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Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-827104 in Pediatric Subjects With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04625101
Recruitment Status : Active, not recruiting
First Posted : November 12, 2020
Last Update Posted : July 12, 2022
Sponsor:
Information provided by (Responsible Party):
Neurocrine Biosciences

Brief Summary:
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).

Condition or disease Intervention/treatment Phase
Epileptic Encephalopathy Continuous Spike and Wave During Sleep Drug: NBI-827104 Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : April 26, 2021
Estimated Primary Completion Date : August 2022
Estimated Study Completion Date : November 2022


Arm Intervention/treatment
Experimental: NBI-827104
NBI-827104 administered orally for 13 weeks.
Drug: NBI-827104
Triple T-type calcium channel blocker.

Placebo Comparator: Placebo
Placebo administered orally for 13 weeks.
Drug: Placebo
Non-active dosage form.




Primary Outcome Measures :
  1. 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 ]

Secondary Outcome Measures :
  1. Ratio of SWI during first hour of NREM sleep, based on centralized evaluation. [ Time Frame: Baseline to Week 12 ]
  2. Caregiver Global Impression of Change (GI-C) and Clinical Global Impression of Change (CGI-C) scores. [ Time Frame: Week 6 and Week 12 ]
    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.

  3. Clinical Global Impression of Severity (CGI-S) scores. [ Time Frame: Baseline to the end of Week 6 and Week 12 ]
    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.



Information from the National Library of Medicine

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Ages Eligible for Study:   4 Years to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Signed informed consent by the parent(s) or legal representative(s) and, if applicable, assent from developmentally capable pediatric subjects.
  2. Diagnosis of EECSWS.
  3. Have diagnosis of EECSWS confirmed by the Diagnosis Confirmation Panel (DCP).
  4. 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.
  5. 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:

  1. Lennox-Gastaut syndrome, Doose syndrome (epilepsy with myoclonic-atonic seizures), or Dravet syndrome.
  2. 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.
  3. 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.
  4. Body weight <10 kg at randomization.
  5. 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.
  6. Have an average triplicate ECG corrected QT interval using Fridericia's formula (QTcF) >450 msec or presence of any significant cardiac abnormality at screening.
  7. Clinically relevant findings in clinical laboratory tests (hematology, clinical chemistry including thyroid function parameters, and urinalysis) at screening as determined by the investigator.
  8. Have aspartate aminotransferase (AST), alanine aminotransferase (ALT), or gamma-glutamyl transferase (GGT) levels >2 × the upper limit of normal (ULN) at screening.
  9. Have mild to severe renal impairment as determined by the investigator.
  10. Have taken cannabinoids, excluding Epidiolex®/Epidyolex®, within 30 days of screening.
  11. Pulse therapy such as systemic corticosteroids and IVIG are prohibited for at least 8 weeks prior to screening.
  12. Planned surgical intervention related to structural abnormalities of the brain from screening through the duration of the study.
  13. 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.
  14. Any circumstances or conditions, which, in the opinion of the investigator, may affect full participation in the study or compliance with the protocol.

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): NCT04625101


Locations
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United States, California
Neurocrine Clinical Site
Orange, California, United States, 92868
United States, Colorado
Neurocrine Clinical Site
Aurora, Colorado, United States, 80045
United States, District of Columbia
Neurocrine Clinical Site
Washington, District of Columbia, United States, 20010
United States, Florida
Neurocrine Clinical Site
Miami, Florida, United States, 33155
United States, Minnesota
Neurocrine Clinical Site
Rochester, Minnesota, United States, 55905
United States, North Carolina
Neurocrine Clinical Site
Durham, North Carolina, United States, 27710
United States, Ohio
Neurocrine Clinical Site
Cleveland, Ohio, United States, 44195
United States, Pennsylvania
Neurocrine Clinical Site
Philadelphia, Pennsylvania, United States, 19104
Canada, Alberta
Neurocrine Clinical Site
Calgary, Alberta, Canada, T3B 6A8
Denmark
Neurocrine Clinical Site
Dianalund, Denmark, 4293
Spain
Neurocrine Clinical Site
Barcelona, Spain, 08950
Neurocrine Clinical Site
Madrid, Spain, 28034
Switzerland
Neurocrine Clinical Site
Basel, Switzerland, 4031
Neurocrine Clinical Site
Zürich, Switzerland, 8032
United Kingdom
Neurocrine Clinical Site
London, United Kingdom, WC1N 3JH
Sponsors and Collaborators
Neurocrine Biosciences
Investigators
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Study Director: Clinical Development Lead Neurocrine Biosciences
Additional Information:
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Responsible Party: Neurocrine Biosciences
ClinicalTrials.gov Identifier: NCT04625101    
Other Study ID Numbers: NBI-827104-CSWS2010
2020-003141-11 ( EudraCT Number )
First Posted: November 12, 2020    Key Record Dates
Last Update Posted: July 12, 2022
Last Verified: July 2022
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
Additional relevant MeSH terms:
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Brain Diseases
Epilepsy
Central Nervous System Diseases
Nervous System Diseases