Phase 2 N=127 Randomized Triple-blind Treatment

Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome

Tourette Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT03325010 ↗

Enrolled (actual)

127

Serious AEs

0.8%

Results posted

Jun 2021

Primary outcome: Primary: Change From Baseline to Week 12 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) — -6.8; -8.9 score on a scale — p=0.1768

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Valbenazine (Drug); Placebo oral capsule (Drug)
Age: Pediatric · 6+ yrs
Sex: All
Sponsor: Neurocrine Biosciences
Primary completion: Nov 2018

Outcome Measures

Outcome	Result	p-value
PRIMARY Change From Baseline to Week 12 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS)	-6.8; -8.9	0.1768
SECONDARY Change From Baseline to Week 12 in the Clinical Global Impression of Tics Severity (CGI-Tics-Severity) Score	-0.7; -1.0	0.1095
SECONDARY Participants Who Are a Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) Responder at Week 12	21; 18	0.8190
SECONDARY Participants Who Are a Clinical Global Impression of Tourette Syndrome Improvement (CGI-TS-Improvement) Responder at Week 12	12; 24	0.0008 sig

Summary

This is a Phase 2b, randomized, double-blind, placebo-controlled, dose-optimization study to evaluate the efficacy, safety, and tolerability of NBI-98854 titrated to the subject's optimal dose administered once daily (qd) for a total of 12 weeks of treatment in pediatric subjects with TS.

Eligibility Criteria

Inclusion Criteria

Have a clinical diagnosis of Tourette Syndrome (TS)
Have at least moderate tic severity
Have TS symptoms that impair school, occupational, and/or social function
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 17 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 VMAT2 inhibitors
Have a blood loss ≥250 mL or donated blood within 30 days prior to screening
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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03325010). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.