Tourette Syndrome
Conditions
Brief summary
This is a Phase 2b, multicenter, open-label study to evaluate the safety and tolerability of optimized doses of NBI-98854 administered once daily for 24 weeks in pediatric subjects with Tourette Syndrome.
Interventions
DRUGValbenazine
vesicular monoamine transporter 2 (VMAT2) inhibitor
Sponsors
Neurocrine Biosciences
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
1. Have participated in and completed Study NBI-98854-TS2003 2. Have a clinical diagnosis of Tourette Syndrome (TS) 3. 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 4. Be in good general health 5. Adolescent subjects (12 to 18 years of age) must have a negative urine drug screen for amphetamines, barbiturates, benzodiazepine, phencyclidine, cocaine, opiates, or cannabinoids and a negative alcohol screen 6. 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
Exclusion criteria
1. Have an active, clinically significant unstable medical condition within 1 month prior to screening 2. Have a known history of long QT syndrome or cardiac arrhythmia 3. Have a known history of neuroleptic malignant syndrome 4. Have a cancer diagnosis within 3 years prior to screening (some exceptions allowed) 5. Have an allergy, hypersensitivity, or intolerance to vesicular monoamine transporter 2 (VMAT2) inhibitors 6. Have a blood loss ≥250 mL or donated blood within 56 days prior to baseline 7. Have a known history of substance (drug) dependence, or substance or alcohol abuse 8. Have a significant risk of suicidal or violent behavior 9. 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 |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | Baseline through Week 24 | 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. |
Countries
Puerto Rico, United States
Participant flow
Recruitment details
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.
Participants by arm
| Arm | Count |
|---|---|
| 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. | 85 |
| Total | 85 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 12 |
| Overall Study | Lack of Efficacy | 7 |
| Overall Study | Lost to Follow-up | 1 |
| Overall Study | Physician Decision | 2 |
| Overall Study | Withdrawal by Subject | 7 |
Baseline characteristics
| Characteristic | Valbenazine |
|---|---|
| Age, Continuous | 12.6 years STANDARD_DEVIATION 2.6 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 25 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 60 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race/Ethnicity, Customized Race American Indian or Alaska Native | 0 Participants |
| Race/Ethnicity, Customized Race Asian | 3 Participants |
| Race/Ethnicity, Customized Race Black or African American | 6 Participants |
| Race/Ethnicity, Customized Race More than one race | 0 Participants |
| Race/Ethnicity, Customized Race Native Hawaiian or Other Pacific Islander | 1 Participants |
| Race/Ethnicity, Customized Race Other | 3 Participants |
| Race/Ethnicity, Customized Race White | 72 Participants |
| Sex: Female, Male Female | 16 Participants |
| Sex: Female, Male Male | 69 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 85 |
| other Total, other adverse events | 50 / 85 |
| serious Total, serious adverse events | 3 / 85 |
Outcome results
Primary
Number of Participants With 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. All qualifying TEAE are reported regardless of threshold.
Time frame: Baseline through Week 24
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Valbenazine | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | 71 Participants |