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This is a Phase 2b, multicenter, open-label study to evaluate the safety and tolerability of optimized doses of NBI-98854 administered once daily for 24 weeks in pediatric subjects with Tourette Syndrome.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Valbenazine | Experimental | Valbenazine administered once daily for up to 24 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Valbenazine | Drug | vesicular monoamine transporter 2 (VMAT2) inhibitor |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | A TEAE is an adverse event not present prior to the initiation of study drug dosing, or is an already present event that worsens either in intensity or frequency following the initiation of study drug dosing. All qualifying TEAE are reported regardless of threshold. | Baseline through Week 24 |
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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 | Sun City | Arizona | 85351 | United States | ||
| Neurocrine Clinical Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33682568 | Background | Farber RH, Angelov A, Kim K, Carmack T, Thai-Cuarto D, Roberts E. Clinical development of valbenazine for tics associated with Tourette syndrome. Expert Rev Neurother. 2021 Apr;21(4):393-404. doi: 10.1080/14737175.2021.1898948. Epub 2021 Apr 1. |
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Up to 120 male and female pediatric participants, 6 to 18 years of age, with a Diagnostic and Statistical Manual of Mental Disorders, 4th or 5th Editions (DSM-IV or -5) diagnosis of Tourette Syndrome, were planned to be enrolled. A total of 85 participants were enrolled and received at least 1 dose of study treatment.
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| ID | Title | Description |
|---|---|---|
| FG000 | Valbenazine | Participants received valbenazine once daily for up to 24 weeks. The starting dose was 20 mg for participants <50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for participants <50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant. |
| 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 6, 2017 | Jan 26, 2022 |
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| Anaheim |
| California |
| 92805 |
| United States |
| Neurocrine Clinical Site | San Diego | California | 92108 | United States |
| Neurocrine Clinical Site | Santa Ana | California | 92705 | United States |
| Neurocrine Clinical Site | Santa Clarita | California | 91321 | United States |
| Neurocrine Clinical Site | New Haven | Connecticut | 06510 | United States |
| Neurocrine Clinical Site | Boca Raton | Florida | 33431 | United States |
| Neurocrine Clinical Site | Gulf Breeze | Florida | 32561 | United States |
| Neurocrine Clinical Site | Hialeah | Florida | 33013 | United States |
| Neurocrine Clinical Site | Orlando | Florida | 32801 | United States |
| Neurocrine Clinical Site | Orlando | Florida | 32803 | United States |
| Neurocrine Clinical Site | St. Petersburg | Florida | 33701 | United States |
| Neurocrine Clinical Site | Tampa | Florida | 33614 | United States |
| Neurocrine Clinical Site | Chicago | Illinois | 60634 | United States |
| Neurocrine Clinical Site | Naperville | Illinois | 60563 | United States |
| Neurocrine Clinical Site | Iowa City | Iowa | 52242 | United States |
| Neurocrine Clinical Site | Leawood | Kansas | 66206 | United States |
| Neurocrine Clinical Site | Ann Arbor | Michigan | 48105 | United States |
| Neurocrine Clinical Site | Bloomfield Hills | Michigan | 48371 | United States |
| Neurocrine Clinical Site | St Louis | Missouri | 63110 | United States |
| Neurocrine Clinical Site | Lincoln | Nebraska | 68526 | United States |
| Neurocrine Clinical Site | Nashua | New Hampshire | 03060 | United States |
| Neurocrine Clinical Site | Mount Arlington | New Jersey | 07856 | United States |
| Neurocrine Clinical Site | Voorhees Township | New Jersey | 08043 | United States |
| Neurocrine Clinical Site | New York | New York | 10036 | United States |
| Neurocrine Clinical Site | The Bronx | New York | 10467 | United States |
| Neurocrine Clinical Site | Durham | North Carolina | 27705 | United States |
| Neurocrine Clinical Site | Mason | Ohio | 45040 | United States |
| Neurocrine Clinical Site | Oklahoma City | Oklahoma | 73112 | United States |
| Neurocrine Clinical Site | Charleston | South Carolina | 29414 | United States |
| Neurocrine Clinical Site | Dallas | Texas | 75243 | United States |
| Neurocrine Clinical Site | Houston | Texas | 77030 | United States |
| Neurocrine Clinical Site | Houston | Texas | 77058 | United States |
| Neurocrine Clinical Site | San Antonio | Texas | 78249 | United States |
| Neurocrine Clinical Site | Everett | Washington | 98201 | United States |
| Neurocrine Clinical Site | Tacoma | Washington | 98405 | United States |
| Neurocrine Clinical Site | San Juan | PR | 00926 | Puerto Rico |
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Valbenazine | Participants received valbenazine once daily for up to 24 weeks. The starting dose was 20 mg for participants <50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for participants <50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant. |
| 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/Ethnicity, Customized | Count of Participants | Participants |
|
| 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 | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | A TEAE is an adverse event not present prior to the initiation of study drug dosing, or is an already present event that worsens either in intensity or frequency following the initiation of study drug dosing. All qualifying TEAE are reported regardless of threshold. | Posted | Count of Participants | Participants | Baseline through Week 24 |
|
|
|
From baseline up to Week 28.
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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 | Valbenazine | Participants received valbenazine once daily for up to 24 weeks. The starting dose was 20 mg for participants <50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for participants <50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant. | 0 | 85 | 3 | 85 | 50 | 85 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Infected bites | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
| |
| Cognitive disorder | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Henoch-Schonlein purpura | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Irritability | General disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Sedation | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (12.0) | Systematic Assessment |
|
Generally, the PI has the right to publish results provided such publication does not violate confidentiality or IP provisions within the contract with the Sponsor. Prior to submission for publication or presentation of results, the PI must provide the Sponsor time for review. The Sponsor can request the PI to withhold or remove information from all publications. For a multi-center study, any publication of results by the PI shall not be made before the first multi-center publication.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Neurocrine Medical Information | Neurocrine Biosciences | 877-641-3461 | medinfo@neurocrine.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 19, 2020 | Jan 26, 2022 | SAP_001.pdf |
| ID | Term |
|---|---|
| D005879 | Tourette Syndrome |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D013981 | Tic Disorders |
| D009069 | Movement Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D065886 | Neurodevelopmental Disorders |
| D001523 | Mental Disorders |
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| ID | Term |
|---|---|
| C000603978 | valbenazine |
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| Unknown or Not Reported |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
|
| White |
|
| More than one race |
|
| Other |
|