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

NCT03325010 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: NBI-98854-TS2003
• Study Type: interventional
• Target: 127
• Locations: 2 countries
• Sites: 35

Brief 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.

Conditions

Tourette Syndrome

Outcome Measures

Primary Outcomes (1)

• Change From Baseline to Week 12 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS)

  The YGTSS is designed to rate the overall severity of motor and phonic tic symptoms across a range of dimensions: number, frequency, intensity, complexity, and interference. The YGTSS was administered by the investigator (or qualified designee) using a computer-based structured clinical interview. The TTS is the sum of the 5 motor tic items and the 5 phonic (vocal) tic items and ranges from 0 to 50, with higher scores representing greater severity.

  Baseline, Week 12

Secondary Outcomes (3)

• Change From Baseline to Week 12 in the Clinical Global Impression of Tics Severity (CGI-Tics-Severity) Score

  Baseline, Week 12

• Participants Who Are a Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) Responder at Week 12

  Baseline, Week 12

• Participants Who Are a Clinical Global Impression of Tourette Syndrome Improvement (CGI-TS-Improvement) Responder at Week 12

  Week 12

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Participants received placebo (matching valbenazine) once daily for 12 weeks.

Drug: Placebo oral capsule

Valbenazine

EXPERIMENTAL

Participants received valbenazine once daily for 12 weeks. The starting dose was 20 mg for participants \<50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for subjects \<50 kg and 80 mg for subjects ≥50 kg to achieve an optimal dose of valbenazine for each participant.

Drug: Valbenazine

Interventions

Valbenazine DRUG

vesicular monoamine transporter 2 (VMAT2) inhibitor

Also known as: Ingrezza, NBI-98854

Valbenazine

Placebo oral capsule DRUG

non-active dosage form

Placebo

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Neurocrine Biosciences: lead

Study Sites (35)

Neurocrine Clinical Site

Sun City, Arizona, 85351, United States

Location

Neurocrine Clinical Site

Rogers, Arkansas, 72758, United States

Location

Neurocrine Clinical Site

Anaheim, California, 92805, United States

Location

Neurocrine Clinical Site

San Diego, California, 92108, United States

Location

Neurocrine Clinical Site

Santa Clarita, California, 91321, United States

Location

Neurocrine Clinical Site

New Haven, Connecticut, 06519, United States

Location

Neurocrine Clinical Site

Hialeah, Florida, 33012, United States

Location

Neurocrine Clinical Site

Hialeah, Florida, 33013, United States

Location

Neurocrine Clinical Site

Hialeah, Florida, 33018, United States

Location

Neurocrine Clinical Site

Loxahatchee Groves, Florida, 33470, United States

Location

Neurocrine Clinical Site

Orange City, Florida, 32763, United States

Location

Neurocrine Clinical Site

Orlando, Florida, 32801, United States

Location

Neurocrine Clinical Site

Orlando, Florida, 32803, United States

Location

Neurocrine Clinical Site

Tampa, Florida, 33609, United States

Location

Neurocrine Clinical Site

Chicago, Illinois, 60634, United States

Location

Neurocrine Clinical Site

Chicago, Illinois, 60637, United States

Location

Neurocrine Clinical Site

Naperville, Illinois, 60563, United States

Location

Neurocrine Clinical Site

Iowa City, Iowa, 52242, United States

Location

Neurocrine Clinical Site

Leawood, Kansas, 66206, United States

Location

Neurocrine Clinical Site

Boston, Massachusetts, 02114, United States

Location

Neurocrine Clinical Site

Lincoln, Nebraska, 68526, United States

Location

Neurocrine Clinical Site

Nashua, New Hampshire, 03060, United States

Location

Neurocrine Clinical Site

Mount Arlington, New Jersey, 07856, United States

Location

Neurocrine Clinical Site

Voorhees Township, New Jersey, 08043, United States

Location

Neurocrine Clinical Site

New York, New York, 10036, United States

Location

Neurocrine Clinical Site

The Bronx, New York, 10467, United States

Location

Neurocrine Clinical Site

Durham, North Carolina, 27705, United States

Location

Neurocrine Clinical Site

Memphis, Tennessee, 38119, United States

Location

Neurocrine Clinical Site

Dallas, Texas, 75243, United States

Location

Neurocrine Clinical Site

Houston, Texas, 77058, United States

Location

Neurocrine Clinical Site

Irving, Texas, 75062, United States

Location

Neurocrine Clinical Site

Everett, Washington, 98201, United States

Location

Neurocrine Clinical Site

Seattle, Washington, 98105, United States

Location

Neurocrine Clinical Site

Spokane, Washington, 99204, United States

Location

Neurocrine Clinical Site

San Juan, PR, 00926, Puerto Rico

Location

MeSH Terms

Conditions

Tourette Syndrome

Interventions

valbenazine

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tic Disorders; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neurodevelopmental Disorders; Mental Disorders

Results Point of Contact

• Title: Neurocrine Medical Information
• Organization: Neurocrine Biosciences

medinfo@neurocrine.com

877-641-3461

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 25, 2017
• First Posted: October 30, 2017
• Study Start: October 5, 2017
• Primary Completion: November 1, 2018
• Study Completion: November 16, 2018
• Last Updated: June 28, 2021
• Results First Posted: June 28, 2021

Record last verified: 2021-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (34); PR (1)