Trial Outcomes & Findings for Safety and Tolerability Study of NBI-98854 for the Treatment of Subjects With Tourette Syndrome (NCT NCT02879578)
NCT ID: NCT02879578
Last Updated: 2021-04-29
Results Overview
A TEAE is an adverse event not present prior to the initiation of study drug dosing, or is an already present event that worsens either in intensity or frequency following the initiation of study drug dosing.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
155 participants
Primary outcome timeframe
Baseline through Week 28
Results posted on
2021-04-29
Participant Flow
The study enrolled male and female children (6 to 11 years of age), adolescents (12 to 17 years of age), and adults (18 to 64 years of age) from 34 centers in the United States. Participants must have a clinical diagnosis of Tourette Syndrome (TS) and must have previously completed participation in Study NB-98854-1501 or Study NBI-98854-1505. The first and last participant were enrolled on 25 July 2016 and 14 April 2017, respectively.
Participant milestones
| Measure |
Valbenazine (Children)
Children (6 to 11 years of age) received valbenazine 10 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 20 mg or continue with the participant's current dose for the remainder of the treatment period.
|
Valbenazine (Adolescents)
Adolescents (12 to 17 years of age) received valbenazine 20 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 40 mg or continue with the participant's current dose for the remainder of the treatment period.
|
Valbenazine (Adults)
Adults (18 to 64 years of age) received valbenazine 40 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 80 mg or continue with the participant's current dose for the remainder of the treatment period.
|
|---|---|---|---|
|
Overall Study
STARTED
|
33
|
41
|
81
|
|
Overall Study
Safety Analysis Set
|
33
|
40
|
79
|
|
Overall Study
COMPLETED
|
23
|
32
|
49
|
|
Overall Study
NOT COMPLETED
|
10
|
9
|
32
|
Reasons for withdrawal
| Measure |
Valbenazine (Children)
Children (6 to 11 years of age) received valbenazine 10 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 20 mg or continue with the participant's current dose for the remainder of the treatment period.
|
Valbenazine (Adolescents)
Adolescents (12 to 17 years of age) received valbenazine 20 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 40 mg or continue with the participant's current dose for the remainder of the treatment period.
|
Valbenazine (Adults)
Adults (18 to 64 years of age) received valbenazine 40 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 80 mg or continue with the participant's current dose for the remainder of the treatment period.
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
1
|
2
|
14
|
|
Overall Study
Protocol Violation
|
0
|
1
|
1
|
|
Overall Study
Withdrawal by Subject
|
8
|
6
|
9
|
|
Overall Study
Lost to Follow-up
|
1
|
0
|
4
|
|
Overall Study
Physician Decision
|
0
|
0
|
4
|
Baseline Characteristics
Safety and Tolerability Study of NBI-98854 for the Treatment of Subjects With Tourette Syndrome
Baseline characteristics by cohort
| Measure |
Valbenazine (Children)
n=33 Participants
Children (6 to 11 years of age) received valbenazine 10 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 20 mg or continue with the participant's current dose for the remainder of the treatment period.
|
Valbenazine (Adolescents)
n=40 Participants
Adolescents (12 to 17 years of age) received valbenazine 20 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 40 mg or continue with the participant's current dose for the remainder of the treatment period.
|
Valbenazine (Adults)
n=79 Participants
Adults (18 to 64 years of age) received valbenazine 40 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 80 mg or continue with the participant's current dose for the remainder of the treatment period.
|
Total
n=152 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
9.5 Years
STANDARD_DEVIATION 1.5 • n=99 Participants
|
13.7 Years
STANDARD_DEVIATION 1.4 • n=107 Participants
|
34.6 Years
STANDARD_DEVIATION 12.9 • n=206 Participants
|
23.6 Years
STANDARD_DEVIATION 14.8 • n=7 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=99 Participants
|
8 Participants
n=107 Participants
|
25 Participants
n=206 Participants
|
40 Participants
n=7 Participants
|
|
Sex: Female, Male
Male
|
26 Participants
n=99 Participants
|
32 Participants
n=107 Participants
|
54 Participants
n=206 Participants
|
112 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
13 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
30 Participants
n=99 Participants
|
34 Participants
n=107 Participants
|
75 Participants
n=206 Participants
|
139 Participants
n=7 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race/Ethnicity, Customized
Race · American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race/Ethnicity, Customized
Race · Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Race/Ethnicity, Customized
Race · Black or African American
|
3 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
6 Participants
n=7 Participants
|
|
Race/Ethnicity, Customized
Race · White
|
30 Participants
n=99 Participants
|
35 Participants
n=107 Participants
|
76 Participants
n=206 Participants
|
141 Participants
n=7 Participants
|
|
Race/Ethnicity, Customized
Race · Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
0 Participants
n=7 Participants
|
|
Race/Ethnicity, Customized
Race · Other
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
1 Participants
n=7 Participants
|
|
Race/Ethnicity, Customized
Race · Multiple
|
0 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
3 Participants
n=7 Participants
|
PRIMARY outcome
Timeframe: Baseline through Week 28Population: Safety analysis set, which includes all participants who received at least one dose of study drug and have any postbaseline data.
A TEAE is an adverse event not present prior to the initiation of study drug dosing, or is an already present event that worsens either in intensity or frequency following the initiation of study drug dosing.
Outcome measures
| Measure |
Valbenazine (Children)
n=33 Participants
Children (6 to 11 years of age) received valbenazine 10 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 20 mg or continue with the participant's current dose for the remainder of the treatment period.
|
Valbenazine (Adolescents)
n=40 Participants
Adolescents (12 to 17 years of age) received valbenazine 20 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 40 mg or continue with the participant's current dose for the remainder of the treatment period.
|
Valbenazine (Adults)
n=79 Participants
Adults (18 to 64 years of age) received valbenazine 40 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 80 mg or continue with the participant's current dose for the remainder of the treatment period.
|
|---|---|---|---|
|
Frequency of Treatment-emergent Adverse Events (TEAEs)
|
20 Participants
|
29 Participants
|
68 Participants
|
Adverse Events
Valbenazine (Children)
Serious events: 0 serious events
Other events: 19 other events
Deaths: 0 deaths
Valbenazine (Adolescents)
Serious events: 0 serious events
Other events: 22 other events
Deaths: 0 deaths
Valbenazine (Adults)
Serious events: 2 serious events
Other events: 57 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Valbenazine (Children)
n=33 participants at risk
Children (6 to 11 years of age) received valbenazine 10 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 20 mg or continue with the participant's current dose for the remainder of the treatment period.
|
Valbenazine (Adolescents)
n=40 participants at risk
Adolescents (12 to 17 years of age) received valbenazine 20 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 40 mg or continue with the participant's current dose for the remainder of the treatment period.
|
Valbenazine (Adults)
n=79 participants at risk
Adults (18 to 64 years of age) received valbenazine 40 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 80 mg or continue with the participant's current dose for the remainder of the treatment period.
|
|---|---|---|---|
|
Nervous system disorders
Extrapyramidal disorder
|
0.00%
0/33 • Up to 28 weeks
|
0.00%
0/40 • Up to 28 weeks
|
1.3%
1/79 • Up to 28 weeks
|
|
Respiratory, thoracic and mediastinal disorders
Chronic obstructive pulmonary disease
|
0.00%
0/33 • Up to 28 weeks
|
0.00%
0/40 • Up to 28 weeks
|
1.3%
1/79 • Up to 28 weeks
|
Other adverse events
| Measure |
Valbenazine (Children)
n=33 participants at risk
Children (6 to 11 years of age) received valbenazine 10 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 20 mg or continue with the participant's current dose for the remainder of the treatment period.
|
Valbenazine (Adolescents)
n=40 participants at risk
Adolescents (12 to 17 years of age) received valbenazine 20 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 40 mg or continue with the participant's current dose for the remainder of the treatment period.
|
Valbenazine (Adults)
n=79 participants at risk
Adults (18 to 64 years of age) received valbenazine 40 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 80 mg or continue with the participant's current dose for the remainder of the treatment period.
|
|---|---|---|---|
|
Gastrointestinal disorders
Nausea
|
6.1%
2/33 • Up to 28 weeks
|
2.5%
1/40 • Up to 28 weeks
|
3.8%
3/79 • Up to 28 weeks
|
|
Gastrointestinal disorders
Vomiting
|
9.1%
3/33 • Up to 28 weeks
|
5.0%
2/40 • Up to 28 weeks
|
1.3%
1/79 • Up to 28 weeks
|
|
General disorders
Fatigue
|
15.2%
5/33 • Up to 28 weeks
|
10.0%
4/40 • Up to 28 weeks
|
16.5%
13/79 • Up to 28 weeks
|
|
General disorders
Irritability
|
12.1%
4/33 • Up to 28 weeks
|
0.00%
0/40 • Up to 28 weeks
|
2.5%
2/79 • Up to 28 weeks
|
|
General disorders
Pyrexia
|
6.1%
2/33 • Up to 28 weeks
|
2.5%
1/40 • Up to 28 weeks
|
1.3%
1/79 • Up to 28 weeks
|
|
Infections and infestations
Pharyngitis streptococcal
|
6.1%
2/33 • Up to 28 weeks
|
0.00%
0/40 • Up to 28 weeks
|
1.3%
1/79 • Up to 28 weeks
|
|
Infections and infestations
Sinusitis
|
3.0%
1/33 • Up to 28 weeks
|
5.0%
2/40 • Up to 28 weeks
|
6.3%
5/79 • Up to 28 weeks
|
|
Infections and infestations
Upper respiratory tract infection
|
6.1%
2/33 • Up to 28 weeks
|
5.0%
2/40 • Up to 28 weeks
|
13.9%
11/79 • Up to 28 weeks
|
|
Investigations
Alanine aminotransferase increased
|
6.1%
2/33 • Up to 28 weeks
|
0.00%
0/40 • Up to 28 weeks
|
0.00%
0/79 • Up to 28 weeks
|
|
Investigations
Aspartate aminotransferase increased
|
6.1%
2/33 • Up to 28 weeks
|
0.00%
0/40 • Up to 28 weeks
|
0.00%
0/79 • Up to 28 weeks
|
|
Investigations
Protein urine present
|
6.1%
2/33 • Up to 28 weeks
|
0.00%
0/40 • Up to 28 weeks
|
0.00%
0/79 • Up to 28 weeks
|
|
Investigations
Weight increased
|
6.1%
2/33 • Up to 28 weeks
|
7.5%
3/40 • Up to 28 weeks
|
3.8%
3/79 • Up to 28 weeks
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
6.1%
2/33 • Up to 28 weeks
|
0.00%
0/40 • Up to 28 weeks
|
1.3%
1/79 • Up to 28 weeks
|
|
Nervous system disorders
Akathisia
|
0.00%
0/33 • Up to 28 weeks
|
2.5%
1/40 • Up to 28 weeks
|
6.3%
5/79 • Up to 28 weeks
|
|
Nervous system disorders
Headache
|
15.2%
5/33 • Up to 28 weeks
|
12.5%
5/40 • Up to 28 weeks
|
6.3%
5/79 • Up to 28 weeks
|
|
Nervous system disorders
Lethargy
|
6.1%
2/33 • Up to 28 weeks
|
0.00%
0/40 • Up to 28 weeks
|
1.3%
1/79 • Up to 28 weeks
|
|
Nervous system disorders
Sedation
|
9.1%
3/33 • Up to 28 weeks
|
20.0%
8/40 • Up to 28 weeks
|
15.2%
12/79 • Up to 28 weeks
|
|
Nervous system disorders
Somnolence
|
9.1%
3/33 • Up to 28 weeks
|
20.0%
8/40 • Up to 28 weeks
|
20.3%
16/79 • Up to 28 weeks
|
|
Psychiatric disorders
Abnormal dreams
|
0.00%
0/33 • Up to 28 weeks
|
0.00%
0/40 • Up to 28 weeks
|
5.1%
4/79 • Up to 28 weeks
|
|
Psychiatric disorders
Depression
|
3.0%
1/33 • Up to 28 weeks
|
0.00%
0/40 • Up to 28 weeks
|
8.9%
7/79 • Up to 28 weeks
|
|
Psychiatric disorders
Insomnia
|
6.1%
2/33 • Up to 28 weeks
|
12.5%
5/40 • Up to 28 weeks
|
3.8%
3/79 • Up to 28 weeks
|
Additional Information
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