Trial Outcomes & Findings for Safety and Efficacy Study of NBI-98854 in Children and Adolescents With Tourette Syndrome (NCT NCT02679079)
NCT ID: NCT02679079
Last Updated: 2021-01-15
Results Overview
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
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
98 participants
Primary outcome timeframe
Baseline, Week 6
Results posted on
2021-01-15
Participant Flow
This study enrolled participants (male and female), 6 to 17 years of age, with a diagnosis of Tourette Syndrome through Diagnostic and Statistical Manual of Mental Disorders, 4th or 5th Editions (DSM-IV or -V) from 30 study centers in the United States. The first participant was enrolled on 23 March 2016 and last participant completed the study on 14 April 2017.
Participant milestones
| Measure |
Placebo
Participants received matching placebo once daily for 6 weeks.
|
NBI-98854 Low Dose
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day. Participants aged 12 to 17 years received 20 mg/day.
|
NBI-98854 High Dose
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day for the first week followed by 20 mg/day for the remainder of the treatment period. Participants aged 12 to 17 years received 20 mg/day for the first week followed by 40 mg/day for the remainder of the treatment period.
|
|---|---|---|---|
|
Overall Study
STARTED
|
33
|
32
|
33
|
|
Overall Study
ITT Analysis Set
|
31
|
32
|
32
|
|
Overall Study
COMPLETED
|
29
|
30
|
31
|
|
Overall Study
NOT COMPLETED
|
4
|
2
|
2
|
Reasons for withdrawal
| Measure |
Placebo
Participants received matching placebo once daily for 6 weeks.
|
NBI-98854 Low Dose
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day. Participants aged 12 to 17 years received 20 mg/day.
|
NBI-98854 High Dose
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day for the first week followed by 20 mg/day for the remainder of the treatment period. Participants aged 12 to 17 years received 20 mg/day for the first week followed by 40 mg/day for the remainder of the treatment period.
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
2
|
0
|
2
|
|
Overall Study
Protocol Violation
|
0
|
1
|
0
|
|
Overall Study
Withdrawal by Subject
|
2
|
1
|
0
|
Baseline Characteristics
Safety and Efficacy Study of NBI-98854 in Children and Adolescents With Tourette Syndrome
Baseline characteristics by cohort
| Measure |
Placebo
n=31 Participants
Participants received matching placebo once daily for 6 weeks.
|
NBI-98854 Low Dose
n=32 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day. Participants aged 12 to 17 years received 20 mg/day.
|
NBI-98854 High Dose
n=32 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day for the first week followed by 20 mg/day for the remainder of the treatment period. Participants aged 12 to 17 years received 20 mg/day for the first week followed by 40 mg/day for the remainder of the treatment period.
|
Total
n=95 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
11.5 Years
n=5 Participants
|
12.0 Years
n=7 Participants
|
11.6 Years
n=5 Participants
|
11.7 Years
n=4 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
20 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
27 Participants
n=5 Participants
|
21 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
75 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
11 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
26 Participants
n=5 Participants
|
28 Participants
n=7 Participants
|
30 Participants
n=5 Participants
|
84 Participants
n=4 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
5 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
27 Participants
n=5 Participants
|
31 Participants
n=7 Participants
|
29 Participants
n=5 Participants
|
87 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS)
|
30.4 Score on scale
n=5 Participants
|
32.8 Score on scale
n=7 Participants
|
32.9 Score on scale
n=5 Participants
|
32.1 Score on scale
n=4 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 6Population: The intent-to-treat (ITT) analysis set included all randomized participants who received at least one dose of study drug, had postbaseline safety data, and had a baseline (Day -1) and at least one postrandomization TTS value reported at a scheduled or mapped early termination (ET) visit during the 6-week treatment period. Dose levels based on age group were prespecified to be combined within each dose arm.
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
Outcome measures
| Measure |
NBI-98854 Low Dose
n=32 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day. Participants aged 12 to 17 years received 20 mg/day.
|
Placebo
n=31 Participants
Participants received matching placebo once daily for 6 weeks.
|
NBI-98854 High Dose
n=32 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day for the first week followed by 20 mg/day for the remainder of the treatment period. Participants aged 12 to 17 years received 20 mg/day for the first week followed by 40 mg/day for the remainder of the treatment period.
|
|---|---|---|---|
|
Change From Baseline to Week 6 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS)
|
-7.3 Score on scale
Standard Error 1.4
|
-8.8 Score on scale
Standard Error 1.5
|
-9.1 Score on scale
Standard Error 1.4
|
SECONDARY outcome
Timeframe: Week 6Population: The ITT analysis set included all randomized participants who received at least one dose of study drug, had postbaseline safety data, and had a baseline (Day -1) and at least one postrandomization TTS value reported at a scheduled or mapped early termination (ET) visit during the 6-week treatment period. Dose levels based on age group were prespecified to be combined within each dose arm.
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.
Outcome measures
| Measure |
NBI-98854 Low Dose
n=32 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day. Participants aged 12 to 17 years received 20 mg/day.
|
Placebo
n=31 Participants
Participants received matching placebo once daily for 6 weeks.
|
NBI-98854 High Dose
n=32 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day for the first week followed by 20 mg/day for the remainder of the treatment period. Participants aged 12 to 17 years received 20 mg/day for the first week followed by 40 mg/day for the remainder of the treatment period.
|
|---|---|---|---|
|
Clinical Global Impression of Tourette Syndrome (CGI-TS) - Improvement Score at Week 6
|
3.5 Score on scale
Standard Error 0.2
|
3.4 Score on scale
Standard Error 0.2
|
3.0 Score on scale
Standard Error 0.2
|
SECONDARY outcome
Timeframe: Baseline, Week 6Population: Participants with observed data in the ITT analysis set, defined as all randomized participants who received at least one dose of study drug, had postbaseline safety data, and had a baseline (Day -1) and at least one postrandomization TTS value reported at a scheduled or mapped early termination (ET) visit during the 6-week treatment period. Dose levels based on age group were prespecified to be combined within each dose arm.
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.
Outcome measures
| Measure |
NBI-98854 Low Dose
n=30 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day. Participants aged 12 to 17 years received 20 mg/day.
|
Placebo
n=29 Participants
Participants received matching placebo once daily for 6 weeks.
|
NBI-98854 High Dose
n=31 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day for the first week followed by 20 mg/day for the remainder of the treatment period. Participants aged 12 to 17 years received 20 mg/day for the first week followed by 40 mg/day for the remainder of the treatment period.
|
|---|---|---|---|
|
Participants Who Are a YGTSS TTS Responder at Week 6
|
9 Participants
|
10 Participants
|
15 Participants
|
SECONDARY outcome
Timeframe: Baseline, Week 6Population: The ITT analysis set included all randomized participants who received at least one dose of study drug, had postbaseline safety data, and had a baseline (Day -1) and at least one postrandomization TTS value reported at a scheduled or mapped early termination (ET) visit during the 6-week treatment period. Dose levels based on age group were prespecified to be combined within each dose arm.
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.
Outcome measures
| Measure |
NBI-98854 Low Dose
n=32 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day. Participants aged 12 to 17 years received 20 mg/day.
|
Placebo
n=31 Participants
Participants received matching placebo once daily for 6 weeks.
|
NBI-98854 High Dose
n=32 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day for the first week followed by 20 mg/day for the remainder of the treatment period. Participants aged 12 to 17 years received 20 mg/day for the first week followed by 40 mg/day for the remainder of the treatment period.
|
|---|---|---|---|
|
Change From Baseline to Week 6 in the YGTSS Impairment Score
|
-7.1 Score on scale
Standard Error 2.1
|
-8.5 Score on scale
Standard Error 2.1
|
-10.4 Score on scale
Standard Error 2.0
|
SECONDARY outcome
Timeframe: Baseline, Week 6Population: The ITT analysis set included all randomized participants who received at least one dose of study drug, had postbaseline safety data, and had a baseline (Day -1) and at least one postrandomization TTS value reported at a scheduled or mapped early termination (ET) visit during the 6-week treatment period. Dose levels based on age group were prespecified to be combined within each dose arm.
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.
Outcome measures
| Measure |
NBI-98854 Low Dose
n=32 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day. Participants aged 12 to 17 years received 20 mg/day.
|
Placebo
n=31 Participants
Participants received matching placebo once daily for 6 weeks.
|
NBI-98854 High Dose
n=32 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day for the first week followed by 20 mg/day for the remainder of the treatment period. Participants aged 12 to 17 years received 20 mg/day for the first week followed by 40 mg/day for the remainder of the treatment period.
|
|---|---|---|---|
|
Change From Baseline to Week 6 in the YGTSS Global Tic Severity Score
|
-14.4 Score on scale
Standard Error 3.1
|
-17.2 Score on scale
Standard Error 3.2
|
-19.5 Score on scale
Standard Error 3.1
|
SECONDARY outcome
Timeframe: Baseline, Week 6Population: Participants with observed data in the ITT analysis set, defined as all randomized participants who received at least one dose of study drug, had postbaseline safety data, and had a baseline (Day -1) and at least one postrandomization TTS value reported at a scheduled or mapped early termination (ET) visit during the 6-week treatment period. Dose levels based on age group were prespecified to be combined within each dose arm.
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.
Outcome measures
| Measure |
NBI-98854 Low Dose
n=27 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day. Participants aged 12 to 17 years received 20 mg/day.
|
Placebo
n=28 Participants
Participants received matching placebo once daily for 6 weeks.
|
NBI-98854 High Dose
n=30 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day for the first week followed by 20 mg/day for the remainder of the treatment period. Participants aged 12 to 17 years received 20 mg/day for the first week followed by 40 mg/day for the remainder of the treatment period.
|
|---|---|---|---|
|
Change From Baseline to Week 6 in the Rush Video-based Tic Rating Scale (RTRS) Total Score
|
-1.6 Score on scale
Standard Error 0.8
|
-1.2 Score on scale
Standard Error 0.8
|
-1.7 Score on scale
Standard Error 0.7
|
SECONDARY outcome
Timeframe: Baseline, Week 6Population: The ITT analysis set included all randomized participants who received at least one dose of study drug, had postbaseline safety data, and had a baseline (Day -1) and at least one postrandomization TTS value reported at a scheduled or mapped early termination (ET) visit during the 6-week treatment period. Excludes those with missing PUTS data. Dose levels based on age group were prespecified to be combined within each dose arm.
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.
Outcome measures
| Measure |
NBI-98854 Low Dose
n=32 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day. Participants aged 12 to 17 years received 20 mg/day.
|
Placebo
n=31 Participants
Participants received matching placebo once daily for 6 weeks.
|
NBI-98854 High Dose
n=31 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day for the first week followed by 20 mg/day for the remainder of the treatment period. Participants aged 12 to 17 years received 20 mg/day for the first week followed by 40 mg/day for the remainder of the treatment period.
|
|---|---|---|---|
|
Change From Baseline to Week 6 in the Premonitory Urge for Tics Scale (PUTS) Total Score
|
-1.0 Score on scale
Standard Error 0.8
|
-0.6 Score on scale
Standard Error 0.8
|
0.3 Score on scale
Standard Error 0.8
|
SECONDARY outcome
Timeframe: Baseline, Week 6Population: The ITT analysis set included all randomized participants who received at least one dose of study drug, had postbaseline safety data, and had a baseline (Day -1) and at least one postrandomization TTS value reported at a scheduled or mapped early termination (ET) visit during the 6-week treatment period. Dose levels based on age group were prespecified to be combined within each dose arm.
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.
Outcome measures
| Measure |
NBI-98854 Low Dose
n=32 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day. Participants aged 12 to 17 years received 20 mg/day.
|
Placebo
n=31 Participants
Participants received matching placebo once daily for 6 weeks.
|
NBI-98854 High Dose
n=32 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day for the first week followed by 20 mg/day for the remainder of the treatment period. Participants aged 12 to 17 years received 20 mg/day for the first week followed by 40 mg/day for the remainder of the treatment period.
|
|---|---|---|---|
|
Change From Baseline to Week 6 in the Clinical Global Impression of Tics (CGI-Tics) - Severity Score
|
-0.5 Score on scale
Standard Error 0.2
|
-1.1 Score on scale
Standard Error 0.2
|
-0.9 Score on scale
Standard Error 0.2
|
SECONDARY outcome
Timeframe: Week 6Population: Participants with observed data in the ITT analysis set, defined as all randomized participants who received at least one dose of study drug, had postbaseline safety data, and had a baseline (Day -1) and at least one postrandomization TTS value reported at a scheduled or mapped early termination (ET) visit during the 6-week treatment period. Dose levels based on age group were prespecified to be combined within each dose arm.
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.
Outcome measures
| Measure |
NBI-98854 Low Dose
n=30 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day. Participants aged 12 to 17 years received 20 mg/day.
|
Placebo
n=29 Participants
Participants received matching placebo once daily for 6 weeks.
|
NBI-98854 High Dose
n=31 Participants
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day for the first week followed by 20 mg/day for the remainder of the treatment period. Participants aged 12 to 17 years received 20 mg/day for the first week followed by 40 mg/day for the remainder of the treatment period.
|
|---|---|---|---|
|
Participants Who Are a CGI-TS-Improvement Responder at Week 6
|
6 Participants
|
6 Participants
|
11 Participants
|
Adverse Events
Placebo
Serious events: 1 serious events
Other events: 9 other events
Deaths: 0 deaths
NBI-98854 Low Dose
Serious events: 0 serious events
Other events: 15 other events
Deaths: 0 deaths
NBI-98854 High Dose
Serious events: 0 serious events
Other events: 19 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=32 participants at risk
Participants received matching placebo once daily for 6 weeks.
|
NBI-98854 Low Dose
n=32 participants at risk
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day. Participants aged 12 to 17 years received 20 mg/day.
|
NBI-98854 High Dose
n=33 participants at risk
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day for the first week followed by 20 mg/day for the remainder of the treatment period. Participants aged 12 to 17 years received 20 mg/day for the first week followed by 40 mg/day for the remainder of the treatment period.
|
|---|---|---|---|
|
Psychiatric disorders
Conversion disorder
|
3.1%
1/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
0.00%
0/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
Other adverse events
| Measure |
Placebo
n=32 participants at risk
Participants received matching placebo once daily for 6 weeks.
|
NBI-98854 Low Dose
n=32 participants at risk
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day. Participants aged 12 to 17 years received 20 mg/day.
|
NBI-98854 High Dose
n=33 participants at risk
Participants received valbenazine once daily for 6 weeks. Participants aged 6 to 11 years received 10 mg/day for the first week followed by 20 mg/day for the remainder of the treatment period. Participants aged 12 to 17 years received 20 mg/day for the first week followed by 40 mg/day for the remainder of the treatment period.
|
|---|---|---|---|
|
Nervous system disorders
Headache
|
9.4%
3/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
18.8%
6/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
9.1%
3/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Nervous system disorders
Somnolence
|
3.1%
1/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
12.5%
4/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
9.1%
3/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Nervous system disorders
Sedation
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.2%
2/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
9.1%
3/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
3.1%
1/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
15.2%
5/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Infections and infestations
Gastroenteritis viral
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.1%
2/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Infections and infestations
Sinusitis
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.1%
2/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Psychiatric disorders
Insomnia
|
3.1%
1/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.2%
2/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
9.1%
3/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Psychiatric disorders
Anxiety
|
3.1%
1/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.1%
2/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Psychiatric disorders
Suicidal ideation
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.1%
2/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Psychiatric disorders
Tic
|
6.2%
2/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
3.1%
1/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
3.0%
1/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
3.1%
1/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.1%
2/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Gastrointestinal disorders
Vomiting
|
3.1%
1/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.2%
2/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
3.0%
1/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.1%
2/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
3.1%
1/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.2%
2/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
0.00%
0/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Gastrointestinal disorders
Nausea
|
6.2%
2/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
3.0%
1/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
General disorders
Irritability
|
9.4%
3/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.2%
2/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.1%
2/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
General disorders
Fatigue
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
3.1%
1/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.1%
2/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.2%
2/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
0.00%
0/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.2%
2/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
0.00%
0/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Investigations
Neutrophil count decreased
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.2%
2/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
3.0%
1/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
6.2%
2/32 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
3.0%
1/33 • Up to 8 weeks
Includes all participants who were randomized and dispensed study drug, with 2 exclusions: (a) participants who withdrew from study and returned all previously dispensed study drug with all doses present, and (b) participants who have no postbaseline safety data collected. Dose levels based on age group were prespecified to be combined within each dose arm.
|
Additional Information
Neurocrine Medical Information
Neurocrine Biosciences, Inc.
Phone: 877-641-3461
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Generally, the PI has the right to publish results provided such publication does not violate confidentiality or IP provisions within the contract with the Sponsor. Prior to submission for publication or presentation of results, the PI must provide the Sponsor time for review. The Sponsor can request the PI to withhold or remove information from all publications. For a multi-center study, any publication of results by the PI shall not be made before the first multi-center publication.
- Publication restrictions are in place
Restriction type: OTHER