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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
98
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
Time frame: Baseline, Week 6
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.
Time frame: Week 6
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.
Time frame: Baseline, Week 6
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.
Time frame: Baseline, Week 6
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.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Unnamed facility
Sun City, Arizona, United States
Unnamed facility
Springdale, Arkansas, United States
Unnamed facility
Long Beach, California, United States
Unnamed facility
San Bernardino, California, United States
Unnamed facility
San Diego, California, United States
Unnamed facility
Upland, California, United States
Unnamed facility
Bradenton, Florida, United States
Unnamed facility
Gainesville, Florida, United States
Unnamed facility
Hialeah, Florida, United States
Unnamed facility
Loxahatchee Groves, Florida, United States
...and 34 more locations
Time frame: Baseline, Week 6
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.
Time frame: Baseline, 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.
Time frame: Baseline, Week 6
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.
Time frame: Baseline, Week 6
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.
Time frame: Week 6