Safety and Efficacy of NBI-98854 in Pediatric Subjects With Tourette Syndrome

NCT ID: NCT03530293

Last Updated: 2022-05-17

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

81 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-04-17

Study Completion Date

2019-07-16

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This is a Phase 2, double-blind, placebo-controlled, randomized withdrawal study to evaluate the safety and maintenance of efficacy of an optimized once-daily (qd) dose of NBI-98854 in pediatric subjects with TS.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Tourette Syndrome

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators
open-label study drug treatment period followed by blinded randomization into treatment arm or placebo arm

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Pre-randomization Valbenazine

Participants received valbenazine once daily for up to 12 weeks, depending on if and when randomization occured. 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 participants \<50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant.

Group Type EXPERIMENTAL

Valbenazine

Intervention Type DRUG

vesicular monoamine transporter 2 (VMAT2) inhibitor

Randomized Placebo

Participants received placebo (matching valbenazine) once daily from randomization (Week 8, 10, or 12) through Week 36. Randomization into this arm occurred after treatment with valbenazine once daily through randomization.

Group Type PLACEBO_COMPARATOR

Placebo oral capsule

Intervention Type DRUG

non-active dosage form

Randomized Valbenazine

Participants received their optimized dose of valbenazine once daily from randomization (Week 8, 10, or 12) through Week 36. Randomization into this arm occurred after treatment with valbenazine once daily through randomization.

Group Type EXPERIMENTAL

Valbenazine

Intervention Type DRUG

vesicular monoamine transporter 2 (VMAT2) inhibitor

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Valbenazine

vesicular monoamine transporter 2 (VMAT2) inhibitor

Intervention Type DRUG

Placebo oral capsule

non-active dosage form

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

NBI-98854

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Have a clinical diagnosis of Tourette Syndrome (TS)
2. Have at least moderate tic severity
3. Have TS symptoms that impair school, occupational, and/or social function
4. 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
5. Be in good general health
6. 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
7. 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 VMAT2 inhibitors
6. Have a blood loss ≥250 mL or donated blood within 30 days prior to screening
7. Have a known history of substance dependence, substance (drug) or alcohol abuse
8. Have a significant risk of suicidal or violent behavior
9. Have initiated Comprehensive Behavioral Intervention for Tics (CBIT) during the screening period or at baseline or plan to initiate CBIT during the study
10. Have received an investigational drug within 30 days before screening or plan to use an investigational drug (other than NBI-98854) during the study
11. Have previously participated in an NBI-98854 clinical study, except for NBI-98854-1403 or NBI-98854-1501.
12. Have HIV, hepatitis B, or hepatitis C
Minimum Eligible Age

6 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Neurocrine Biosciences

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Clinical Development Lead

Role: STUDY_DIRECTOR

Neurocrine Biosciences

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Neuricrine Clinical Site

Sun City, Arizona, United States

Site Status

Neurocrine Clinical Site

Little Rock, Arkansas, United States

Site Status

Neurocrine Clinical Site

Rogers, Arkansas, United States

Site Status

Neurocrine Clinical Site

Anaheim, California, United States

Site Status

Neurocrine Clinical Site

Fullerton, California, United States

Site Status

Neurocrine Clinical Site

San Diego, California, United States

Site Status

Neurocrine Clinical Site

Santa Ana, California, United States

Site Status

Neurocrine Clinical Site

Pueblo, Colorado, United States

Site Status

Neurocrine Clinical Site

Stamford, Connecticut, United States

Site Status

Neurocrine Clinical Site

Washington D.C., District of Columbia, United States

Site Status

Neurocrine Clinical Site

Gulf Breeze, Florida, United States

Site Status

Neurocrine Clinical Site

Hialeah, Florida, United States

Site Status

Neurocrine Clinical Site

Miami, Florida, United States

Site Status

Neurocrine Clinical Site

Miami, Florida, United States

Site Status

Neurocrine Clinical Site

North Miami, Florida, United States

Site Status

Neurocrine Clinical Site

Orlando, Florida, United States

Site Status

Neurocrine Clinical Site

Palmetto Bay, Florida, United States

Site Status

Neurocrine Clinical Site

Spring Hill, Florida, United States

Site Status

Neurocrine Clinical Site

St. Petersburg, Florida, United States

Site Status

Neurocrine Clinical Site

Tallahassee, Florida, United States

Site Status

Neurocrine Clinical Site

Atlanta, Georgia, United States

Site Status

Neurocrine Clinical Site

Fayetteville, Georgia, United States

Site Status

Neurocrine Clinical Site

Savannah, Georgia, United States

Site Status

Neurocrine Clinical Site

Chicago, Illinois, United States

Site Status

Neurocrine Clinical Site

South Bend, Indiana, United States

Site Status

Neurocrine Clinical Site

Boston, Massachusetts, United States

Site Status

Neurocrine Clinical Site

Ann Arbor, Michigan, United States

Site Status

Neurocrine Clinical Site

Bloomfield Hills, Michigan, United States

Site Status

Neurocrine Clinical Site

West Bloomfield, Michigan, United States

Site Status

Neurocrine Clinical Site

Lincoln, Nebraska, United States

Site Status

Neurocrine Clinical Site

Nashua, New Hampshire, United States

Site Status

Neurocrine Clinical Site

Cherry Hill, New Jersey, United States

Site Status

Neurocrine Clinical Site

Mount Arlington, New Jersey, United States

Site Status

Neurocrine Clinical Site

New York, New York, United States

Site Status

Neurocrine Clinical Site

South Setauket, New York, United States

Site Status

Neurocrine Clinical Site

Charlotte, North Carolina, United States

Site Status

Neurocrine Clinical Site

Mason, Ohio, United States

Site Status

Neurocrine Clinical Site

Oklahoma City, Oklahoma, United States

Site Status

Neurocrine Clinical Site

Dallas, Texas, United States

Site Status

Neurocrine Clinical Site

DeSoto, Texas, United States

Site Status

Neurocrine Clinical Site

Houston, Texas, United States

Site Status

Neurocrine Clinical Site

Houston, Texas, United States

Site Status

Neurocrine Clinical Site

Irving, Texas, United States

Site Status

Neurocrine Clinical Site

San Antonio, Texas, United States

Site Status

Neurocrine Clinical Site

Charlottesville, Virginia, United States

Site Status

Neurocrine Clinical Site

Bothell, Washington, United States

Site Status

Neurocrine Clinical Site

Everett, Washington, United States

Site Status

Neurocrine Clinical Site

Spokane, Washington, United States

Site Status

Neurocrine Clinical Site

San Juan, , Puerto Rico

Site Status

Countries

Review the countries where the study has at least one active or historical site.

United States Puerto Rico

Provided Documents

Download supplemental materials such as informed consent forms, study protocols, or participant manuals.

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

NBI-98854-TS2005

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

A Study to Prevent Infantile Spasms Relapse
NCT06819670 RECRUITING PHASE2
A Novel Approach to Infantile Spasms
NCT03347526 SUSPENDED PHASE3