Safety and Efficacy Study of NBI-98854 in Children and Adolescents With Tourette Syndrome
A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of NBI-98854 in Pediatric Subjects With Tourette Syndrome
1 other identifier
interventional
98
1 country
44
Brief Summary
Phase 2, double-blind, placebo-controlled study to assess the safety and efficacy of NBI-98854 administered once daily (qd) for a total of 6 weeks of treatment. This study will enroll approximately 90 male and female pediatric subjects clinically diagnosed with Tourette Syndrome.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_2
Started Mar 2016
Shorter than P25 for phase_2
44 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 5, 2016
CompletedFirst Posted
Study publicly available on registry
February 10, 2016
CompletedStudy Start
First participant enrolled
March 23, 2016
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 14, 2017
CompletedStudy Completion
Last participant's last visit for all outcomes
April 14, 2017
CompletedResults Posted
Study results publicly available
January 15, 2021
CompletedJanuary 15, 2021
December 1, 2020
1.1 years
February 5, 2016
September 3, 2020
December 23, 2020
Conditions
Outcome Measures
Primary Outcomes (1)
Change From Baseline to Week 6 in the 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
Baseline, Week 6
Secondary Outcomes (8)
Clinical Global Impression of Tourette Syndrome (CGI-TS) - Improvement Score at Week 6
Week 6
Participants Who Are a YGTSS TTS Responder at Week 6
Baseline, Week 6
Change From Baseline to Week 6 in the YGTSS Impairment Score
Baseline, Week 6
Change From Baseline to Week 6 in the YGTSS Global Tic Severity Score
Baseline, Week 6
Change From Baseline to Week 6 in the Rush Video-based Tic Rating Scale (RTRS) Total Score
Baseline, Week 6
- +3 more secondary outcomes
Study Arms (3)
Placebo
PLACEBO COMPARATORAdministered once daily for 6 weeks
Dose Group 1
EXPERIMENTALAdministered once daily for 6 weeks
Dose Group 2
EXPERIMENTALAdministered once daily for 6 weeks
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (44)
Unknown Facility
Sun City, Arizona, United States
Unknown Facility
Springdale, Arkansas, United States
Unknown Facility
Long Beach, California, United States
Unknown Facility
San Bernardino, California, United States
Unknown Facility
San Diego, California, United States
Unknown Facility
Upland, California, United States
Unknown Facility
Bradenton, Florida, United States
Unknown Facility
Gainesville, Florida, United States
Unknown Facility
Hialeah, Florida, United States
Unknown Facility
Loxahatchee Groves, Florida, United States
Unknown Facility
Melbourne, Florida, United States
Unknown Facility
Orlando, Florida, United States
Unknown Facility
St. Petersburg, Florida, United States
Unknown Facility
Tampa, Florida, United States
Unknown Facility
Atlanta, Georgia, United States
Unknown Facility
Chicago, Illinois, United States
Unknown Facility
Naperville, Illinois, United States
Unknown Facility
Wichita, Kansas, United States
Unknown Facility
Boston, Massachusetts, United States
Unknown Facility
Natick, Massachusetts, United States
Unknown Facility
Ann Arbor, Michigan, United States
Unknown Facility
Bloomfield Hills, Michigan, United States
Unknown Facility
Lincoln, Nebraska, United States
Unknown Facility
Nashua, New Hampshire, United States
Unknown Facility
Marlton, New Jersey, United States
Unknown Facility
Summit, New Jersey, United States
Unknown Facility
Manhasset, New York, United States
Unknown Facility
New York, New York, United States
Unknown Facility
Rochester, New York, United States
Unknown Facility
Cincinnati, Ohio, United States
Unknown Facility
Middleburg Heights, Ohio, United States
Unknown Facility
Oklahoma City, Oklahoma, United States
Unknown Facility
Franklin, Tennessee, United States
Unknown Facility
Nashville, Tennessee, United States
Unknown Facility
Dallas, Texas, United States
Unknown Facility
DeSoto, Texas, United States
Unknown Facility
Fort Worth, Texas, United States
Unknown Facility
Houston, Texas, United States
Unknown Facility
Irving, Texas, United States
Unknown Facility
Orem, Utah, United States
Unknown Facility
Salt Lake City, Utah, United States
Unknown Facility
Kirkland, Washington, United States
Unknown Facility
Seattle, Washington, United States
Unknown Facility
Spokane, Washington, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Neurocrine Medical Information
- Organization
- Neurocrine Biosciences, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 5, 2016
First Posted
February 10, 2016
Study Start
March 23, 2016
Primary Completion
April 14, 2017
Study Completion
April 14, 2017
Last Updated
January 15, 2021
Results First Posted
January 15, 2021
Record last verified: 2020-12