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

NCT ID: NCT03444038

Last Updated: 2022-02-23

Study Results

Results available

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

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

85 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-02-08

Study Completion Date

2019-07-12

Brief Summary

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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

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Conditions

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Tourette Syndrome

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Valbenazine

Valbenazine administered once daily for up to 24 weeks

Group Type EXPERIMENTAL

Valbenazine

Intervention Type DRUG

vesicular monoamine transporter 2 (VMAT2) inhibitor

Interventions

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Valbenazine

vesicular monoamine transporter 2 (VMAT2) inhibitor

Intervention Type DRUG

Other Intervention Names

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NBI-98854

Eligibility Criteria

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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
Minimum Eligible Age

6 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Neurocrine Biosciences

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Development Lead

Role: STUDY_DIRECTOR

Neurocrine Biosciences

Locations

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Neurocrine Clinical Site

Sun City, Arizona, United States

Site Status

Neurocrine Clinical Site

Anaheim, California, United States

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San Diego, California, United States

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Santa Ana, California, United States

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Santa Clarita, California, United States

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New Haven, Connecticut, United States

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Boca Raton, Florida, United States

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Gulf Breeze, Florida, United States

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Hialeah, Florida, United States

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Orlando, Florida, United States

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Orlando, Florida, United States

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St. Petersburg, Florida, United States

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Tampa, Florida, United States

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Chicago, Illinois, United States

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Naperville, Illinois, United States

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Iowa City, Iowa, United States

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Leawood, Kansas, United States

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Ann Arbor, Michigan, United States

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Bloomfield Hills, Michigan, United States

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St Louis, Missouri, United States

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Lincoln, Nebraska, United States

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Nashua, New Hampshire, United States

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Mount Arlington, New Jersey, United States

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Voorhees Township, New Jersey, United States

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New York, New York, United States

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The Bronx, New York, United States

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Durham, North Carolina, United States

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Neurocrine Clinical Site

Mason, Ohio, United States

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Oklahoma City, Oklahoma, United States

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Neurocrine Clinical Site

Charleston, South Carolina, United States

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Neurocrine Clinical Site

Dallas, Texas, United States

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Houston, Texas, United States

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Neurocrine Clinical Site

Houston, Texas, United States

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Neurocrine Clinical Site

San Antonio, Texas, United States

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Neurocrine Clinical Site

Everett, Washington, United States

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Neurocrine Clinical Site

Tacoma, Washington, United States

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Neurocrine Clinical Site

San Juan, PR, Puerto Rico

Site Status

Countries

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United States Puerto Rico

References

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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.

Reference Type BACKGROUND
PMID: 33682568 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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NBI-98854-TS2004

Identifier Type: -

Identifier Source: org_study_id

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