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

Study Overview
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Brief Summary
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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.

Official Title
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Open-Label Safety and Tolerability Study of Optimized Doses of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome

Conditions
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Tourette Syndrome

Intervention / Treatment
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* Drug: Valbenazine


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Have participated in and completed Study NBI-98854-TS2003 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 Be in good general health 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 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: 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 an allergy, hypersensitivity, or intolerance to vesicular monoamine transporter 2 (VMAT2) inhibitors Have a blood loss ≥250 mL or donated blood within 56 days prior to baseline Have a known history of substance (drug) dependence, or substance or alcohol abuse Have a significant risk of suicidal or violent behavior 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
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Minimum Age: 6 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Valbenazine<br>Valbenazine administered once daily for up to 24 weeks | Drug: Valbenazine<br>* vesicular monoamine transporter 2 (VMAT2) inhibitor<br>* Other names: NBI-98854;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | Baseline through Week 24 |