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Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome

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. Identifier: NCT03325010
Recruitment Status : Completed
First Posted : October 30, 2017
Last Update Posted : March 18, 2019
Information provided by (Responsible Party):
Neurocrine Biosciences

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Tourette Syndrome Drug: Valbenazine Drug: Placebo oral capsule Phase 2

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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

Resource links provided by the National Library of Medicine

Drug Information available for: Valbenazine

Arm Intervention/treatment
Placebo Comparator: Placebo
Capsule, administered once daily for 12 weeks.
Drug: Placebo oral capsule
non-active dosage form

Experimental: Valbenazine
Capsule, administered once daily for 12 weeks.
Drug: Valbenazine
vesicular monoamine transporter 2 (VMAT2) inhibitor
Other Names:
  • Ingrezza
  • NBI-98854

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. Overall improvement of Tourette Syndrome with Clinical Global Impression-Tourette Syndrome (CGI-TS-Improvement) [ Time Frame: Week 12 ]

Information from the National Library of Medicine

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.

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Ages Eligible for Study:   6 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Have a clinical diagnosis of Tourette Syndrome (TS)
  2. Have at least moderate tic severity
  3. Have TS symptoms that impair school, occupational, and/or social function
  4. 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
  5. Be in good general health
  6. 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
  7. 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:

  1. Have an active, clinically significant unstable medical condition within 1 month prior to screening
  2. Have a known history of long QT syndrome or cardiac arrhythmia
  3. Have a known history of neuroleptic malignant syndrome
  4. Have a cancer diagnosis within 3 years prior to screening (some exceptions allowed)
  5. Have an allergy, hypersensitivity, or intolerance to VMAT2 inhibitors
  6. Have a blood loss ≥250 mL or donated blood within 30 days prior to screening
  7. Have a known history of substance dependence, substance (drug) or alcohol abuse
  8. Have a significant risk of suicidal or violent behavior
  9. Have initiated Comprehensive Behavioral Intervention for Tics (CBIT) during the screening period or at baseline or plan to initiate CBIT during the study
  10. Have received an investigational drug within 30 days before screening or plan to use an investigational drug (other than NBI-98854) during the study

Information from the National Library of Medicine

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 identifier (NCT number): NCT03325010

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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
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Anaheim, California, United States, 92805
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San Diego, California, United States, 92108
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Santa Clarita, California, United States, 91321
United States, Connecticut
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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
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Orlando, Florida, United States, 32801
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Orlando, Florida, United States, 32803
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Tampa, Florida, United States, 33609
United States, Illinois
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Chicago, Illinois, United States, 60634
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Chicago, Illinois, United States, 60637
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Naperville, Illinois, United States, 60563
United States, Iowa
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Iowa City, Iowa, United States, 52242
United States, Kansas
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Leawood, Kansas, United States, 66206
United States, Massachusetts
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Boston, Massachusetts, United States, 02114
United States, Nebraska
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Lincoln, Nebraska, United States, 68526
United States, New Hampshire
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Nashua, New Hampshire, United States, 03060
United States, New Jersey
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Mount Arlington, New Jersey, United States, 07856
Neurocrine Clinical Site
Voorhees, New Jersey, United States, 08043
United States, New York
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Bronx, New York, United States, 10467
Neurocrine Clinical Site
New York, New York, United States, 10036
United States, North Carolina
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Durham, North Carolina, United States, 27705
United States, Tennessee
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Memphis, Tennessee, United States, 38119
United States, Texas
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Dallas, Texas, United States, 75243
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Houston, Texas, United States, 77058
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Irving, Texas, United States, 75062
United States, Washington
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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
Sponsors and Collaborators
Neurocrine Biosciences
Additional Information:
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Responsible Party: Neurocrine Biosciences 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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Tourette Syndrome
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