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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-003140-83 | EudraCT Number |
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This study was prematurely terminated due to sponsor decision.
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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Participants will receive matching placebo for up to 18 weeks. |
|
| NBI-921352 | Experimental | In the first 6 weeks participants will receive increasing doses of NBI-921352 (Titration Period) based on weight, followed by 10 weeks of treatment at their final tolerated dose (Maintenance Period) and 2 weeks of treatment with decreasing doses (Taper Period). |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NBI-921352 | Drug | Administered orally |
| |
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage Change From Baseline in 28-day Seizure Frequency for Countable Motor Seizures During the 16-week Treatment Period | Planned time frame: Baseline to Week 16 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Development Lead | Neurocrine Biosciences | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Medical Center | San Francisco | California | 94158 | United States | ||
| Children's National Hospital |
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Study was prematurely terminated due to sponsor decision. Due to the early termination, only 8 participants were enrolled and completed the study. As prespecified, analyses were pooled by treatment received (NBI-921352 or placebo), rather than specific dose level received.
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| ID | Title | Description |
|---|---|---|
| FG000 | NBI-921352 | Participants received oral doses of NBI-921352 3 times a day based on body weight. |
| FG001 | Placebo | Participants received oral doses of placebo matching NBI-921352 3 times a day. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 30, 2021 | Sep 23, 2025 |
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| Drug |
Administered orally |
|
| 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. | 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. | 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). | 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). | 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). | 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). | Planned time frame: Baseline through Week 16 |
| Washington D.C. |
| District of Columbia |
| 20010 |
| United States |
| University of Rochester | Rochester | New York | 14642 | United States |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | United States |
| Received Study Drug |
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| COMPLETED |
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| NOT COMPLETED |
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Safety analysis set: Included all participants who received at least 1 dose of study treatment.
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| ID | Title | Description |
|---|---|---|
| BG000 | NBI-921352 | Participants received oral doses of NBI-921352 3 times a day based on body weight. |
| BG001 | Placebo | Participants received oral doses of placebo matching NBI-921352 3 times a day. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Percentage Change From Baseline in 28-day Seizure Frequency for Countable Motor Seizures During the 16-week Treatment Period | Due to early study termination, analyses of efficacy data were not performed due to an insufficient sample size to allow for meaningful statistical analysis of the data, as pre-specified in the protocol. | Posted | Planned time frame: Baseline to Week 16 |
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| |||||||||||||||||||||||
| Secondary | 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. | Due to early study termination, analyses of efficacy data were not performed due to an insufficient sample size to allow for meaningful statistical analysis of the data, as pre-specified in the protocol. | Posted | Planned time frame: Baseline to Week 16 |
|
| ||||||||||||||||||||||
| Secondary | Percentage Change From Baseline in 28-day Seizure Frequency for Countable Motor Seizures During the 10-week Maintenance Period | Due to early study termination, analyses of efficacy data were not performed due to an insufficient sample size to allow for meaningful statistical analysis of the data, as pre-specified in the protocol. | Posted | Planned time frame: Baseline, Week 6 to Week 16 |
|
| |||||||||||||||||||||||
| Secondary | 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. | Due to early study termination, analyses of efficacy data were not performed due to an insufficient sample size to allow for meaningful statistical analysis of the data, as pre-specified in the protocol. | Posted | Planned time frame: Baseline to Week 16 |
|
| ||||||||||||||||||||||
| Secondary | 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. | Due to early study termination, analyses of efficacy data were not performed due to an insufficient sample size to allow for meaningful statistical analysis of the data, as pre-specified in the protocol. | Posted | Planned time frame: Baseline, Week 6 to Week 16 |
|
| ||||||||||||||||||||||
| Secondary | 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). | Due to early study termination, analyses of efficacy data were not performed due to an insufficient sample size to allow for meaningful statistical analysis of the data, as pre-specified in the protocol. | Posted | Planned time frame: Up to Week 16 |
|
| ||||||||||||||||||||||
| Secondary | 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). | Due to early study termination, analyses of efficacy data were not performed due to an insufficient sample size to allow for meaningful statistical analysis of the data, as pre-specified in the protocol. | Posted | Planned time frame: Up to Week 16 |
|
| ||||||||||||||||||||||
| Secondary | 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). | Due to early study termination, analyses of efficacy data were not performed due to an insufficient sample size to allow for meaningful statistical analysis of the data, as pre-specified in the protocol. | Posted | Planned time frame: Baseline to Week 16 |
|
| ||||||||||||||||||||||
| Secondary | 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). | Due to early study termination, analyses of efficacy data were not performed due to an insufficient sample size to allow for meaningful statistical analysis of the data, as pre-specified in the protocol. | Posted | Planned time frame: Baseline through Week 16 |
|
|
Up to 30 weeks
Safety Analysis Set: Included all participants who received at least 1 dose of study treatment.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | NBI-921352 | Participants received oral doses of NBI-921352 3 times a day based on body weight. | 0 | 4 | 2 | 4 | 4 | 4 |
| EG001 | Placebo | Participants received oral doses of placebo matching NBI-921352 3 times a day. | 0 | 4 | 1 | 4 | 4 | 4 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Parainfluenzae virus infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Tachycardia | Cardiac disorders | MedDRA (26.0) | Systematic Assessment |
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| Constipation | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
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| Gait disturbance | General disorders | MedDRA (26.0) | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA (26.0) | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| Enterovirus infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| Otitis media | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| Rhinovirus Infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| Sinusitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| Arthropod sting | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
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| Face injury | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
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| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
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| Eosinophil count increased | Investigations | MedDRA (26.0) | Systematic Assessment |
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| Urine leukocyte esterase positive | Investigations | MedDRA (26.0) | Systematic Assessment |
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| White blood cells urine | Investigations | MedDRA (26.0) | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
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| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
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| Coordination abnormal | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
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| Drooling | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
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| Lethargy | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
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| Seizure | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
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| Somnolence | Nervous system disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Urinary retention | Renal and urinary disorders | MedDRA (26.0) | Systematic Assessment |
| |
| Rash papular | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
|
Due to the early termination, only 8 participants were enrolled and completed the study.
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Neurocrine Medical Information | Neurocrine Biosciences | +1 877-641-3461 | medinfo@neurocrine.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 12, 2024 | Sep 23, 2025 | SAP_001.pdf |
| ID | Term |
|---|---|
| D004827 | Epilepsy |
| ID | Term |
|---|---|
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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