Tourette Syndrome
Conditions
Brief summary
Phase 2, open-label, fixed-dose titration study to evaluate the safety and tolerability of NBI-98854 administered once daily for a total of 24 weeks in children, adolescents, and adults with Tourette Syndrome (TS).
Interventions
DRUGValbenazine
Sponsors
Neurocrine Biosciences
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to 64 Years
Healthy volunteers
No
Inclusion criteria
* Have participated in and completed the NBI-98854-1501 (T-Force Green) or NBI-98854-1505 (T-Forward) Phase 2 study * Have a clinical diagnosis of Tourette Syndrome (TS) * 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 * 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 * Adolescent and adult subjects (12 to 64 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 who are on stable doses of prescribed and supervised (not as needed) benzodiazepines, opiates, or psychostimulants (for subjects with comorbid ADHD) are allowed to participate in the study * Be in good general health
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 a blood loss ≥250 mL or donated blood within 56 days prior to screening (subjects 6 to 17 years of age); have a blood loss ≥550 mL or donated blood within 30 days prior to screening (subjects 18 to 64 years of age) * 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Frequency of Treatment-emergent Adverse Events (TEAEs) | Baseline through Week 28 | 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. |
Countries
United States
Participant flow
Recruitment details
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.
Participants by arm
| Arm | Count |
|---|---|
| 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. | 33 |
| 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. | 40 |
| 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. | 79 |
| Total | 152 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Adverse Event | 1 | 2 | 14 |
| Overall Study | Lost to Follow-up | 1 | 0 | 4 |
| Overall Study | Physician Decision | 0 | 0 | 4 |
| Overall Study | Protocol Violation | 0 | 1 | 1 |
| Overall Study | Withdrawal by Subject | 8 | 6 | 9 |
Baseline characteristics
| Characteristic | Valbenazine (Children) | Valbenazine (Adolescents) | Valbenazine (Adults) | Total |
|---|---|---|---|---|
| Age, Continuous | 9.5 Years STANDARD_DEVIATION 1.5 | 13.7 Years STANDARD_DEVIATION 1.4 | 34.6 Years STANDARD_DEVIATION 12.9 | 23.6 Years STANDARD_DEVIATION 14.8 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 3 Participants | 6 Participants | 4 Participants | 13 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 30 Participants | 34 Participants | 75 Participants | 139 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Race American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Race Asian | 0 Participants | 0 Participants | 1 Participants | 1 Participants |
| Race/Ethnicity, Customized Race Black or African American | 3 Participants | 2 Participants | 1 Participants | 6 Participants |
| Race/Ethnicity, Customized Race Multiple | 0 Participants | 2 Participants | 1 Participants | 3 Participants |
| Race/Ethnicity, Customized Race Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race/Ethnicity, Customized Race Other | 0 Participants | 1 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized Race White | 30 Participants | 35 Participants | 76 Participants | 141 Participants |
| Sex: Female, Male Female | 7 Participants | 8 Participants | 25 Participants | 40 Participants |
| Sex: Female, Male Male | 26 Participants | 32 Participants | 54 Participants | 112 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 33 | 0 / 40 | 0 / 79 |
| other Total, other adverse events | 19 / 33 | 22 / 40 | 57 / 79 |
| serious Total, serious adverse events | 0 / 33 | 0 / 40 | 2 / 79 |
Outcome results
Primary
Frequency of Treatment-emergent Adverse Events (TEAEs)
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.
Time frame: Baseline through Week 28
Population: Safety analysis set, which includes all participants who received at least one dose of study drug and have any postbaseline data.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Valbenazine (Children) | Frequency of Treatment-emergent Adverse Events (TEAEs) | 20 Participants |
| Valbenazine (Adolescents) | Frequency of Treatment-emergent Adverse Events (TEAEs) | 29 Participants |
| Valbenazine (Adults) | Frequency of Treatment-emergent Adverse Events (TEAEs) | 68 Participants |