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

Phase 2

Completed

• Conditions: Tourette Syndrome
• Interventions: Drug: Valbenazine

• Registration Number: NCT03444038
• Lead Sponsor: Neurocrine Biosciences

Brief 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2018-02-08
• Study First Submitted: 2018-02-19
• Study First Submitted QC: 2018-02-19
• Study First Posted: 2018-02-23
• Primary Completion: 2019-07-12
• Study Completion: 2019-07-12
• Disposition First Submitted: 2020-01-30
• Disposition First Submitted QC: 2020-02-10
• Disposition First Posted: 2020-02-17
• Status Verified: 2022-01
• Results First Submitted: 2022-01-29
• Results First Submitted QC: 2022-01-29
• Last Update Submitted: 2022-01-29
• Results First Posted: 2022-02-23
• Last Update Posted: 2022-02-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 85

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Valbenazine	Valbenazine	Valbenazine administered once daily for up to 24 weeks

Primary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

Neurocrine Clinical Site; 🇵🇷 San Juan, Puerto Rico