Safety and Tolerability Study of NBI-98854 for the Treatment of Subjects With Tourette Syndrome

Study Overview
=================
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).

Conditions
-----------------
Tourette Syndrome

Intervention / Treatment
-----------------
* Drug: Valbenazine


Participation Criteria
=================
Eligibility Criteria
-----------------
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

Ages Eligible for Study
-----------------
Minimum Age: 6 Years
Maximum Age: 64 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Valbenazine (Children)<br>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. | Drug: Valbenazine<br> <br> * Other names: NBI-98854;|
| Experimental: Valbenazine (Adolescents)<br>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. | Drug: Valbenazine<br> <br> * Other names: NBI-98854;|
| Experimental: Valbenazine (Adults)<br>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. | Drug: Valbenazine<br> <br> * Other names: NBI-98854;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | Baseline through Week 28 |