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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.
 
ClinicalTrials.gov Identifier: NCT03325010
Recruitment Status : Completed
First Posted : October 30, 2017
Results First Posted : June 28, 2021
Last Update Posted : June 28, 2021
Sponsor:
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 16, 2018

Resource links provided by the National Library of Medicine

Drug Information available for: Valbenazine

Arm Intervention/treatment
Placebo Comparator: Placebo
Participants received placebo (matching valbenazine) once daily for 12 weeks.
Drug: Placebo oral capsule
non-active dosage form

Experimental: 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.
Drug: Valbenazine
vesicular monoamine transporter 2 (VMAT2) inhibitor
Other Names:
  • Ingrezza
  • NBI-98854




Primary Outcome Measures :
  1. Change From Baseline to Week 12 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) [ Time Frame: Baseline, 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 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.


Secondary Outcome Measures :
  1. Change From Baseline to Week 12 in the Clinical Global Impression of Tics Severity (CGI-Tics-Severity) Score [ Time Frame: Baseline, Week 12 ]
    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.

  2. Participants Who Are a Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) Responder at Week 12 [ Time Frame: Baseline, 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.

  3. Participants Who Are a Clinical Global Impression of Tourette Syndrome Improvement (CGI-TS-Improvement) Responder at Week 12 [ Time Frame: 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.



Information from the National Library of Medicine

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

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 ClinicalTrials.gov identifier (NCT number): NCT03325010


Locations
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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
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
Sponsors and Collaborators
Neurocrine Biosciences
  Study Documents (Full-Text)

Documents provided by Neurocrine Biosciences:
Study Protocol  [PDF] July 31, 2018
Statistical Analysis Plan  [PDF] September 25, 2018

Additional Information:
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Responsible Party: Neurocrine Biosciences
ClinicalTrials.gov Identifier: NCT03325010    
Other Study ID Numbers: NBI-98854-TS2003
First Posted: October 30, 2017    Key Record Dates
Results First Posted: June 28, 2021
Last Update Posted: June 28, 2021
Last Verified: June 2021
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
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