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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04873869
Recruitment Status : Recruiting
First Posted : May 5, 2021
Last Update Posted : February 9, 2023
Sponsor:
Information provided by (Responsible Party):
Neurocrine Biosciences

Study Description
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Brief Summary:
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).

Condition or disease Intervention/treatment Phase
SCN8A Developmental and Epileptic Encephalopathy Syndrome Drug: NBI-921352 Drug: Placebo Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 52 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: 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)
Actual Study Start Date : January 31, 2022
Estimated Primary Completion Date : January 2024
Estimated Study Completion Date : February 2024

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Epilepsy

Arms and Interventions
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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


Outcome Measures
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Primary Outcome Measures :
  1. 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 ]

Secondary Outcome Measures :
  1. 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 ]
  2. 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 ]
  3. 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 ]
  4. 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 ]
  5. Clinical Global Impression of Change (CGIC) [ Time Frame: Treatment Period: Up to Week 16 ]
  6. Parent/Caregiver Global Impression of Change (GIC) [ Time Frame: Treatment Period: Up to Week 16 ]
  7. Change from Baseline in Clinical Global Impression of Severity (CGIS) [ Time Frame: Baseline, Treatment Period: Up to Week 16 ]
  8. Change from Baseline in Parent/Caregiver Global Impression of Severity (GIS) [ Time Frame: Baseline, Treatment Period: Up to Week 16 ]

Eligibility Criteria
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Ages Eligible for Study:   2 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04873869


Contacts
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Contact: Neurocrine Medical Information Call Center 877-641-3461 medinfo@neurocrine.com

Locations
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United States, California
UCSF Medical Center Recruiting
San Francisco, California, United States, 94158
Contact: Lucy Liu    415-353-2437    Lucy.liu3@ucsf.edu   
United States, District of Columbia
Children's National Hospital Recruiting
Washington, District of Columbia, United States, 20010
Contact: Zachary Kramer       zkramer@childrensnational.org   
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Enrique Rojas       erojas@luriechildrens.org   
United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Bridget Neja    507-266-9150    neja.bridget@mayo.edu   
United States, New York
University of Rochester Recruiting
Rochester, New York, United States, 14642
Contact: Noreen Connolly       Noreen_Connolly@urmc.rochester.edu   
United States, North Carolina
Wake Forest Baptist Health Not yet recruiting
Winston-Salem, North Carolina, United States, 27157
Contact: Carolyn Hedrick       cwhedric@wakehealth.edu   
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Jennie Minnick       ENGIN@chop.edu   
United States, Texas
Cook Children's Medical Center Recruiting
Fort Worth, Texas, United States, 76104
Contact: Dianna Grado    682-885-2844    Dianna.grado@cookchildrens.org   
United States, Utah
University of Utah Not yet recruiting
Salt Lake City, Utah, United States, 84132
Contact: Carly Straley       clinicaltrialsoffice@hsc.utah.edu   
Sponsors and Collaborators
Neurocrine Biosciences
Investigators
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Study Director: Clinical Development Lead Neurocrine Biosciences
More Information
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Additional Information:

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Responsible Party: Neurocrine Biosciences
ClinicalTrials.gov Identifier: NCT04873869    
Other Study ID Numbers: NBI-921352-DEE2012
2020-003140-83 ( EudraCT Number )
First Posted: May 5, 2021    Key Record Dates
Last Update Posted: February 9, 2023
Last Verified: February 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Neurocrine Biosciences:
Epilepsy
Sodium channel
voltage-gated
 type VIII
alpha subunit (SCN8A)
NaV1.6 inhibitor
Additional relevant MeSH terms:
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Brain Diseases
Epilepsy
Syndrome
Disease
 Pathologic Processes
Central Nervous System Diseases
Nervous System Diseases