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

NCT ID: NCT04625101

Last Updated: 2025-09-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-04-26
• Study Completion Date: 2022-10-11

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).

Conditions

Epileptic Encephalopathy; Continuous Spike and Wave During Sleep

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

NBI-827104

NBI-827104 administered orally for 13 weeks.

Group Type: EXPERIMENTAL

NBI-827104

Intervention Type: DRUG

Triple T-type calcium channel blocker.

Placebo

Placebo administered orally for 13 weeks.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Non-active dosage form.

Interventions

NBI-827104

Triple T-type calcium channel blocker.

Intervention Type: DRUG

Placebo

Non-active dosage form.

Intervention Type: DRUG

Eligibility 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.

• Minimum Eligible Age: 4 Years
• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Neurocrine Biosciences

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Development Lead

Role: STUDY_DIRECTOR

Neurocrine Biosciences

Locations

Neurocrine Clinical Site

Orange, California, United States

Neurocrine Clinical Site

Aurora, Colorado, United States

Neurocrine Clinical Site

Washington D.C., District of Columbia, United States

Neurocrine Clinical Site

Miami, Florida, United States

Neurocrine Clinical Site

Rochester, Minnesota, United States

Neurocrine Clinical Site

Durham, North Carolina, United States

Neurocrine Clinical Site

Cleveland, Ohio, United States

Neurocrine Clinical Site

Philadelphia, Pennsylvania, United States

Neurocrine Clinical Site

Calgary, Alberta, Canada

Neurocrine Clinical Site

Dianalund, , Denmark

Neurocrine Clinical Site

Barcelona, , Spain

Neurocrine Clinical Site

Madrid, , Spain

Neurocrine Clinical Site

Basel, , Switzerland

Neurocrine Clinical Site

Zurich, , Switzerland

Neurocrine Clinical Site

London, , United Kingdom

Countries

United States; Canada; Denmark; Spain; Switzerland; United Kingdom

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2020-003141-11

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

NBI-827104-CSWS2010

Identifier Type: -

Identifier Source: org_study_id