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Phase 2, open-label, fixed-dose titration study to evaluate the safety and tolerability of NBI-98854 administered once daily for a total of 24 weeks in children, adolescents, and adults with Tourette Syndrome (TS).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Valbenazine (Children) | Experimental | Children (6 to 11 years of age) received valbenazine 10 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 20 mg or continue with the participant's current dose for the remainder of the treatment period. |
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| Valbenazine (Adolescents) | Experimental | Adolescents (12 to 17 years of age) received valbenazine 20 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 40 mg or continue with the participant's current dose for the remainder of the treatment period. |
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| Valbenazine (Adults) | Experimental | Adults (18 to 64 years of age) received valbenazine 40 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 80 mg or continue with the participant's current dose for the remainder of the treatment period. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Valbenazine | Drug |
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| Measure | Description | Time Frame |
|---|---|---|
| Frequency of 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. | Baseline through Week 28 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Sun City | Arizona | United States | ||||
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The study enrolled male and female children (6 to 11 years of age), adolescents (12 to 17 years of age), and adults (18 to 64 years of age) from 34 centers in the United States. Participants must have a clinical diagnosis of Tourette Syndrome (TS) and must have previously completed participation in Study NB-98854-1501 or Study NBI-98854-1505. The first and last participant were enrolled on 25 July 2016 and 14 April 2017, respectively.
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| ID | Title | Description |
|---|---|---|
| FG000 | Valbenazine (Children) | Children (6 to 11 years of age) received valbenazine 10 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 20 mg or continue with the participant's current dose for the remainder of the treatment period. |
| FG001 | Valbenazine (Adolescents) |
| 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 | Mar 1, 2017 | Apr 1, 2021 |
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| San Diego |
| California |
| United States |
| Loxahatchee Groves | Florida | United States |
| St. Petersburg | Florida | United States |
| Tampa | Florida | United States |
| Naperville | Illinois | United States |
| Lincoln | Nebraska | United States |
| Summit | New Jersey | United States |
| New York | New York | United States |
| Norristown | Pennsylvania | United States |
| Nashville | Tennessee | United States |
| Fort Worth | Texas | United States |
| Spokane | Washington | United States |
Adolescents (12 to 17 years of age) received valbenazine 20 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 40 mg or continue with the participant's current dose for the remainder of the treatment period. |
| FG002 | Valbenazine (Adults) | Adults (18 to 64 years of age) received valbenazine 40 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 80 mg or continue with the participant's current dose for the remainder of the treatment period. |
| Safety Analysis Set | Safety analysis set includes all participants who received at least one dose of study drug and have any postbaseline data. |
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| COMPLETED |
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| NOT COMPLETED |
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Safety analysis set, which includes all participants who received at least one dose of study drug and have any postbaseline data.
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| ID | Title | Description |
|---|---|---|
| BG000 | Valbenazine (Children) | Children (6 to 11 years of age) received valbenazine 10 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 20 mg or continue with the participant's current dose for the remainder of the treatment period. |
| BG001 | Valbenazine (Adolescents) | Adolescents (12 to 17 years of age) received valbenazine 20 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 40 mg or continue with the participant's current dose for the remainder of the treatment period. |
| BG002 | Valbenazine (Adults) | Adults (18 to 64 years of age) received valbenazine 40 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 80 mg or continue with the participant's current dose for the remainder of the treatment period. |
| BG003 | 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 |
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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 | Frequency of 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. | Safety analysis set, which includes all participants who received at least one dose of study drug and have any postbaseline data. | Posted | Count of Participants | Participants | Baseline through Week 28 |
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Up to 28 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 | Valbenazine (Children) | Children (6 to 11 years of age) received valbenazine 10 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 20 mg or continue with the participant's current dose for the remainder of the treatment period. | 0 | 33 | 0 | 33 | 19 | 33 |
| EG001 | Valbenazine (Adolescents) | Adolescents (12 to 17 years of age) received valbenazine 20 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 40 mg or continue with the participant's current dose for the remainder of the treatment period. | 0 | 40 | 0 | 40 | 22 | 40 |
| EG002 | Valbenazine (Adults) | Adults (18 to 64 years of age) received valbenazine 40 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 80 mg or continue with the participant's current dose for the remainder of the treatment period. | 0 | 79 | 2 | 79 | 57 | 79 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Extrapyramidal disorder | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| 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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| Pyrexia | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Pharyngitis streptococcal | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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| Sinusitis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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| Alanine aminotransferase increased | Investigations | MedDRA (12.0) | Systematic Assessment |
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| Aspartate aminotransferase increased | Investigations | MedDRA (12.0) | Systematic Assessment |
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| Protein urine present | Investigations | MedDRA (12.0) | Systematic Assessment |
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| Weight increased | Investigations | MedDRA (12.0) | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
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| Akathisia | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
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| Lethargy | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
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| Sedation | 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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| Abnormal dreams | Psychiatric disorders | MedDRA (12.0) | Systematic Assessment |
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| Depression | Psychiatric disorders | MedDRA (12.0) | Systematic Assessment |
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| Insomnia | 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 | Mar 6, 2018 | Apr 1, 2021 | 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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| 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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