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Phase 2, double-blind, placebo-controlled study to assess the safety and efficacy of NBI-98854 administered once daily (qd) for a total of 8 weeks of treatment. This study will enroll approximately 90 male and female subjects clinically diagnosed with Tourette Syndrome.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | Placebo administered once daily for 8 weeks |
|
| Dose Group 1 | Experimental | Fixed dose administered once daily for 8 weeks |
|
| Dose Group 2 | Experimental | Fixed dose administered once daily for 8 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NBI-98854 | Drug |
| ||
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline to Week 8 in the YGTSS 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 8 |
| Measure | Description | Time Frame |
|---|---|---|
| Clinical Global Impression of Change Tourette Syndrome (CGI-TS)-Improvement Score at Week 8 | The CGI-TS-Improvement scale is used to assess overall improvement since the initiation of study drug dosing on a 7-point scale. Each of the CGI-TS-Improvement response categories was assigned a numerical score as follows: 1 = Very much improved; 2 = Much improved; 3 = Minimally improved; 4 = Not changed; 5 = Minimally worse; 6 = Much worse; 7 = Very much worse. |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Long Beach | California | United States | ||||
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This study enrolled participants (male and female) with a diagnosis of Tourette Syndrome through Diagnostic and Statistical Manual of Mental Disorders, 4th or 5th Editions (DSM-IV or -V) from approximately 40 study centers in the United States
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Placebo administered once daily for 8 weeks |
| FG001 | NBI-98854 40 mg | Fixed dose of NBI-98854 40 mg administered once daily for 8 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 | Jun 29, 2016 | Mar 31, 2020 |
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|
| Week 8 |
| Change From Baseline to Week 8 in the Clinical Global Impression of Tics (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. | Baseline, Week 8 |
| Change From Baseline to Last Visit in the Rush Video-based Tic Rating Scale (RTRS) Total Score | A modified RTRS was used in this study that includes short video recordings to measure 5 tic variables: number of body areas affected, frequency of motor and phonic tics, and severity of motor and phonic tics. The RTRS total score is calculated as the sum of the 5 domain scores, and ranges from 0 to 20, with higher scores representing greater severity. The final on-treatment visit was used in subjects who discontinued prior to Week 8. | Baseline, Week 8 |
| Change From Baseline to Week 8 in the YGTSS Global Tic Severity Score | The YGTSS Global Tic Severity score is the sum of the YGTSS TTS and the YGTSS Impairment score and ranges from 0 to 100, with higher scores representing greater severity. | Baseline, Week 8 |
| Change From Baseline to Week 8 in the Premonitory Urge for Tics Scale (PUTS) Total Score | The PUTS is an instrument for quantifying the premonitory urge phenomena associated with tics. It consists of 9 items, each of which is scored on a 4-point scale (1=not at all true, 2=a little true, 3=pretty much true, 4=very much true). The PUTS total score is calculated as the sum of the scores for the 9 items. The maximum possible total score is 36. | Baseline, Week 8 |
| National City |
| California |
| United States |
| San Bernardino | California | United States |
| San Diego | California | United States |
| Upland | California | United States |
| Gainesville | Florida | United States |
| Hialeah | Florida | United States |
| St. Petersburg | Florida | United States |
| Tampa | Florida | United States |
| Atlanta | Georgia | United States |
| Chicago | Illinois | United States |
| Boston | Massachusetts | United States |
| Natick | Massachusetts | United States |
| St Louis | Missouri | United States |
| Nashua | New Hampshire | United States |
| Summit | New Jersey | United States |
| Albany | New York | United States |
| Manhasset | New York | United States |
| New York | New York | United States |
| Staten Island | New York | United States |
| Cincinnati | Ohio | United States |
| Oklahoma City | Oklahoma | United States |
| Norristown | Pennsylvania | United States |
| Nashville | Tennessee | United States |
| Dallas | Texas | United States |
| Irving | Texas | United States |
| Orem | Utah | United States |
| Salt Lake City | Utah | United States |
| Kirkland | Washington | United States |
| Spokane | Washington | United States |
| FG002 | NBI-98854 80 mg | NBI-98854 80 mg dose will be titrated in a blinded fashion (subjects will receive 40 mg for the first week followed by 80 mg for the remainder of the 8-week treatment period) |
| COMPLETED |
|
| NOT COMPLETED |
|
the intent-to-treat (ITT) analysis set, that includes all randomized subjects who received at least one dose of study drug, have postbaseline safety data & have at least one post randomization Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) value at a scheduled visit or mapped early termination (ET) visit during 8-week treatment period
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Placebo administered once daily for 8 weeks |
| BG001 | NBI-98854 40 mg | Fixed dose of NBI-98854 40 mg administered once daily for 8 weeks |
| BG002 | NBI-98854 80 mg | NBI-98854 80 mg dose will be titrated in a blinded fashion (subjects will receive 40 mg for the first week followed by 80 mg for the remainder of the 8-week treatment period) |
| BG003 | 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 | Full Range | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| ||||||||||||||||
| Body mass index (BMI) at Screening | Mean | Full Range | kg/m^2 (kilogram/meter^2) |
| |||||||||||||||
| 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 | Full Range | 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 8 in the YGTSS 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 | The intent-to-treat (ITT) analysis set, that includes all randomized subjects who received at least one dose of study drug, have postbaseline safety data & have at least one post randomization Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) value at a scheduled visit or mapped early termination (ET) visit during 8-week treatment period | Posted | Least Squares Mean | Standard Error | Least Squares Mean (Standard Error) | Baseline, Week 8 |
|
|
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Clinical Global Impression of Change Tourette Syndrome (CGI-TS)-Improvement Score at Week 8 | The CGI-TS-Improvement scale is used to assess overall improvement since the initiation of study drug dosing on a 7-point scale. Each of the CGI-TS-Improvement response categories was assigned a numerical score as follows: 1 = Very much improved; 2 = Much improved; 3 = Minimally improved; 4 = Not changed; 5 = Minimally worse; 6 = Much worse; 7 = Very much worse. | the intent-to-treat (ITT) analysis set, that includes all randomized subjects who received at least one dose of study drug, have postbaseline safety data & have at least one post randomization Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) value at a scheduled visit or mapped early termination (ET) visit during 8-week treatment period | Posted | Least Squares Mean | Standard Error | score on a scale | Week 8 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline to Week 8 in the Clinical Global Impression of Tics (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. | the intent-to-treat (ITT) analysis set, that includes all randomized subjects who received at least one dose of study drug, have postbaseline safety data & have at least one post randomization Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) value at a scheduled visit or mapped early termination (ET) visit during 8-week treatment period | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline, Week 8 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline to Last Visit in the Rush Video-based Tic Rating Scale (RTRS) Total Score | A modified RTRS was used in this study that includes short video recordings to measure 5 tic variables: number of body areas affected, frequency of motor and phonic tics, and severity of motor and phonic tics. The RTRS total score is calculated as the sum of the 5 domain scores, and ranges from 0 to 20, with higher scores representing greater severity. The final on-treatment visit was used in subjects who discontinued prior to Week 8. | the intent-to-treat (ITT) analysis set, that includes all randomized subjects who received at least one dose of study drug, have postbaseline safety data & have at least one post randomization Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) value at a scheduled visit or mapped early termination (ET) visit during 8-week treatment period | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline, Week 8 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline to Week 8 in the YGTSS Global Tic Severity Score | The YGTSS Global Tic Severity score is the sum of the YGTSS TTS and the YGTSS Impairment score and ranges from 0 to 100, with higher scores representing greater severity. | the intent-to-treat (ITT) analysis set, that includes all randomized subjects who received at least one dose of study drug, have postbaseline safety data & have at least one post randomization Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) value at a scheduled visit or mapped early termination (ET) visit during 8-week treatment period | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline, Week 8 |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline to Week 8 in the Premonitory Urge for Tics Scale (PUTS) Total Score | The PUTS is an instrument for quantifying the premonitory urge phenomena associated with tics. It consists of 9 items, each of which is scored on a 4-point scale (1=not at all true, 2=a little true, 3=pretty much true, 4=very much true). The PUTS total score is calculated as the sum of the scores for the 9 items. The maximum possible total score is 36. | The analysis population include intent-to-treat (ITT):all subjects in the safety analysis set with at least one post-randomization TTS value reported at a scheduled visit or mapped Early Termination (ET) visit during the treatment period. | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline, Week 8 |
|
Up to 10 Weeks
All TEAEs,whether observed by investigator,reported by subject,noted from lab. findings,or identified by other means, were recorded from time subject signed ICF until subject's follow-up visit(Week 10 or ET).
The analysis set include all subjects who are randomized to a treatment group and dispensed study drug, with two exclusions:(a)subjects who withdraw from study & return all previously dispensed study drug with all doses present,&(b) subjects who have no postbaseline safety data collected.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Placebo administered once daily for 8 weeks | 0 | 40 | 1 | 40 | 8 | 40 |
| EG001 | NBI-98854 40 mg | Fixed dose of NBI-98854 40 mg administered once daily for 8 weeks | 0 | 42 | 1 | 42 | 22 | 42 |
| EG002 | NBI-98854 80 mg | NBI-98854 80 mg dose was titrated in a blinded fashion (subjects will receive 40 mg for the first week followed by 80 mg for the remainder of the 8-week treatment period | 0 | 42 | 2 | 42 | 32 | 42 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Septic shock | Infections and infestations | MedDRA Version 12.0 | Systematic Assessment |
| |
| Pelvic inflammatory disease | Infections and infestations | MedDRA Version 12.0 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA Version 12.0 | Systematic Assessment |
| |
| Brachial plexus injury | Injury, poisoning and procedural complications | MedDRA Version 12.0 | Systematic Assessment |
| |
| Pneumonia streptococcal | Infections and infestations | MedDRA Version 12.0 | Systematic Assessment |
| |
| Renal failure acute | Renal and urinary disorders | MedDRA Version 12.0 | Systematic Assessment |
| |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | MedDRA Version 12.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Somnolence | Nervous system disorders | MedDRA Version 12.0 | Systematic Assessment |
| |
| Akathisia | Nervous system disorders | MedDRA Version 12.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA Version 12.0 | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA Version 12.0 | Systematic Assessment |
| |
| Sedation | Nervous system disorders | MedDRA Version 12.0 | Systematic Assessment |
| |
| Disturbance in attention | Nervous system disorders | MedDRA Version 12.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA Version 12.0 | Systematic Assessment | The Organ system term used to generate the source table is General disorders and administration site conditions |
|
| Anxiety | Psychiatric disorders | MedDRA Version 12.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA Version 12.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA Version 12.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA Version 12.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA Version 12.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA Version 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, Inc. | 877-641-3461 | medinfo@neurocrine.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 14, 2016 | Mar 31, 2020 | 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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Not provided
| ID | Term |
|---|---|
| C000603978 | valbenazine |
Not provided
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Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Black or African American |
|
| White |
|
| Native Hawaiian or Other Pacific Islander |
|
| Other |
|
| American Indian or Alaska Native, White |
|
| Black or African American, White |
|
| White, Other: German |
|
| Other: Thai/White |
|
| Mean Difference (Net) |
| -2.5 |
| Standard Error of the Mean |
| 1.9 |
| 2-Sided |
| 95 |
| -6.2 |
| 1.2 |
| Superiority |
|
|
|
|
|
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
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| Units | Counts |
|---|---|
| Participants |
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