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

• Valbenazine (Drug), also known as NBI-98854: vesicular monoamine transporter 2 (VMAT2) inhibitor

Primary outcomes

• 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.

Sex: All, ages: 6 Years to 18 Years

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

Participant flow

Outcome results

• Number of Participants With Treatment-emergent Adverse Events (TEAEs) (Primary), Count of participants, Participants: Valbenazine 71

Adverse events

Time frame: From baseline up to Week 28.

Serious adverse events by term

Other adverse events by term

• Farber RH, Angelov A, Kim K, Carmack T, Thai-Cuarto D, Roberts E. Clinical development of valbenazine for tics associated with Tourette syndrome. Expert Rev Neurother. 2021 Apr;21(4):393-404. doi: 10.1080/14737175.2021.1898948. Epub 2021 Apr 1. PMID 33682568

• Study Protocol (PDF, 2017-11-06)
• Statistical Analysis Plan (PDF, 2020-02-19)

• Clinical Development Lead, Study director, Neurocrine Biosciences