NCT03444038

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
85

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Feb 2018

Shorter than P25 for phase_2

Geographic Reach
2 countries

37 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

February 8, 2018

Completed
11 days until next milestone

First Submitted

Initial submission to the registry

February 19, 2018

Completed
4 days until next milestone

First Posted

Study publicly available on registry

February 23, 2018

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 12, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 12, 2019

Completed
2.6 years until next milestone

Results Posted

Study results publicly available

February 23, 2022

Completed
Last Updated

February 23, 2022

Status Verified

January 1, 2022

Enrollment Period

1.4 years

First QC Date

February 19, 2018

Results QC Date

January 29, 2022

Last Update Submit

January 29, 2022

Conditions

Outcome Measures

Primary Outcomes (1)

  • Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    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.

    Baseline through Week 24

Study Arms (1)

Valbenazine

EXPERIMENTAL

Valbenazine administered once daily for up to 24 weeks

Drug: Valbenazine

Interventions

vesicular monoamine transporter 2 (VMAT2) inhibitor

Also known as: NBI-98854
Valbenazine

Eligibility Criteria

Age6 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

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 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (37)

Neurocrine Clinical Site

Sun City, Arizona, 85351, 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 Ana, California, 92705, United States

Location

Neurocrine Clinical Site

Santa Clarita, California, 91321, United States

Location

Neurocrine Clinical Site

New Haven, Connecticut, 06510, United States

Location

Neurocrine Clinical Site

Boca Raton, Florida, 33431, United States

Location

Neurocrine Clinical Site

Gulf Breeze, Florida, 32561, United States

Location

Neurocrine Clinical Site

Hialeah, Florida, 33013, United States

Location

Neurocrine Clinical Site

Orlando, Florida, 32801, United States

Location

Neurocrine Clinical Site

Orlando, Florida, 32803, United States

Location

Neurocrine Clinical Site

St. Petersburg, Florida, 33701, United States

Location

Neurocrine Clinical Site

Tampa, Florida, 33614, United States

Location

Neurocrine Clinical Site

Chicago, Illinois, 60634, 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

Ann Arbor, Michigan, 48105, United States

Location

Neurocrine Clinical Site

Bloomfield Hills, Michigan, 48371, United States

Location

Neurocrine Clinical Site

St Louis, Missouri, 63110, 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

Mason, Ohio, 45040, United States

Location

Neurocrine Clinical Site

Oklahoma City, Oklahoma, 73112, United States

Location

Neurocrine Clinical Site

Charleston, South Carolina, 29414, United States

Location

Neurocrine Clinical Site

Dallas, Texas, 75243, United States

Location

Neurocrine Clinical Site

Houston, Texas, 77030, United States

Location

Neurocrine Clinical Site

Houston, Texas, 77058, United States

Location

Neurocrine Clinical Site

San Antonio, Texas, 78249, United States

Location

Neurocrine Clinical Site

Everett, Washington, 98201, United States

Location

Neurocrine Clinical Site

Tacoma, Washington, 98405, United States

Location

Neurocrine Clinical Site

San Juan, PR, 00926, Puerto Rico

Location

Related Publications (1)

  • Farber RH, Angelov A, Kim K, Carmack T, Thai-Cuarto D, Roberts E. Clinical development of valbenazine for tics associated with Tourette syndrome. Expert Rev Neurother. 2021 Apr;21(4):393-404. doi: 10.1080/14737175.2021.1898948. Epub 2021 Apr 1.

    PMID: 33682568BACKGROUND

MeSH Terms

Conditions

Tourette Syndrome

Interventions

valbenazine

Condition Hierarchy (Ancestors)

Basal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesTic DisordersMovement DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNeurodevelopmental DisordersMental Disorders

Results Point of Contact

Title
Neurocrine Medical Information
Organization
Neurocrine Biosciences

Study Officials

  • Clinical Development Lead

    Neurocrine Biosciences

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 19, 2018

First Posted

February 23, 2018

Study Start

February 8, 2018

Primary Completion

July 12, 2019

Study Completion

July 12, 2019

Last Updated

February 23, 2022

Results First Posted

February 23, 2022

Record last verified: 2022-01

Data Sharing

IPD Sharing
Will not share

Locations