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

Overview

About this study

The purposes of this research are to assess the effect of NBI-827104, compared with placebo (no active drug), on the overnight epileptiform electroencephalogram (EEG) activity in pediatric participants with epileptic encephalopathy with continuous spike-and-wave during sleep (EECSWS), to evaluate the safety and tolerability of multiple doses of NBI-827104 in pediatric participants with EECSWS, and to evaluate the effect of NBI-827104 on sleep and on cognition in pediatric participants with EECSWS.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

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.

Eligibility last updated 8/10/23. Questions regarding updates should be directed to the study team contact.

 

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status

Rochester, Minn.

Mayo Clinic principal investigator

Anthony Fine, M.D.

Closed for enrollment

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