Tourette SyndromePhase 2NCT03444038

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

A Phase 2 study testing Valbenazine in people with Tourette Syndrome, run by Neurocrine Biosciences at 37 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 6 to 18 years
Conditions the study is for: Tourette Syndrome
What is being tested?: Valbenazine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 85 participants
Where: 37 sitesin 2 countries
Study dates: 8 Feb 2018to 12 Jul 2019
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 6 to 18 years
All sexes
No healthy volunteers
Child, Adult

Who may be able to join

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

Who cannot join

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

Show all 9Show fewer

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

Trial locations and contacts

37 sites in 2 countries.

Country

City, postcode or hospital

37 sites in 2 countries

United States36 sites

Neurocrine Clinical Site
Anaheim, California, 92805
Neurocrine Clinical Site
Ann Arbor, Michigan, 48105
Neurocrine Clinical Site
Bloomfield Hills, Michigan, 48371
Neurocrine Clinical Site
Boca Raton, Florida, 33431
Neurocrine Clinical Site
Charleston, South Carolina, 29414
Neurocrine Clinical Site
Chicago, Illinois, 60634
Neurocrine Clinical Site
Dallas, Texas, 75243
Neurocrine Clinical Site
Durham, North Carolina, 27705
Neurocrine Clinical Site
Everett, Washington, 98201
Neurocrine Clinical Site
Gulf Breeze, Florida, 32561
Neurocrine Clinical Site
Hialeah, Florida, 33013
Neurocrine Clinical Site
Houston, Texas, 77030
Neurocrine Clinical Site
Houston, Texas, 77058
Neurocrine Clinical Site
Iowa City, Iowa, 52242
Neurocrine Clinical Site
Leawood, Kansas, 66206
Neurocrine Clinical Site
Lincoln, Nebraska, 68526
Neurocrine Clinical Site
Mason, Ohio, 45040
Neurocrine Clinical Site
Mount Arlington, New Jersey, 07856
Neurocrine Clinical Site
Naperville, Illinois, 60563
Neurocrine Clinical Site
Nashua, New Hampshire, 03060
Neurocrine Clinical Site
New Haven, Connecticut, 06510
Neurocrine Clinical Site
New York, New York, 10036
Neurocrine Clinical Site
Oklahoma City, Oklahoma, 73112
Neurocrine Clinical Site
Orlando, Florida, 32801
Neurocrine Clinical Site
Orlando, Florida, 32803
Neurocrine Clinical Site
San Antonio, Texas, 78249
Neurocrine Clinical Site
San Diego, California, 92108
Neurocrine Clinical Site
Santa Ana, California, 92705
Neurocrine Clinical Site
Santa Clarita, California, 91321
Neurocrine Clinical Site
St Louis, Missouri, 63110
Neurocrine Clinical Site
St. Petersburg, Florida, 33701
Neurocrine Clinical Site
Sun City, Arizona, 85351
Neurocrine Clinical Site
Tacoma, Washington, 98405
Neurocrine Clinical Site
Tampa, Florida, 33614
Neurocrine Clinical Site
The Bronx, New York, 10467
Neurocrine Clinical Site
Voorhees Township, New Jersey, 08043

Other trials in United States

Puerto Rico1 site

Neurocrine Clinical Site
San Juan, PR, 00926

Other trials in Puerto Rico

About this study

Official title: Open-Label Safety and Tolerability Study of Optimized Doses of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome

Conditions: Tourette Syndrome
Being tested: Valbenazine
Medical subject headings: Tourette Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

ValbenazineDrug
Also called NBI-98854
vesicular monoamine transporter 2 (VMAT2) inhibitor

Study groups in NCT03444038
Study group	Role	What they receive
Valbenazine	Experimental	Valbenazine administered once daily for up to 24 weeks Receives: Drug: Valbenazine

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Baseline through Week 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

8 Feb 201812 Jul 2019

First posted23 Feb 2018
Study start8 Feb 2018
Primary completion12 Jul 2019
Study completion12 Jul 2019

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Tourette SyndromeStudies of the same condition, open and under way.
More trials run by Neurocrine BiosciencesThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 85 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03444038
Sponsor's protocol ID: NBI-98854-TS2004
Status last verified: Jan 2022
First submitted: 19 Feb 2018
Last updated: 23 Feb 2022
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

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.
PubMed 33682568

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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Evaluating and Promoting Resilience in Adolescents With Tourette Syndrome
Tourette Syndrome, Tic Disorders
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Transcutaneous Auricular Vagus Nerve Stimulation for Chronic Tic Disorders/Tourette's Syndrome in Children and Adolescents
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Deep Brain Stimulation Therapy in Movement Disorders
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Recruiting
Exploring the Role of Neuroactive Steroids in Tourette Syndrome
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All Tourette Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 23 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Open-Label Safety and Tolerability Study of Optimized Doses of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome
ClinicalTrials.gov ID: NCT03444038
Acronym / sponsor ID: NBI-98854-TS2004
Conditions: Tourette Syndrome
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 85 participants (actual)
Lead sponsor: Neurocrine BiosciencesIndustry (pharma, biotech, devices)
Study start: 8 Feb 2018 (actual)
Primary completion: 12 Jul 2019 (actual)
Study completion: 12 Jul 2019 (actual)
First posted: 23 Feb 2018 (actual)
Results: Posted 23 Feb 2022

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Valbenazine	Experimental	Drug: ValbenazineValbenazine administered once daily for up to 24 weeks

Who may qualify

Ages: 6 to 18 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult

Key inclusion criteria (6 listed)

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

Key exclusion criteria (9 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
ValbenazineAlso: NBI-98854vesicular monoamine transporter 2 (VMAT2) inhibitor	Drug	Valbenazine

Primary outcome

Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Time frame: 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.

Results and safety

Results posted to the registry on 23 Feb 2022, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Valbenazine	85	56

Primary outcome: Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Time frame: Baseline through Week 24. Count of participants. Unit: Participants

Measure	Valbenazine n = 85
Value	71

Adverse events

Time frame: From baseline up to Week 28.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Valbenazine	3 of 85 (3.5%)	50 of 85 (58.8%)	0 of 85

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 23 Feb 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 33682568Farber 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.
Individual participant dataSharing planned: No

Related trials for Tourette Syndrome

Not yet recruiting
Evaluating and Promoting Resilience in Adolescents With Tourette Syndrome
Tourette Syndrome, Tic Disorders
Not yet recruiting
Transcutaneous Auricular Vagus Nerve Stimulation for Chronic Tic Disorders/Tourette's Syndrome in Children and Adolescents
Tourette Syndrome, Chronic Tic Disorder
Recruiting
Deep Brain Stimulation Therapy in Movement Disorders
Parkinson's Disease
Recruiting
Exploring the Role of Neuroactive Steroids in Tourette Syndrome
Tourette Syndrome in Children
Active, not recruiting
Tourette Deep Brain Stimulation (DBS) Target Detection & Suppression
Tourette Syndrome

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Clinical Development Lead study director

Neurocrine Biosciences

37 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Valbenazine in people with Tourette Syndrome, run by Neurocrine Biosciences at 37 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 23 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 37 sites in United States, Puerto Rico. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 23 Feb 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Treatment-emergent Adverse Events (TEAEs) (time frame: Baseline through Week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03444038, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Feb 2022.