Phase 2 N=85 Treatment

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

Tourette Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT03444038 ↗

Enrolled (actual)

Serious AEs

3.5%

Results posted

Feb 2022

Primary outcome: Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 71 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Valbenazine (Drug)
Age: Pediatric, Adult · 6+ yrs
Sex: All
Sponsor: Neurocrine Biosciences
Primary completion: Jul 2019

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Treatment-emergent Adverse Events (TEAEs)	71	—

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.

Eligibility Criteria

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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03444038). 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.