Back to resultsSafety and Efficacy Study of NBI-98854 in Children and Adolescents 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
- 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
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
Administered once daily for 6 weeks
Administered once daily for 6 weeks
Administered once daily for 6 weeks
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
Clinical Global Impression of Tourette Syndrome (CGI-TS) - Improvement Score at Week 6
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.
Participants Who Are a YGTSS TTS Responder at Week 6
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.
Change From Baseline to Week 6 in the YGTSS Impairment Score
The YGTSS Impairment item is used to rate impairment due to tics using the following 50-point anchored scale: 0 = None; 10 = Minimal; 20 = Mild; 30 = Moderate; 40 = Marked; 50 = Severe.
Change From Baseline to Week 6 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 6 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 participants who discontinued prior to Week 6.
Change From Baseline to Week 6 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 total score ranges from 9 to 36, with higher scores indicating a worse outcome.
Change From Baseline to Week 6 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.
Participants Who Are a CGI-TS-Improvement Responder at Week 6
A participant is classified as a CGI-TS-Improvement responder at a given visit if their CGI-TS-Improvement score is either a "1" ("very much improved") or a "2" ("much improved") at the visit.
Full Information
NCT ID
NCT02679079
First Posted
February 5, 2016
Last Updated
December 23, 2020
Sponsor
Neurocrine Biosciences
1. Study Identification
Unique Protocol Identification Number
NCT02679079
Brief Title
Safety and Efficacy Study of NBI-98854 in Children and Adolescents With Tourette Syndrome
Official Title
A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of NBI-98854 in Pediatric Subjects With Tourette Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
December 2020
Overall Recruitment Status
Completed
Study Start Date
March 23, 2016 (Actual)
Primary Completion Date
April 14, 2017 (Actual)
Study Completion Date
April 14, 2017 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neurocrine Biosciences
4. Oversight
Data Monitoring Committee
Yes
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 6 weeks of treatment. This study will enroll approximately 90 male and female pediatric 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
98 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Administered once daily for 6 weeks
Arm Title
Dose Group 1
Arm Type
Experimental
Arm Description
Administered once daily for 6 weeks
Arm Title
Dose Group 2
Arm Type
Experimental
Arm Description
Administered once daily for 6 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 6 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (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 6
Secondary Outcome Measure Information:
Title
Clinical Global Impression of Tourette Syndrome (CGI-TS) - Improvement Score at Week 6
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 6
Title
Participants Who Are a YGTSS TTS Responder at Week 6
Description
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.
Time Frame
Baseline, Week 6
Title
Change From Baseline to Week 6 in the YGTSS Impairment Score
Description
The YGTSS Impairment item is used to rate impairment due to tics using the following 50-point anchored scale: 0 = None; 10 = Minimal; 20 = Mild; 30 = Moderate; 40 = Marked; 50 = Severe.
Time Frame
Baseline, Week 6
Title
Change From Baseline to Week 6 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 6
Title
Change From Baseline to Week 6 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 participants who discontinued prior to Week 6.
Time Frame
Baseline, Week 6
Title
Change From Baseline to Week 6 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 total score ranges from 9 to 36, with higher scores indicating a worse outcome.
Time Frame
Baseline, Week 6
Title
Change From Baseline to Week 6 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 6
Title
Participants Who Are a CGI-TS-Improvement Responder at Week 6
Description
A participant is classified as a CGI-TS-Improvement responder at a given visit if their CGI-TS-Improvement score is either a "1" ("very much improved") or a "2" ("much improved") at the visit.
Time Frame
Week 6
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
17 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
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
Facility Information:
City
Sun City
State/Province
Arizona
Country
United States
City
Springdale
State/Province
Arkansas
Country
United States
City
Long Beach
State/Province
California
Country
United States
City
San Bernardino
State/Province
California
Country
United States
City
San Diego
State/Province
California
Country
United States
City
Upland
State/Province
California
Country
United States
City
Bradenton
State/Province
Florida
Country
United States
City
Gainesville
State/Province
Florida
Country
United States
City
Hialeah
State/Province
Florida
Country
United States
City
Loxahatchee Groves
State/Province
Florida
Country
United States
City
Melbourne
State/Province
Florida
Country
United States
City
Orlando
State/Province
Florida
Country
United States
City
Saint Petersburg
State/Province
Florida
Country
United States
City
Tampa
State/Province
Florida
Country
United States
City
Atlanta
State/Province
Georgia
Country
United States
City
Chicago
State/Province
Illinois
Country
United States
City
Naperville
State/Province
Illinois
Country
United States
City
Wichita
State/Province
Kansas
Country
United States
City
Boston
State/Province
Massachusetts
Country
United States
City
Natick
State/Province
Massachusetts
Country
United States
City
Ann Arbor
State/Province
Michigan
Country
United States
City
Bloomfield Hills
State/Province
Michigan
Country
United States
City
Lincoln
State/Province
Nebraska
Country
United States
City
Nashua
State/Province
New Hampshire
Country
United States
City
Marlton
State/Province
New Jersey
Country
United States
City
Summit
State/Province
New Jersey
Country
United States
City
Manhasset
State/Province
New York
Country
United States
City
New York
State/Province
New York
Country
United States
City
Rochester
State/Province
New York
Country
United States
City
Cincinnati
State/Province
Ohio
Country
United States
City
Middleburg Heights
State/Province
Ohio
Country
United States
City
Oklahoma City
State/Province
Oklahoma
Country
United States
City
Franklin
State/Province
Tennessee
Country
United States
City
Nashville
State/Province
Tennessee
Country
United States
City
Dallas
State/Province
Texas
Country
United States
City
DeSoto
State/Province
Texas
Country
United States
City
Fort Worth
State/Province
Texas
Country
United States
City
Houston
State/Province
Texas
Country
United States
City
Irving
State/Province
Texas
Country
United States
City
Orem
State/Province
Utah
Country
United States
City
Salt Lake City
State/Province
Utah
Country
United States
City
Kirkland
State/Province
Washington
Country
United States
City
Seattle
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 Children and Adolescents With Tourette Syndrome
We'll reach out to this number within 24 hrs