- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04625101
Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-827104 in Pediatric Subjects With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep
August 2, 2023 updated by: Neurocrine Biosciences
Phase 2 Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-827104 in Pediatric Subjects With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep
This is a phase 2, double-blind study to assess the efficacy, safety, tolerability, and pharmacokinetics of NBI-827104 when administered once daily for 13 weeks in pediatric subjects with Epileptic Encephalopathy with Continuous Spike-and-Wave During Sleep (EECSWS).
Study Overview
Status
Completed
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
24
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 Locations
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Alberta
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Calgary, Alberta, Canada, T3B 6A8
- Neurocrine Clinical Site
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Dianalund, Denmark, 4293
- Neurocrine Clinical Site
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Barcelona, Spain, 08950
- Neurocrine Clinical Site
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Madrid, Spain, 28034
- Neurocrine Clinical Site
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Basel, Switzerland, 4031
- Neurocrine Clinical Site
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Zürich, Switzerland, 8032
- Neurocrine Clinical Site
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London, United Kingdom, WC1N 3JH
- Neurocrine Clinical Site
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California
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Orange, California, United States, 92868
- Neurocrine Clinical Site
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Colorado
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Aurora, Colorado, United States, 80045
- Neurocrine Clinical Site
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District of Columbia
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Washington, District of Columbia, United States, 20010
- Neurocrine Clinical Site
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Florida
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Miami, Florida, United States, 33155
- Neurocrine Clinical Site
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Minnesota
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Rochester, Minnesota, United States, 55905
- Neurocrine Clinical Site
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North Carolina
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Durham, North Carolina, United States, 27710
- Neurocrine Clinical Site
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Ohio
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Cleveland, Ohio, United States, 44195
- Neurocrine Clinical Site
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- Neurocrine Clinical Site
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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 10 years (Child)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Signed informed consent by the parent(s) or legal representative(s) and, if applicable, assent from developmentally capable pediatric subjects.
- Diagnosis of EECSWS.
- Have diagnosis of EECSWS confirmed by the Diagnosis Confirmation Panel (DCP).
- Stable dosage and stable time of intake of at least 1 and up to 3 antiseizure medications (ASMs) excluding systemic corticosteroids and intravenous immunoglobulin (IVIG), from 4 weeks prior to screening and anticipated to be stable from screening until end of study (EOS). Vagal nerve stimulator (VNS) and ketogenic diet are not counted as ASMs.
- Treatment other than ASMs (excluding systemic corticosteroids and IVIG) must be at a stable dosage from 2 weeks prior to screening and anticipated to be stable from screening until EOS.
Exclusion Criteria:
- Lennox-Gastaut syndrome, Doose syndrome (epilepsy with myoclonic-atonic seizures), or Dravet syndrome.
- Presence of a relevant psychiatric disease interfering with cognitive or behavioral functioning (eg, depression, schizophrenia, autism spectrum disorder) unless associated with the EECSWS diagnosis as assessed by the investigator.
- Presence of relevant neurological disorders other than EECSWS and its underlying conditions as judged by the investigator. Symptomatic conditions underlying EECSWS (eg, neonatal strokes) have to be stable for at least 1 year prior to screening.
- Body weight <10 kg at randomization.
- Clinically relevant findings in systolic blood pressure (SBP), diastolic blood pressure (DBP), or pulse rate at screening or Day 1 as determined by the investigator.
- Have an average triplicate ECG corrected QT interval using Fridericia's formula (QTcF) >450 msec or presence of any significant cardiac abnormality at screening.
- Clinically relevant findings in clinical laboratory tests (hematology, clinical chemistry including thyroid function parameters, and urinalysis) at screening as determined by the investigator.
- Have aspartate aminotransferase (AST), alanine aminotransferase (ALT), or gamma-glutamyl transferase (GGT) levels >2 × the upper limit of normal (ULN) at screening.
- Have mild to severe renal impairment as determined by the investigator.
- Have taken cannabinoids, excluding Epidiolex®/Epidyolex®, within 30 days of screening.
- Pulse therapy such as systemic corticosteroids and IVIG are prohibited for at least 8 weeks prior to screening.
- Planned surgical intervention related to structural abnormalities of the brain from screening through the duration of the study.
- Have received any other investigational drug within 30 days or 5 half-lives (if known), whichever is longer, of Day 1 or plan to use an investigational drug (other than the study treatment) during the study.
- Any circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol.
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 |
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Experimental: NBI-827104
NBI-827104 administered orally for 13 weeks.
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Triple T-type calcium channel blocker.
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Placebo Comparator: Placebo
Placebo administered orally for 13 weeks.
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Non-active dosage form.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
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Ratio of spike-wave index (SWI) during first hour of nonrapid eye movement (NREM) sleep based on centralized video-EEG reading.
Time Frame: Baseline to Week 6
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Baseline to Week 6
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Ratio of SWI during first hour of NREM sleep, based on centralized evaluation.
Time Frame: Baseline to Week 12
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Baseline to Week 12
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Caregiver Global Impression of Change (GI-C) and Clinical Global Impression of Change (CGI-C) scores.
Time Frame: Week 6 and Week 12
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The Caregiver GI-C is a 7-point scale that rates the overall global improvement since the initiation of study drug dosing, ranging from 1 (very much improved) to 7 (very much worse), as assessed by the caregiver.
The CGI-C is a 7-point scale that rates the overall global improvement since the initiation of study drug dosing, ranging from 1 (very much improved) to 7 (very much worse), as assessed by the clinician.
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Week 6 and Week 12
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Clinical Global Impression of Severity (CGI-S) scores.
Time Frame: Baseline to the end of Week 6 and Week 12
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The CGI-S rates overall symptom severity on a 7-point scale ranging from 1 (normal, not at all ill) to 7 (among the most extremely ill patients), as assessed by the investigator.
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Baseline to the end of Week 6 and Week 12
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
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)
April 26, 2021
Primary Completion (Actual)
August 8, 2022
Study Completion (Actual)
October 11, 2022
Study Registration Dates
First Submitted
November 6, 2020
First Submitted That Met QC Criteria
November 6, 2020
First Posted (Actual)
November 12, 2020
Study Record Updates
Last Update Posted (Actual)
August 4, 2023
Last Update Submitted That Met QC Criteria
August 2, 2023
Last Verified
August 1, 2023
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- NBI-827104-CSWS2010
- 2020-003141-11 (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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