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This is a Phase 2b, randomized, double-blind, placebo-controlled, dose-optimization study to evaluate the efficacy, safety, and tolerability of NBI-98854 titrated to the subject's optimal dose administered once daily (qd) for a total of 12 weeks of treatment in pediatric subjects with TS.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Participants received placebo (matching valbenazine) once daily for 12 weeks. |
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| Valbenazine | Experimental | Participants received valbenazine once daily for 12 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 subjects <50 kg and 80 mg for subjects ≥50 kg to achieve an optimal dose of valbenazine for each participant. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Valbenazine | Drug | vesicular monoamine transporter 2 (VMAT2) inhibitor |
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| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline to Week 12 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) | The YGTSS is designed to rate the overall severity of motor and phonic tic symptoms across a range of dimensions: number, frequency, intensity, complexity, and interference. The YGTSS was administered by the investigator (or qualified designee) using a computer-based structured clinical interview. The TTS is the sum of the 5 motor tic items and the 5 phonic (vocal) tic items and ranges from 0 to 50, with higher scores representing greater severity. | Baseline, Week 12 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline to Week 12 in the Clinical Global Impression of Tics Severity (CGI-Tics-Severity) Score | The CGI-Tics-Severity scale is used to assess overall severity on a 7-point scale. Each of the CGI-Tics-Severity response categories was assigned a numerical score as follows: 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; 7 = Among the most extremely ill patient. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Neurocrine Clinical Site | Sun City | Arizona | 85351 | United States | ||
| Neurocrine Clinical Site |
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This study enrolled male and female pediatric participants, 6 to 17 years of age, with a Diagnostic and Statistical Manual of Mental Disorders, 4th or 5th Editions (DSM-IV or -V) diagnosis of Tourette Syndrome (TS) in the United States. The first and last participants were enrolled on 05 October 2017 and 19 July 2018, respectively.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Participants received placebo (matching valbenazine) once daily for 12 weeks. |
| FG001 | Valbenazine | Participants received valbenazine once daily for 12 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 subjects <50 kg and 80 mg for subjects ≥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 | Jul 31, 2018 | Apr 1, 2021 |
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| Placebo oral capsule | Drug | non-active dosage form |
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| Baseline, Week 12 |
| Participants Who Are a Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) Responder at Week 12 | A TTS responder is defined, on a per-visit basis, as a participant whose TTS value is reduced by at least 30% from baseline at the specified postbaseline visit. | Baseline, Week 12 |
| Participants Who Are a Clinical Global Impression of Tourette Syndrome Improvement (CGI-TS-Improvement) Responder at Week 12 | The CGI-TS-Improvement scale is used to assess overall improvement since the initiation of study drug dosing on a 7-point scale. A CGI-TS-Improvement responder is defined, on a per-visit basis, as a participant whose CGI-TS-Improvement score is 1 ("Very much improved") or 2 ("Much improved") at the specified postbaseline visit. | Week 12 |
| Rogers |
| Arkansas |
| 72758 |
| United States |
| Neurocrine Clinical Site | Anaheim | California | 92805 | United States |
| Neurocrine Clinical Site | San Diego | California | 92108 | United States |
| Neurocrine Clinical Site | Santa Clarita | California | 91321 | United States |
| Neurocrine Clinical Site | New Haven | Connecticut | 06519 | United States |
| Neurocrine Clinical Site | Hialeah | Florida | 33012 | United States |
| Neurocrine Clinical Site | Hialeah | Florida | 33013 | United States |
| Neurocrine Clinical Site | Hialeah | Florida | 33018 | United States |
| Neurocrine Clinical Site | Loxahatchee Groves | Florida | 33470 | United States |
| Neurocrine Clinical Site | Orange City | Florida | 32763 | United States |
| Neurocrine Clinical Site | Orlando | Florida | 32801 | United States |
| Neurocrine Clinical Site | Orlando | Florida | 32803 | United States |
| Neurocrine Clinical Site | Tampa | Florida | 33609 | United States |
| Neurocrine Clinical Site | Chicago | Illinois | 60634 | United States |
| Neurocrine Clinical Site | Chicago | Illinois | 60637 | 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 | Boston | Massachusetts | 02114 | 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 | Memphis | Tennessee | 38119 | United States |
| Neurocrine Clinical Site | Dallas | Texas | 75243 | United States |
| Neurocrine Clinical Site | Houston | Texas | 77058 | United States |
| Neurocrine Clinical Site | Irving | Texas | 75062 | United States |
| Neurocrine Clinical Site | Everett | Washington | 98201 | United States |
| Neurocrine Clinical Site | Seattle | Washington | 98105 | United States |
| Neurocrine Clinical Site | Spokane | Washington | 99204 | United States |
| Neurocrine Clinical Site | San Juan | PR | 00926 | Puerto Rico |
| Safety Analysis Set | The safety analysis set includes all participants who received at least one dose of study drug and have any post-baseline safety data collected. |
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| Full Analysis Set | The full analysis set includes all randomized participants who have at least one evaluable change from baseline value for Total Tic Score reported at a visit (either scheduled or mapped early termination visit) during the treatment period. |
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| COMPLETED |
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| NOT COMPLETED |
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Full analysis set, which includes all randomized participants who have at least one evaluable change from baseline value for Total Tic Score reported at a visit (either scheduled or mapped early termination visit) during the treatment period.
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Participants received placebo (matching valbenazine) once daily for 12 weeks. |
| BG001 | Valbenazine | Participants received valbenazine once daily for 12 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 subjects <50 kg and 80 mg for subjects ≥50 kg to achieve an optimal dose of valbenazine for each participant. |
| 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, Continuous | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) | The YGTSS is designed to rate the overall severity of motor and phonic tic symptoms across a range of dimensions: number, frequency, intensity, complexity, and interference. The YGTSS was administered by the investigator (or qualified designee) using a computer-based structured clinical interview. The TTS is the sum of the 5 motor tic items and the 5 phonic (vocal) tic items and ranges from 0 to 50, with higher scores representing greater severity. | Mean | Standard Deviation | score on scale |
|
| 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 | Change From Baseline to Week 12 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) | The YGTSS is designed to rate the overall severity of motor and phonic tic symptoms across a range of dimensions: number, frequency, intensity, complexity, and interference. The YGTSS was administered by the investigator (or qualified designee) using a computer-based structured clinical interview. The TTS is the sum of the 5 motor tic items and the 5 phonic (vocal) tic items and ranges from 0 to 50, with higher scores representing greater severity. | Full analysis set, which includes all randomized participants who have at least one evaluable change from baseline value for TTS reported at a visit (either scheduled or mapped early termination visit) during the treatment period. Dose levels based on weight group and each individual participant's optimized dosing titration were prespecified to be combined within each arm. | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline, Week 12 |
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| Secondary | Change From Baseline to Week 12 in the Clinical Global Impression of Tics Severity (CGI-Tics-Severity) Score | The CGI-Tics-Severity scale is used to assess overall severity on a 7-point scale. Each of the CGI-Tics-Severity response categories was assigned a numerical score as follows: 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; 7 = Among the most extremely ill patient. | Full analysis set, which includes all randomized participants who have at least one evaluable change from baseline value for TTS reported at a visit (either scheduled or mapped early termination visit) during the treatment period. Dose levels based on weight group and each individual participant's optimized dosing titration were prespecified to be combined within each arm. | Posted | Least Squares Mean | Standard Error | Score on scale | Baseline, Week 12 |
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| Secondary | Participants Who Are a Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) Responder at Week 12 | A TTS responder is defined, on a per-visit basis, as a participant whose TTS value is reduced by at least 30% from baseline at the specified postbaseline visit. | Full analysis set, which includes all randomized participants who have at least one evaluable change from baseline value for TTS reported at a visit (either scheduled or mapped early termination visit) during the treatment period. Dose levels based on weight group and each individual participant's optimized dosing titration were prespecified to be combined within each arm. Analyses include participants with outcome data available at the specified visit. | Posted | Count of Participants | Participants | Baseline, Week 12 |
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| Secondary | Participants Who Are a Clinical Global Impression of Tourette Syndrome Improvement (CGI-TS-Improvement) Responder at Week 12 | The CGI-TS-Improvement scale is used to assess overall improvement since the initiation of study drug dosing on a 7-point scale. A CGI-TS-Improvement responder is defined, on a per-visit basis, as a participant whose CGI-TS-Improvement score is 1 ("Very much improved") or 2 ("Much improved") at the specified postbaseline visit. | Full analysis set, which includes all randomized participants who have at least one evaluable change from baseline value for TTS reported at a visit (either scheduled or mapped early termination visit) during the treatment period. Dose levels based on weight group and each individual participant's optimized dosing titration were prespecified to be combined within each arm. Analyses include participants with outcome data available at the specified visit. | Posted | Count of Participants | Participants | Week 12 |
|
Up to 14 Weeks
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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 | Placebo | Participants received placebo (matching valbenazine) once daily for 12 weeks. | 0 | 62 | 1 | 62 | 17 | 62 |
| EG001 | Valbenazine | Participants received valbenazine once daily for 12 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 subjects <50 kg and 80 mg for subjects ≥50 kg to achieve an optimal dose of valbenazine for each participant. | 0 | 59 | 0 | 59 | 32 | 59 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Rash | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Fatigue | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Irritability | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Influenza | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
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| Somnolence | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
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| Anxiety | Psychiatric disorders | MedDRA (12.0) | Systematic Assessment |
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| Restlessness | Psychiatric disorders | MedDRA (12.0) | Systematic Assessment |
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| Suicidal ideation | Psychiatric disorders | MedDRA (12.0) | Systematic Assessment |
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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 | Sep 25, 2018 | Apr 1, 2021 | SAP_001.pdf |
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| 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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| 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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| Black or African American |
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| White |
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| Native Hawaiian or Other Pacific Islander |
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| Other |
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| Multiple |
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| Units | Counts |
|---|---|
| Participants |
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