Back to resultsSafety and Efficacy Study of NBI-98854 in Adults With Tourette Syndrome
Primary Purpose
Tourette Syndrome
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
NBI-98854
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Tourette Syndrome
Eligibility Criteria
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
- Have a negative urine drug screen for amphetamines, barbiturates, benzodiazepine, phencyclidine, cocaine, or opiates, 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 tachy-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 known history of substance dependence, substance (drug) or alcohol abuse
- Have a significant risk of suicidal or violent behavior
- Are currently pregnant or breastfeeding
- 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
- Have a blood loss ≥550 mL or donated blood within 30 days prior to screening
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Placebo Comparator
Experimental
Experimental
Arm Label
Placebo
Dose Group 1
Dose Group 2
Arm Description
Placebo administered once daily for 8 weeks
Fixed dose administered once daily for 8 weeks
Fixed dose administered once daily for 8 weeks
Outcomes
Primary Outcome Measures
Change From Baseline to Week 8 in the YGTSS 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
Secondary Outcome Measures
Clinical Global Impression of Change Tourette Syndrome (CGI-TS)-Improvement Score at Week 8
The CGI-TS-Improvement scale is used to assess overall improvement since the initiation of study drug dosing on a 7-point scale. Each of the CGI-TS-Improvement response categories was assigned a numerical score as follows: 1 = Very much improved; 2 = Much improved; 3 = Minimally improved; 4 = Not changed; 5 = Minimally worse; 6 = Much worse; 7 = Very much worse.
Change From Baseline to Week 8 in the Clinical Global Impression of Tics (CGI-Tics)-Severity Score
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.
Change From Baseline to Last Visit in the Rush Video-based Tic Rating Scale (RTRS) Total Score
A modified RTRS was used in this study that includes short video recordings to measure 5 tic variables: number of body areas affected, frequency of motor and phonic tics, and severity of motor and phonic tics. The RTRS total score is calculated as the sum of the 5 domain scores, and ranges from 0 to 20, with higher scores representing greater severity. The final on-treatment visit was used in subjects who discontinued prior to Week 8.
Change From Baseline to Week 8 in the YGTSS Global Tic Severity Score
The YGTSS Global Tic Severity score is the sum of the YGTSS TTS and the YGTSS Impairment score and ranges from 0 to 100, with higher scores representing greater severity.
Change From Baseline to Week 8 in the Premonitory Urge for Tics Scale (PUTS) Total Score
The PUTS is an instrument for quantifying the premonitory urge phenomena associated with tics. It consists of 9 items, each of which is scored on a 4-point scale (1=not at all true, 2=a little true, 3=pretty much true, 4=very much true). The PUTS total score is calculated as the sum of the scores for the 9 items. The maximum possible total score is 36.
Full Information
NCT ID
NCT02581865
First Posted
October 19, 2015
Last Updated
April 27, 2020
Sponsor
Neurocrine Biosciences
1. Study Identification
Unique Protocol Identification Number
NCT02581865
Brief Title
Safety and Efficacy Study of NBI-98854 in Adults With Tourette Syndrome
Official Title
A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of NBI-98854 in Adult Subjects With Tourette Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
April 2020
Overall Recruitment Status
Completed
Study Start Date
November 2015 (Actual)
Primary Completion Date
December 2016 (Actual)
Study Completion Date
December 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neurocrine Biosciences
4. Oversight
5. Study Description
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 8 weeks of treatment. This study will enroll approximately 90 male and female subjects clinically diagnosed with Tourette Syndrome.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Tourette Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
124 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo administered once daily for 8 weeks
Arm Title
Dose Group 1
Arm Type
Experimental
Arm Description
Fixed dose administered once daily for 8 weeks
Arm Title
Dose Group 2
Arm Type
Experimental
Arm Description
Fixed dose administered once daily for 8 weeks
Intervention Type
Drug
Intervention Name(s)
NBI-98854
Intervention Type
Drug
Intervention Name(s)
Placebo
Primary Outcome Measure Information:
Title
Change From Baseline to Week 8 in the YGTSS TTS
Description
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
Time Frame
Baseline, Week 8
Secondary Outcome Measure Information:
Title
Clinical Global Impression of Change Tourette Syndrome (CGI-TS)-Improvement Score at Week 8
Description
The CGI-TS-Improvement scale is used to assess overall improvement since the initiation of study drug dosing on a 7-point scale. Each of the CGI-TS-Improvement response categories was assigned a numerical score as follows: 1 = Very much improved; 2 = Much improved; 3 = Minimally improved; 4 = Not changed; 5 = Minimally worse; 6 = Much worse; 7 = Very much worse.
Time Frame
Week 8
Title
Change From Baseline to Week 8 in the Clinical Global Impression of Tics (CGI-Tics)-Severity Score
Description
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.
Time Frame
Baseline, Week 8
Title
Change From Baseline to Last Visit in the Rush Video-based Tic Rating Scale (RTRS) Total Score
Description
A modified RTRS was used in this study that includes short video recordings to measure 5 tic variables: number of body areas affected, frequency of motor and phonic tics, and severity of motor and phonic tics. The RTRS total score is calculated as the sum of the 5 domain scores, and ranges from 0 to 20, with higher scores representing greater severity. The final on-treatment visit was used in subjects who discontinued prior to Week 8.
Time Frame
Baseline, Week 8
Title
Change From Baseline to Week 8 in the YGTSS Global Tic Severity Score
Description
The YGTSS Global Tic Severity score is the sum of the YGTSS TTS and the YGTSS Impairment score and ranges from 0 to 100, with higher scores representing greater severity.
Time Frame
Baseline, Week 8
Title
Change From Baseline to Week 8 in the Premonitory Urge for Tics Scale (PUTS) Total Score
Description
The PUTS is an instrument for quantifying the premonitory urge phenomena associated with tics. It consists of 9 items, each of which is scored on a 4-point scale (1=not at all true, 2=a little true, 3=pretty much true, 4=very much true). The PUTS total score is calculated as the sum of the scores for the 9 items. The maximum possible total score is 36.
Time Frame
Baseline, Week 8
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
64 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Have a negative urine drug screen for amphetamines, barbiturates, benzodiazepine, phencyclidine, cocaine, or opiates, 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 tachy-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 known history of substance dependence, substance (drug) or alcohol abuse
Have a significant risk of suicidal or violent behavior
Are currently pregnant or breastfeeding
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
Have a blood loss ≥550 mL or donated blood within 30 days prior to screening
Facility Information:
City
Long Beach
State/Province
California
Country
United States
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National City
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California
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United States
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San Bernardino
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California
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United States
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San Diego
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California
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United States
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Upland
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California
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United States
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Gainesville
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United States
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Hialeah
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Saint Petersburg
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Tampa
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United States
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Atlanta
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Georgia
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Chicago
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Illinois
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Boston
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Massachusetts
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Natick
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Massachusetts
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Saint Louis
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Missouri
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Nashua
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New Hampshire
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Summit
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New Jersey
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United States
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Albany
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New York
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United States
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Manhasset
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New York
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United States
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New York
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New York
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United States
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Staten Island
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New York
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Cincinnati
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Ohio
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Oklahoma City
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Oklahoma
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Norristown
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Pennsylvania
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United States
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Nashville
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Tennessee
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United States
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Dallas
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United States
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Irving
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Texas
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United States
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Orem
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Utah
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United States
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Salt Lake City
State/Province
Utah
Country
United States
City
Kirkland
State/Province
Washington
Country
United States
City
Spokane
State/Province
Washington
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Safety and Efficacy Study of NBI-98854 in Adults With Tourette Syndrome
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