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


About this study

The primary objective of this study is to evaluate the long-term safety and tolerability of NBI-827104 in pediatric subjects with epileptic encephalopathy with continuous spike-and-wave during sleep (EECSWS) when administered for up to 106 weeks.

Its secondary objectives are to investigate the effect of NBI-827104 on the overnight epileptiform video-electroeItephalogram (video-EEG) activity in pediatric subjects with EECSWS, and to investigate the long-term effect of NBI-827104 on the symptoms of EECSWS, including sleep and cognition, in pediatric subjects.


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:

  • Written or oral pediatric assent from the subject deemed capable of providing assent and written informed consent from the subject’s parent(s) or legal guardian(s) in accordance with the governing Institutional Review Board (IRB) or Independent Ethics Committee (IEC) and according to local laws and regulations. Informed consent/assent may be done remotely, if allowed per site and remote consenting procedures are in place.
  • Completed 12 weeks of treatment (3 weeks titration and 9 weeks maintenance) in Study NBI-827104-CSWS2010.
  • Subjects of childbearing potential must agree to use highly effective birth control methods consistently while participating in the study until 90 days after the last dose of the study treatment.
  • A female subject of childbearing potential is defined as a subject who has had her first menstrual cycle (ie, menarche). A male subject of childbearing potential is defined as a subject who has reached spermarche.
  • Highly effective methods of birth control for female subjects of childbearing potential are:
    • Combined (estrogen and progestogen containing) hormonal contraception or progestogen-only hormonal contraception used with an effective nonhormonal method of contraception (eg, barrier contraception used with spermicide);
    • Intrauterine hormone-releasing system used with an effective nonhormonal method of contraception (eg, barrier contraception used with spermicide);
    • Intrauterine device;
    • Bilateral tubal occlusion;
    • Vasectomized partner;
    • True abstinence from sexual intercourse as the preferred lifestyle;
    • Note that periodic abstinence (e.g., calendar, ovulation, symptothermal, postovulation methods) is not acceptable.     
  • Female subjects of childbearing potential must have a negative urine pregnancy test at Day 1.
  • The acceptable methods of contraception for male subjects of childbearing potential are:
    • Condom with spermicide (cream, spray, foam, gel, suppository, or polymer film);
    • Vasectomy at least 3 months prior to screening with medically confirmed successful procedure;
    • True abstinence from sexual intercourse as the preferred lifestyle. Note that periodic abstinence is not acceptable.
  • A subject who becomes of childbearing potential during the study will be required to use contraception as described.
  • Willing to comply with all study procedures and restrictions.

Exclusion Criteria:

  • Pregnant or breastfeeding.
  • Planned surgical intervention related to structural abnormalities of the brain from screening through the Week 6 Visit.
  • Used any active investigational drug other than NBI-827104 in the context of a clinical study within 30 days or 5 half-lives (whichever is longer) before Day 1 or plans to use such an investigational drug (other than NBI-827104) during the study.
  • It is anticipated that the subject will require treatment with at least 1 of the prohibited concomitant medications or other restrictions during the specified study time frame.
  • Have developed any other disorder for which the treatment takes priority over treatment of EECSWS or is likely to interfere with study treatment or impair treatment compliance.
  • The subject or the subject’s parent(s)/caregiver(s) are, in the investigator’s opinion, unlikely to comply with the protocol, including the requirement to travel to the study sites for study visits, or is unsuitable for any reason.

Eligibility last updated 2/18/22. 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 Contact

Rochester, Minn.

Mayo Clinic principal investigator

Anthony Fine, M.D.

Open for enrollment

Contact information:

Bridget Neja C.N.A.

(507) 266-9150


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