Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03325010 |
Recruitment Status :
Completed
First Posted : October 30, 2017
Last Update Posted : March 18, 2019
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Tourette Syndrome | Drug: Valbenazine Drug: Placebo oral capsule | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 127 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Primary Purpose: | Treatment |
Official Title: | A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Dose Optimization Study to Assess the Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome |
Actual Study Start Date : | October 5, 2017 |
Actual Primary Completion Date : | November 1, 2018 |
Actual Study Completion Date : | November 18, 2018 |

Arm | Intervention/treatment |
---|---|
Placebo Comparator: Placebo
Capsule, administered once daily for 12 weeks.
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Drug: Placebo oral capsule
non-active dosage form |
Experimental: Valbenazine
Capsule, administered once daily for 12 weeks.
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Drug: Valbenazine
vesicular monoamine transporter 2 (VMAT2) inhibitor
Other Names:
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- Severity of tic symptoms assessed by Yale Global Tic Severity Scale (YGTSS) [ Time Frame: Week 12 ]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 scale also includes an impairment assessment. The YGTSS total tic score (TTS) will be the primary measure for this study and represents the sum of the 5 subcategories (number, frequency, intensity, complexity, and interference) for both motor and phonic tics, each scored from 0 to 5, with higher values representing a worsening assessment.
- Overall improvement of Tourette Syndrome with Clinical Global Impression-Tourette Syndrome (CGI-TS-Improvement) [ Time Frame: Week 12 ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 6 Years to 17 Years (Child) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Have a clinical diagnosis of Tourette Syndrome (TS)
- Have at least moderate tic severity
- Have TS symptoms that impair school, occupational, and/or social function
- If using maintenance medication(s) for TS or TS spectrum diagnoses (e.g. obsessive-compulsive disorder [OCD], Attention-Deficit Hyperactivity Disorder [ADHD]), be on stable doses
- Be in good general health
- Adolescent subjects (12 to 17 years of age) must have a negative urine drug screen for amphetamines, barbiturates, benzodiazepine, phencyclidine, cocaine, opiates, or cannabinoids and a negative alcohol screen
- Subjects of childbearing potential who do not practice total abstinence must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) consistently during the screening, treatment and follow-up periods of the study
Exclusion Criteria:
- Have an active, clinically significant unstable medical condition within 1 month prior to screening
- Have a known history of long QT syndrome or cardiac arrhythmia
- Have a known history of neuroleptic malignant syndrome
- Have a cancer diagnosis within 3 years prior to screening (some exceptions allowed)
- Have an allergy, hypersensitivity, or intolerance to VMAT2 inhibitors
- Have a blood loss ≥250 mL or donated blood within 30 days prior to screening
- Have a known history of substance dependence, substance (drug) or alcohol abuse
- Have a significant risk of suicidal or violent behavior
- Have initiated Comprehensive Behavioral Intervention for Tics (CBIT) during the screening period or at baseline or plan to initiate CBIT during the study
- Have received an investigational drug within 30 days before screening or plan to use an investigational drug (other than NBI-98854) during the study

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03325010
United States, Arizona | |
Neurocrine Clinical Site | |
Sun City, Arizona, United States, 85351 | |
United States, Arkansas | |
Neurocrine Clinical Site | |
Rogers, Arkansas, United States, 72758 | |
United States, California | |
Neurocrine Clinical Site | |
Anaheim, California, United States, 92805 | |
Neurocrine Clinical Site | |
San Diego, California, United States, 92108 | |
Neurocrine Clinical Site | |
Santa Clarita, California, United States, 91321 | |
United States, Connecticut | |
Neurocrine Clinical Site | |
New Haven, Connecticut, United States, 06519 | |
United States, Florida | |
Neurocrine Clinical Site | |
Hialeah, Florida, United States, 33012 | |
Neurocrine Clinical Site | |
Hialeah, Florida, United States, 33013 | |
Neurocrine Clinical Site | |
Hialeah, Florida, United States, 33018 | |
Neurocrine Clinical Site | |
Loxahatchee Groves, Florida, United States, 33470 | |
Neurocrine Clinical Site | |
Orange City, Florida, United States, 32763 | |
Neurocrine Clinical Site | |
Orlando, Florida, United States, 32801 | |
Neurocrine Clinical Site | |
Orlando, Florida, United States, 32803 | |
Neurocrine Clinical Site | |
Tampa, Florida, United States, 33609 | |
United States, Illinois | |
Neurocrine Clinical Site | |
Chicago, Illinois, United States, 60634 | |
Neurocrine Clinical Site | |
Chicago, Illinois, United States, 60637 | |
Neurocrine Clinical Site | |
Naperville, Illinois, United States, 60563 | |
United States, Iowa | |
Neurocrine Clinical Site | |
Iowa City, Iowa, United States, 52242 | |
United States, Kansas | |
Neurocrine Clinical Site | |
Leawood, Kansas, United States, 66206 | |
United States, Massachusetts | |
Neurocrine Clinical Site | |
Boston, Massachusetts, United States, 02114 | |
United States, Nebraska | |
Neurocrine Clinical Site | |
Lincoln, Nebraska, United States, 68526 | |
United States, New Hampshire | |
Neurocrine Clinical Site | |
Nashua, New Hampshire, United States, 03060 | |
United States, New Jersey | |
Neurocrine Clinical Site | |
Mount Arlington, New Jersey, United States, 07856 | |
Neurocrine Clinical Site | |
Voorhees, New Jersey, United States, 08043 | |
United States, New York | |
Neurocrine Clinical Site | |
Bronx, New York, United States, 10467 | |
Neurocrine Clinical Site | |
New York, New York, United States, 10036 | |
United States, North Carolina | |
Neurocrine Clinical Site | |
Durham, North Carolina, United States, 27705 | |
United States, Tennessee | |
Neurocrine Clinical Site | |
Memphis, Tennessee, United States, 38119 | |
United States, Texas | |
Neurocrine Clinical Site | |
Dallas, Texas, United States, 75243 | |
Neurocrine Clinical Site | |
Houston, Texas, United States, 77058 | |
Neurocrine Clinical Site | |
Irving, Texas, United States, 75062 | |
United States, Washington | |
Neurocrine Clinical Site | |
Everett, Washington, United States, 98201 | |
Neurocrine Clinical Site | |
Seattle, Washington, United States, 98105 | |
Neurocrine Clinical Site | |
Spokane, Washington, United States, 99204 | |
Puerto Rico | |
Neurocrine Clinical Site | |
San Juan, Puerto Rico, 00926 |
Responsible Party: | Neurocrine Biosciences |
ClinicalTrials.gov Identifier: | NCT03325010 |
Other Study ID Numbers: |
NBI-98854-TS2003 |
First Posted: | October 30, 2017 Key Record Dates |
Last Update Posted: | March 18, 2019 |
Last Verified: | March 2019 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Tourette Syndrome Syndrome Disease Pathologic Processes Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases |
Tic Disorders Movement Disorders Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn Neurodevelopmental Disorders Mental Disorders |