Tourette SyndromePhase 2NCT02581865

Safety and Efficacy Study of NBI-98854 in Adults With Tourette Syndrome

A Phase 2 study testing NBI-98854 in people with Tourette Syndrome, run by Neurocrine Biosciences at 30 sites in United States.

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

Phase 2, double-blind, placebo-controlled study to assess the safety and efficacy of NBI-98854 administered once daily (qd) for a total of 8 weeks of treatment. This study will enroll approximately 90 male and female subjects clinically diagnosed with Tourette Syndrome. Read more about this study

Who is it for?: Ages 18 to 64 years
Conditions the study is for: Tourette Syndrome
What is being tested?: NBI-98854
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 124 participants
Where: 30 sitesUnited States
Study dates: Nov 2015to Dec 2016
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 18 to 64 years
All sexes
No healthy volunteers
Adult

Who may be able to join

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
Have a negative urine drug screen for amphetamines, barbiturates, benzodiazepine, phencyclidine, cocaine, or opiates, 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 tachy-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 known history of substance dependence, substance (drug) or alcohol abuse

Show all 11Show fewer

Have a significant risk of suicidal or violent behavior
Are currently pregnant or breastfeeding
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
Have a blood loss ≥550 mL or donated blood within 30 days prior to screening

Trial locations and contacts

30 sites in 1 country.

City, postcode or hospital

30 sites in 1 country

United States30 sites

Study site
Albany, New York
Study site
Atlanta, Georgia
Study site
Boston, Massachusetts
Study site
Chicago, Illinois
Study site
Cincinnati, Ohio
Study site
Dallas, Texas
Study site
Gainesville, Florida
Study site
Hialeah, Florida
Study site
Irving, Texas
Study site
Kirkland, Washington
Study site
Long Beach, California
Study site
Manhasset, New York
Study site
Nashua, New Hampshire
Study site
Nashville, Tennessee
Study site
Natick, Massachusetts
Study site
National City, California
Study site
New York, New York
Study site
Norristown, Pennsylvania
Study site
Oklahoma City, Oklahoma
Study site
Orem, Utah
Study site
Salt Lake City, Utah
Study site
San Bernardino, California
Study site
San Diego, California
Study site
Spokane, Washington
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St Louis, Missouri
Study site
St. Petersburg, Florida
Study site
Staten Island, New York
Study site
Summit, New Jersey
Study site
Tampa, Florida
Study site
Upland, California

Other trials in United States

About this study

Official title: A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of NBI-98854 in Adult Subjects With Tourette Syndrome

Conditions: Tourette Syndrome
Being tested: NBI-98854, Placebo
Medical subject headings: Tourette Syndrome

What participants receive

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

NBI-98854Drug
PlaceboDrug

Study groups in NCT02581865
Study group	Role	What they receive
Placebo	Placebo comparator	Placebo administered once daily for 8 weeks Receives: Drug: Placebo
Dose Group 1	Experimental	Fixed dose administered once daily for 8 weeks Receives: Drug: NBI-98854
Dose Group 2	Experimental	Fixed dose administered once daily for 8 weeks Receives: Drug: NBI-98854

What is being measured

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

Change From Baseline to Week 8 in the YGTSS 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
Measured: Baseline, Week 8

Secondary outcomes (5)

Clinical Global Impression of Change Tourette Syndrome (CGI-TS)-Improvement Score at Week 8
The CGI-TS-Improvement scale is used to assess overall improvement since the initiation of study drug dosing on a 7-point scale. Each of the CGI-TS-Improvement response categories was assigned a numerical score as follows: 1 = Very much improved; 2 = Much improved; 3 = Minimally improved; 4 = Not changed; 5 = Minimally worse; 6 = Much worse; 7 = Very much worse.
Measured: Week 8
Change From Baseline to Week 8 in the Clinical Global Impression of Tics (CGI-Tics)-Severity Score
The CGI-Tics-Severity scale is used to assess overall severity on a 7-point scale. Each of the CGI-Tics-Severity response categories was assigned a numerical score as follows: 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; 7 = Among the most extremely ill patient.
Measured: Baseline, Week 8
Change From Baseline to Last Visit in the Rush Video-based Tic Rating Scale (RTRS) Total Score
A modified RTRS was used in this study that includes short video recordings to measure 5 tic variables: number of body areas affected, frequency of motor and phonic tics, and severity of motor and phonic tics. The RTRS total score is calculated as the sum of the 5 domain scores, and ranges from 0 to 20, with higher scores representing greater severity. The final on-treatment visit was used in subjects who discontinued prior to Week 8.
Measured: Baseline, Week 8
Change From Baseline to Week 8 in the YGTSS Global Tic Severity Score
The YGTSS Global Tic Severity score is the sum of the YGTSS TTS and the YGTSS Impairment score and ranges from 0 to 100, with higher scores representing greater severity.
Measured: Baseline, Week 8
Change From Baseline to Week 8 in the Premonitory Urge for Tics Scale (PUTS) Total Score
The PUTS is an instrument for quantifying the premonitory urge phenomena associated with tics. It consists of 9 items, each of which is scored on a 4-point scale (1=not at all true, 2=a little true, 3=pretty much true, 4=very much true). The PUTS total score is calculated as the sum of the scores for the 9 items. The maximum possible total score is 36.
Measured: Baseline, Week 8

Timeline

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

Nov 2015Dec 2016

First posted21 Oct 2015 (estimated)
Study startNov 2015
Primary completionDec 2016
Study completionDec 2016

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.
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: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 124 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02581865
Sponsor's protocol ID: NBI-98854-1505
Status last verified: Apr 2020
First submitted: 19 Oct 2015
Last updated: 7 May 2020
Results posted: Yes, on ClinicalTrials.gov

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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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 7 May 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of NBI-98854 in Adult Subjects With Tourette Syndrome
ClinicalTrials.gov ID: NCT02581865
Acronym / sponsor ID: NBI-98854-1505
Conditions: Tourette Syndrome
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 124 participants (actual)
Lead sponsor: Neurocrine BiosciencesIndustry (pharma, biotech, devices)
Study start: Nov 2015 (actual)
Primary completion: Dec 2016 (actual)
Study completion: Dec 2016 (actual)
First posted: 21 Oct 2015 (estimated)
Results: Posted 7 May 2020

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: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 3

Arm	Type	Receives
Placebo	Placebo comparator	Drug: PlaceboPlacebo administered once daily for 8 weeks
Dose Group 1	Experimental	Drug: NBI-98854Fixed dose administered once daily for 8 weeks
Dose Group 2	Experimental	Drug: NBI-98854Fixed dose administered once daily for 8 weeks

Who may qualify

Ages: 18 to 64 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (7 listed)

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

Key exclusion criteria (11 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 tachy-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

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
NBI-98854	Drug	Dose Group 1, Dose Group 2
Placebo	Drug	Placebo

Primary outcome

Change From Baseline to Week 8 in the YGTSS TTS
Time frame: Baseline, Week 8
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

5 secondary outcomes

Clinical Global Impression of Change Tourette Syndrome (CGI-TS)-Improvement Score at Week 8
Time frame: Week 8
Change From Baseline to Week 8 in the Clinical Global Impression of Tics (CGI-Tics)-Severity Score
Time frame: Baseline, Week 8
Change From Baseline to Last Visit in the Rush Video-based Tic Rating Scale (RTRS) Total Score
Time frame: Baseline, Week 8
Change From Baseline to Week 8 in the YGTSS Global Tic Severity Score
Time frame: Baseline, Week 8
Change From Baseline to Week 8 in the Premonitory Urge for Tics Scale (PUTS) Total Score
Time frame: Baseline, Week 8

Results and safety

Results posted to the registry on 7 May 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo	40	36
NBI-98854 40 mg	42	35
NBI-98854 80 mg	42	26

Primary outcome: Change From Baseline to Week 8 in the YGTSS TTS

Time frame: Baseline, Week 8. Least squares mean (Standard Error). Unit: Least Squares Mean (Standard Error)

Measure	Placebo n = 37	NBI-98854 40 mg n = 35	NBI-98854 80 mg n = 27
Value	-6.1 (1.1)	-7.4 (1.7)	-8.0 (1.1)

Placebo vs NBI-98854 40 mg: Mean Difference (Net): -1.4 (95% CI -4.9 to 2.1), p = 0.4326, Mixed Models Analysis
Placebo vs NBI-98854 80 mg: Mean Difference (Net): -2.5 (95% CI -6.2 to 1.2), p = 0.1835, Mixed Models Analysis

Population analysed: The intent-to-treat (ITT) analysis set, that includes all randomized subjects who received at least one dose of study drug, have postbaseline safety data \& have at least one post randomization Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) value at a scheduled visit or mapped early termination (ET) visit during 8-week treatment period

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: Up to 10 Weeks. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Placebo	1 of 40 (2.5%)	8 of 40 (20%)	0 of 40
NBI-98854 40 mg	1 of 42 (2.4%)	22 of 42 (52.4%)	0 of 42
NBI-98854 80 mg	2 of 42 (4.8%)	32 of 42 (76.2%)	0 of 42

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

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
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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.

The record lists no contact. Use the official record to reach the sponsor.

30 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing NBI-98854 in people with Tourette Syndrome, run by Neurocrine Biosciences at 30 sites in United States.

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 7 inclusion and 11 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 7 May 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 30 sites in United States. 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. Some participants in this study may receive a placebo. 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 7 May 2020). 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?

Change From Baseline to Week 8 in the YGTSS TTS (time frame: Baseline, Week 8). 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 NCT02581865.

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 7 May 2020.