
        • Contact:
    • Texas
      • Fort Worth, Texas, United States, 76104
    • Utah

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

2 years to 21 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Male or female 2 to 21 years of age, inclusive.
  2. Have a diagnosis of SCN8A-DEE supported by both clinical and genetic findings
  3. Have on average at least 1 countable motor seizure per week and not be seizure-free for more than 20 consecutive days
  4. Being treated with at least 1 other antiseizure medication (ASM), but no more than 4 ASMs
  5. Have failed to achieve seizure freedom with at least 2 ASMs
  6. Must be using a nocturnal alerting system or practice consistent with standards of care at the time of screening and continue to use this for the duration of the study
  7. Must have an adequate rescue medication regimen per the investigator's judgment in place at the time of screening and for the duration of the study
  8. Have a body weight of at least 10 kg
  9. The subject's parent/caregiver is able to accurately identify seizure types, especially countable motor seizures (defined as GTCS, tonic, atonic or FOS with noticeable motor component) and is able to complete seizure diary

Exclusion Criteria:

  1. Have previously been enrolled in this study and received blinded treatment
  2. Have participated in an interventional clinical trial < 30 days prior to screening
  3. Have symptoms that would be more consistent with another epilepsy disorder such as Dravet syndrome (eg, fever-induced episodes of status epilepticus, frequent myoclonic seizures, worsening on sodium channel blockers, absence seizures with generalized spike-and-wave EEG as the sole seizure type)
  4. Are currently receiving cannabinoids or medical marijuana except Epidiolex/Epidyolex, unless approved by the Sponsor
  5. Are currently taking systemic steroids (excluding inhaled medication for asthma treatments and intranasal steroids for allergies). If subject has received these medications in the past, must be off these medications for at least 3 months prior to the screening visit and these drugs may not be initiated during the duration of the study. Intermittent steroids to treat nonepilepsy related diseases (such as allergies or dermatological conditions) are not exclusionary
  6. 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
  7. Have a clinically significant medical condition or chronic disease, that in the opinion of the investigator would preclude the subject from participating in and completing the study or that could confound interpretation of study outcome
  8. Have clinically significant abnormal vital signs at the screening visit as determined by the investigator
  9. Have one or more clinical laboratory test values outside the reference range, based on blood samples taken at the screening visit, that are of potential risk to the subject's safety as determined by the investigator
  10. Have, at the screening visit, an electrocardiogram (ECG) finding of a corrected QT interval using Fridericia's formula (QTcF) > 450 msec or presence of any significant cardiac abnormality.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Participants will receive matching placebo for up to 18 weeks.
Drug: Placebo
Administered orally
Experimental: NBI-921352
In the first 6 weeks participants will receive increasing doses of NBI-921352 (Titration Period) based on weight, followed by 10 weeks of treatment at their final tolerated dose (Maintenance Period) and 2 weeks of treatment with decreasing doses (Taper Period).
Drug: NBI-921352
Administered orally

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Percentage Change from Baseline in 28-day Seizure Frequency for Countable Motor Seizures During the Treatment Period
Time Frame: Baseline, Treatment Period: Day 1 to Week 16
Baseline, Treatment Period: Day 1 to Week 16

Secondary Outcome Measures

Outcome Measure
Time Frame
Percentage of Participants with a ≥ 50% Treatment Response for Countable Motor Seizures During the Treatment Period
Time Frame: Baseline, Treatment Period: Day 1 to Week 16
Baseline, Treatment Period: Day 1 to Week 16
Percentage Change from Baseline in 28-day Seizure Frequency for Countable Motor Seizures During the Maintenance Period
Time Frame: Baseline, Maintenance Period: Week 6 to Week 16
Baseline, Maintenance Period: Week 6 to Week 16
Percentage of Participants with a ≥ 25%, ≥ 75%, or 100% Treatment Response During the Treatment Period
Time Frame: Baseline, Treatment Period: Day 1 to Week 16
Baseline, Treatment Period: Day 1 to Week 16
Percentage of Participants with a ≥ 25%, ≥ 50%, ≥ 75%, or 100% Treatment Response During the Maintenance Period
Time Frame: Baseline, Maintenance Period: Week 6 to Week 16
Baseline, Maintenance Period: Week 6 to Week 16
Clinical Global Impression of Change (CGIC)
Time Frame: Treatment Period: Up to Week 16
Treatment Period: Up to Week 16
Parent/Caregiver Global Impression of Change (GIC)
Time Frame: Treatment Period: Up to Week 16
Treatment Period: Up to Week 16
Change from Baseline in Clinical Global Impression of Severity (CGIS)
Time Frame: Baseline, Treatment Period: Up to Week 16
Baseline, Treatment Period: Up to Week 16
Change from Baseline in Parent/Caregiver Global Impression of Severity (GIS)
Time Frame: Baseline, Treatment Period: Up to Week 16
Baseline, Treatment Period: Up to Week 16

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Neurocrine Biosciences

Investigators

  • Study Director: Clinical Development Lead, Neurocrine Biosciences

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 31, 2022

Primary Completion (Estimated)

December 1, 2025

Study Completion (Estimated)

December 1, 2025

Study Registration Dates

First Submitted

April 30, 2021

First Submitted That Met QC Criteria

April 30, 2021

First Posted (Actual)

May 5, 2021

Study Record Updates

Last Update Posted (Actual)

December 22, 2023

Last Update Submitted That Met QC Criteria

December 21, 2023

Last Verified

December 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NBI-921352-DEE2012
  • 2020-003140-83 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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