Trial Outcomes & Findings for Open-Label Safety and Tolerability Study of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome (NCT NCT03444038)
NCT ID: NCT03444038
Last Updated: 2022-02-23
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. All qualifying TEAE are reported regardless of threshold.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
85 participants
Primary outcome timeframe
Baseline through Week 24
Results posted on
2022-02-23
Participant Flow
Up to 120 male and female pediatric participants, 6 to 18 years of age, with a Diagnostic and Statistical Manual of Mental Disorders, 4th or 5th Editions (DSM-IV or -5) diagnosis of Tourette Syndrome, were planned to be enrolled. A total of 85 participants were enrolled and received at least 1 dose of study treatment.
Participant milestones
| Measure |
Valbenazine
Participants received valbenazine once daily for up to 24 weeks. The starting dose was 20 mg for participants \<50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for participants \<50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant.
|
|---|---|
|
Overall Study
STARTED
|
85
|
|
Overall Study
COMPLETED
|
56
|
|
Overall Study
NOT COMPLETED
|
29
|
Reasons for withdrawal
| Measure |
Valbenazine
Participants received valbenazine once daily for up to 24 weeks. The starting dose was 20 mg for participants \<50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for participants \<50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant.
|
|---|---|
|
Overall Study
Adverse Event
|
12
|
|
Overall Study
Withdrawal by Subject
|
7
|
|
Overall Study
Lost to Follow-up
|
1
|
|
Overall Study
Physician Decision
|
2
|
|
Overall Study
Lack of Efficacy
|
7
|
Baseline Characteristics
Open-Label Safety and Tolerability Study of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome
Baseline characteristics by cohort
| Measure |
Valbenazine
n=85 Participants
Participants received valbenazine once daily for up to 24 weeks. The starting dose was 20 mg for participants \<50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for participants \<50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant.
|
|---|---|
|
Age, Continuous
|
12.6 years
STANDARD_DEVIATION 2.6 • n=5 Participants
|
|
Sex: Female, Male
Female
|
16 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
69 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
25 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
60 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Asian
|
3 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Native Hawaiian or Other Pacific Islander
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Black or African American
|
6 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · White
|
72 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · More than one race
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Other
|
3 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline through Week 24A 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. All qualifying TEAE are reported regardless of threshold.
Outcome measures
| Measure |
Valbenazine
n=85 Participants
Participants received valbenazine once daily for up to 24 weeks. The starting dose was 20 mg for participants \<50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for participants \<50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant.
|
|---|---|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
|
71 Participants
|
Adverse Events
Valbenazine
Serious events: 3 serious events
Other events: 50 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Valbenazine
n=85 participants at risk
Participants received valbenazine once daily for up to 24 weeks. The starting dose was 20 mg for participants \<50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for participants \<50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant.
|
|---|---|
|
Infections and infestations
Infected bites
|
1.2%
1/85 • From baseline up to Week 28.
|
|
Nervous system disorders
Cognitive disorder
|
1.2%
1/85 • From baseline up to Week 28.
|
|
Skin and subcutaneous tissue disorders
Henoch-Schonlein purpura
|
1.2%
1/85 • From baseline up to Week 28.
|
Other adverse events
| Measure |
Valbenazine
n=85 participants at risk
Participants received valbenazine once daily for up to 24 weeks. The starting dose was 20 mg for participants \<50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for participants \<50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain upper
|
5.9%
5/85 • From baseline up to Week 28.
|
|
Gastrointestinal disorders
Nausea
|
10.6%
9/85 • From baseline up to Week 28.
|
|
General disorders
Fatigue
|
12.9%
11/85 • From baseline up to Week 28.
|
|
General disorders
Irritability
|
9.4%
8/85 • From baseline up to Week 28.
|
|
Nervous system disorders
Headache
|
10.6%
9/85 • From baseline up to Week 28.
|
|
Nervous system disorders
Sedation
|
11.8%
10/85 • From baseline up to Week 28.
|
|
Nervous system disorders
Somnolence
|
20.0%
17/85 • From baseline up to Week 28.
|
|
Psychiatric disorders
Depression
|
5.9%
5/85 • From baseline up to Week 28.
|
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