The objective of this study is to assess the efficacy, safety, and pharmacokinetics of NBI-921352 as adjunctive therapy for seizures in subjects with SCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
8
Administered orally
Administered orally
UCSF Medical Center
San Francisco, California, United States
Children's National Hospital
Washington D.C., District of Columbia, United States
University of Rochester
Rochester, New York, United States
Cook Children's Medical Center
Fort Worth, Texas, United States
Percentage Change From Baseline in 28-day Seizure Frequency for Countable Motor Seizures During the 16-week Treatment Period
Time frame: Planned time frame: Baseline to Week 16
Percentage of Participants With a Treatment Response
Treatment response was defined as a ≥50% decrease from baseline in 28-day seizure frequency for countable motor seizures during the treatment period of the study.
Time frame: Planned time frame: Baseline to Week 16
Percentage Change From Baseline in 28-day Seizure Frequency for Countable Motor Seizures During the 10-week Maintenance Period
Time frame: Planned time frame: Baseline, Week 6 to Week 16
Percentage of Participants With a ≥ 25%, ≥ 75%, or 100% Treatment Response During the 16-week Treatment Period
Treatment response was defined as a ≥25%, ≥50%, ≥75%, or 100% decrease from baseline in 28-day seizure frequency for countable motor seizures during the treatment period of the study.
Time frame: Planned time frame: Baseline to Week 16
Percentage of Participants With a ≥25%, ≥50%, ≥75%, or 100% Treatment Response During the 10-week Maintenance Period
Treatment response was defined as a ≥25%, ≥50%, ≥75%, or 100% decrease from baseline in 28-day seizure frequency for countable motor seizures during the treatment period of the study.
Time frame: Planned time frame: Baseline, Week 6 to Week 16
Clinical Global Impression of Change (CGIC) Score at Each Visit During the 16-week Treatment Period
The CGIC scale, which is based on a 7-point scale (range: 1=very much improved to 7=very much worse), was used to rate the overall global improvement since the initiation of study treatment dosing, as rated by the investigator (or qualified designee).
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Time frame: Planned time frame: Up to Week 16
Parent/Caregiver Global Impression of Change (GIC) Score at Each Visit During the 16-week Treatment Period
The GIC scale was used to assess the parent/caregiver's impression of change in the participant's overall condition since starting study treatment and was rated on a 7-point scale (1=very much improved to 7=very much worse).
Time frame: Planned time frame: Up to Week 16
Change From Baseline in Clinical Global Impression of Severity (CGIS) Scores at Each Visit During the 16-week Treatment Period
The CGIS scale was used to assess overall severity on a 5-point scale (range: 1=normal, not at all ill to 5=among the most extremely ill).
Time frame: Planned time frame: Baseline to Week 16
Change From Baseline in Parent/Caregiver Global Impression of Severity (GIS) Scores at Each Visit During the 16-week Treatment Period
The GIS scale was used to assess overall severity on a 5-point scale (range: 1=none to 5=very severe).
Time frame: Planned time frame: Baseline through Week 16