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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-003141-11 | EudraCT Number |
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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NBI-827104 | Experimental | NBI-827104 administered orally for 13 weeks. |
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| Placebo | Placebo Comparator | Placebo administered orally for 13 weeks. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NBI-827104 | Drug | Triple T-type calcium channel blocker. |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Ratio of Spike-Wave Index (SWI) During First Hour of Nonrapid Eye Movement (NREM) Sleep at Week 6 | The ratio of SWI at the end of Week 6 to baseline during the first hour (60 minutes) of NREM sleep based on centralized video-electroencephalograph (EEG) reading using a log base 10 scale. Baseline was defined as the last value measured prior to intake of study treatment on Day 1. SWI was defined as the percentage of seconds with ≥1 spike-wave complex(es) during defined periods of overnight NREM sleep. | Baseline to Week 6 |
| Measure | Description | Time Frame |
|---|---|---|
| Ratio of SWI During First Hour of NREM Sleep at Week 12 | The ratio of SWI at the end of Week 12 to baseline during the first hour (60 minutes) of NREM sleep based on centralized video-EEG reading using a log base 10 scale. Baseline was defined as the last value measured prior to intake of study treatment on Day 1. SWI was defined as the percentage of seconds with ≥1 spike-wave complex(es) during defined periods of overnight NREM sleep. |
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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 |
|---|---|---|---|---|---|---|
| Neurocrine Clinical Site | Orange | California | 92868 | United States | ||
| Neurocrine Clinical Site |
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| ID | Title | Description |
|---|---|---|
| FG000 | NBI-827104 | NBI-827104 administered orally daily for up to 13 weeks. |
| FG001 | Placebo | Placebo administered orally daily for up to 13 weeks. |
| 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 | Aug 5, 2021 | Aug 1, 2025 |
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| Placebo |
| Drug |
Non-active dosage form. |
|
| Baseline to Week 12 |
| Number of Participants Considered as Responders as Assessed by the Caregiver Global Impression of Change (CaGI-C) Score | The CaGI-C is a 7-point scale that rates the caregiver's assessment of the overall improvement in the participants symptoms since the initiation of study treatment, ranging from 1 (very much improved) to 7 (very much worse). A responder was defined as a participant with a score of 1 (very much improved) or 2 (much improved). | Week 6 and Week 12 |
| Number of Participants Considered as Responders as Assessed by the Clinician Global Impression of Change (CGI-C) Score | The CGI-C is a 7-point scale that rates the clinician's assessment of overall improvement in the participant's symptoms since the initiation of study treatment, ranging from 1 (very much improved) to 7 (very much worse). A responder was defined as a participant with a score of 1 (very much improved) or 2 (much improved). | Week 6 and Week 12 |
| Number of Participants Considered as Responders as Assessed by the Clinical Global Impression of Severity (CGI-S) Scores | The CGI-S is a 7-point scale that rates the clinician's assessment of overall symptom severity, ranging from 1 (normal, not at all ill) to 7 (among the most extremely ill patients). A responder was defined as a participant with at least 1-point improvement in the CGI-S score from baseline. | Weeks 6 and 12 |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Neurocrine Clinical Site | Washington D.C. | District of Columbia | 20010 | United States |
| Neurocrine Clinical Site | Miami | Florida | 33155 | United States |
| Neurocrine Clinical Site | Rochester | Minnesota | 55905 | United States |
| Neurocrine Clinical Site | Durham | North Carolina | 27710 | United States |
| Neurocrine Clinical Site | Cleveland | Ohio | 44195 | United States |
| Neurocrine Clinical Site | Philadelphia | Pennsylvania | 19104 | United States |
| Neurocrine Clinical Site | Calgary | Alberta | T3B 6A8 | Canada |
| Neurocrine Clinical Site | Dianalund | 4293 | Denmark |
| Neurocrine Clinical Site | Barcelona | 08950 | Spain |
| Neurocrine Clinical Site | Madrid | 28034 | Spain |
| Neurocrine Clinical Site | Basel | 4031 | Switzerland |
| Neurocrine Clinical Site | Zurich | 8032 | Switzerland |
| Neurocrine Clinical Site | London | WC1N 3JH | United Kingdom |
| Received at Least 1 Dose of Study Drug |
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| Full Analysis Set |
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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 one dose of study treatment.
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| ID | Title | Description |
|---|---|---|
| BG000 | NBI-827104 | NBI-827104 administered orally daily for up to 13 weeks. |
| BG001 | Placebo | Placebo administered orally daily for up to 13 weeks. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race/Ethnicity, Customized | 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 | Ratio of Spike-Wave Index (SWI) During First Hour of Nonrapid Eye Movement (NREM) Sleep at Week 6 | The ratio of SWI at the end of Week 6 to baseline during the first hour (60 minutes) of NREM sleep based on centralized video-electroencephalograph (EEG) reading using a log base 10 scale. Baseline was defined as the last value measured prior to intake of study treatment on Day 1. SWI was defined as the percentage of seconds with ≥1 spike-wave complex(es) during defined periods of overnight NREM sleep. | Full Analysis Set included all randomized participants who had at least 1 dose of study treatment and at least 1 efficacy video EEG assessment. The number of participants analyzed = the number of participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | Log-transformed ratio | Baseline to Week 6 |
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| Secondary | Ratio of SWI During First Hour of NREM Sleep at Week 12 | The ratio of SWI at the end of Week 12 to baseline during the first hour (60 minutes) of NREM sleep based on centralized video-EEG reading using a log base 10 scale. Baseline was defined as the last value measured prior to intake of study treatment on Day 1. SWI was defined as the percentage of seconds with ≥1 spike-wave complex(es) during defined periods of overnight NREM sleep. | Full Analysis Set included all randomized participants who had at least 1 dose of study treatment and at least 1 efficacy video EEG assessment. | Posted | Least Squares Mean | Standard Error | Log-transformed ratio | Baseline to Week 12 |
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| Secondary | Number of Participants Considered as Responders as Assessed by the Caregiver Global Impression of Change (CaGI-C) Score | The CaGI-C is a 7-point scale that rates the caregiver's assessment of the overall improvement in the participants symptoms since the initiation of study treatment, ranging from 1 (very much improved) to 7 (very much worse). A responder was defined as a participant with a score of 1 (very much improved) or 2 (much improved). | Full Analysis Set included all randomized participants who had at least 1 dose of study treatment and at least 1 efficacy video EEG assessment. | Posted | Count of Participants | Participants | Week 6 and Week 12 |
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| Secondary | Number of Participants Considered as Responders as Assessed by the Clinician Global Impression of Change (CGI-C) Score | The CGI-C is a 7-point scale that rates the clinician's assessment of overall improvement in the participant's symptoms since the initiation of study treatment, ranging from 1 (very much improved) to 7 (very much worse). A responder was defined as a participant with a score of 1 (very much improved) or 2 (much improved). | Full Analysis Set included all randomized participants who had at least 1 dose of study treatment and at least 1 efficacy video EEG assessment. | Posted | Count of Participants | Participants | Week 6 and Week 12 |
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| Secondary | Number of Participants Considered as Responders as Assessed by the Clinical Global Impression of Severity (CGI-S) Scores | The CGI-S is a 7-point scale that rates the clinician's assessment of overall symptom severity, ranging from 1 (normal, not at all ill) to 7 (among the most extremely ill patients). A responder was defined as a participant with at least 1-point improvement in the CGI-S score from baseline. | Full Analysis Set included all randomized participants who had at least 1 dose of study treatment and at least 1 efficacy video EEG assessment. | Posted | Count of Participants | Participants | Weeks 6 and 12 |
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Day 1 (after dosing) up to Week 17
Safety analysis set included all randomized participants who had 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-827104 | NBI-827104 administered orally daily for up to 13 weeks. | 0 | 16 | 1 | 16 | 11 | 16 |
| EG001 | Placebo | Placebo administered orally daily for up to 13 weeks. | 0 | 8 | 0 | 8 | 8 | 8 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Seizure | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Flushing | Vascular disorders | MedDRA 24.0 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 24.0 | Systematic Assessment |
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| Pyrexia | General disorders | MedDRA 24.0 | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Affect lability | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
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| Aggression | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
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| Depressed mood | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
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| Irritability | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
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| Restlessness | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
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| Suicidal ideation | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
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| Terminal insomnia | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
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| Alanine aminotransferase increased | Investigations | MedDRA 24.0 | Systematic Assessment |
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| Blood creatine phosphokinase increased | Investigations | MedDRA 24.0 | Systematic Assessment |
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| Blood triglycerides increased | Investigations | MedDRA 24.0 | Systematic Assessment |
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| Lymphocyte count decreased | Investigations | MedDRA 24.0 | Systematic Assessment |
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| Neutrophil count decreased | Investigations | MedDRA 24.0 | Systematic Assessment |
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| Protein urine present | Investigations | MedDRA 24.0 | Systematic Assessment |
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| White blood cell count decreased | Investigations | MedDRA 24.0 | Systematic Assessment |
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| Vitamin D decreased | Investigations | MedDRA 24.0 | Systematic Assessment |
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| Face injury | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
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| Tachycardia | Cardiac disorders | MedDRA 24.0 | Systematic Assessment |
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| Dizziness | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
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| Partial seizures | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
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| Somnolence | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
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| Tremor | Cardiac disorders | MedDRA 24.0 | Systematic Assessment |
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| Eye pain | Eye disorders | MedDRA 24.0 | Systematic Assessment |
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| Abdominal pain lower | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Haematochezia | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
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| Keratosis pilaris | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
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| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
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| Precocious puberty | Endocrine disorders | MedDRA 24.0 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
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| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Otitis media | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Rash pustular | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Suspected COVID-19 | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Viral infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
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| Decreased appetite | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
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| Tremor | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
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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 | Nov 18, 2022 | Aug 1, 2025 | SAP_001.pdf |
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| >=65 years |
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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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| Black or African American |
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| Other |
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