Tourette SyndromePhase 2NCT03530293
Safety and Efficacy of NBI-98854 in Pediatric Subjects With Tourette Syndrome
A Phase 2 study testing Valbenazine in people with Tourette Syndrome, run by Neurocrine Biosciences at 49 sites in 2 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Tourette Syndrome
- What is being tested?
- Valbenazine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 81 participants
- Where
- 49 sitesin 2 countries
- Study dates
- 17 Apr 2018to 16 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 17 years
- All sexes
- No healthy volunteers
- Child
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
- 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
- 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 VMAT2 inhibitors
- Have a blood loss ≥250 mL or donated blood within 30 days prior to screening
Show all 12Show fewer
- Have a known history of substance dependence, substance (drug) or alcohol abuse
- Have a significant risk of suicidal or violent behavior
- 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 previously participated in an NBI-98854 clinical study, except for NBI-98854-1403 or NBI-98854-1501.
- Have HIV, hepatitis B, or hepatitis C
Trial locations and contacts
49 sites in 2 countries.
49 sites in 2 countries
United States48 sites
Neurocrine Clinical Site
Anaheim, California, 92805
Neurocrine Clinical Site
Ann Arbor, Michigan, 48015
Neurocrine Clinical Site
Atlanta, Georgia, 30338
Neurocrine Clinical Site
Bloomfield Hills, Michigan, 48302
Neurocrine Clinical Site
Boston, Massachusetts, 02114
Neurocrine Clinical Site
Bothell, Washington, 98011
Neurocrine Clinical Site
Charlotte, North Carolina, 29141
Neurocrine Clinical Site
Charlottesville, Virginia, 22903
Neurocrine Clinical Site
Cherry Hill, New Jersey, 08002
Neurocrine Clinical Site
Chicago, Illinois, 60634
Neurocrine Clinical Site
Dallas, Texas, 75243
Neurocrine Clinical Site
DeSoto, Texas, 75115
Neurocrine Clinical Site
Everett, Washington, 98201
Neurocrine Clinical Site
Fayetteville, Georgia, 30214
Neurocrine Clinical Site
Fullerton, California, 92835
Neurocrine Clinical Site
Gulf Breeze, Florida, 32561
Neurocrine Clinical Site
Hialeah, Florida, 33012
Neurocrine Clinical Site
Houston, Texas, 77030
Neurocrine Clinical Site
Houston, Texas, 77058
Neurocrine Clinical Site
Irving, Texas, 75062
Neurocrine Clinical Site
Lincoln, Nebraska, 68526
Neurocrine Clinical Site
Little Rock, Arkansas, 72205
Neurocrine Clinical Site
Mason, Ohio, 45040
Neurocrine Clinical Site
Miami, Florida, 33125
Neurocrine Clinical Site
Miami, Florida, 33136
Neurocrine Clinical Site
Mount Arlington, New Jersey, 07856
Neurocrine Clinical Site
Nashua, New Hampshire, 03060
Neurocrine Clinical Site
New York, New York, 10036
Neurocrine Clinical Site
North Miami, Florida, 33161
Neurocrine Clinical Site
Oklahoma City, Oklahoma, 73112
Neurocrine Clinical Site
Orlando, Florida, 32803
Neurocrine Clinical Site
Palmetto Bay, Florida, 33157
Neurocrine Clinical Site
Pueblo, Colorado, 81003
Neurocrine Clinical Site
Rogers, Arkansas, 72758
Neurocrine Clinical Site
San Antonio, Texas, 78249
Neurocrine Clinical Site
San Diego, California, 92108
Neurocrine Clinical Site
Santa Ana, California, 92705
Neurocrine Clinical Site
Savannah, Georgia, 31406
Neurocrine Clinical Site
South Bend, Indiana, 46617
Neurocrine Clinical Site
South Setauket, New York, 11720
Neurocrine Clinical Site
Spokane, Washington, 99202
Neurocrine Clinical Site
Spring Hill, Florida, 34609
Neurocrine Clinical Site
St. Petersburg, Florida, 33701
Neurocrine Clinical Site
Stamford, Connecticut, 06905
Neuricrine Clinical Site
Sun City, Arizona, 85351
Neurocrine Clinical Site
Tallahassee, Florida, 32308
Neurocrine Clinical Site
Washington D.C., District of Columbia, 22207
Neurocrine Clinical Site
West Bloomfield, Michigan, 48322
About this study
Official title: A Phase 2, Double-Blind, Placebo-Controlled, Randomized Withdrawal Study to Evaluate the Safety and Efficacy of NBI-98854 in Pediatric Subjects With Tourette Syndrome
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.
- Conditions
- Tourette Syndrome
- Being tested
- Valbenazine, Placebo oral capsule
- 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
Placebo oral capsuleDrug
non-active dosage form
| Study group | Role | What they receive |
|---|---|---|
| Pre-randomization Valbenazine | Experimental | 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. Receives: Drug: Valbenazine |
| Randomized Placebo | Placebo comparator | 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. Receives: Drug: Placebo oral capsule |
| Randomized Valbenazine | Experimental | 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. Receives: Drug: Valbenazine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to Loss of Treatment Response
Loss of treatment response during the withdrawal period was defined as: 2 consecutive visits with 1) an increase in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) of greater than 35% or 7 points from the randomized withdrawal period baseline and 2) an increase in CGI-Tics-Severity score of ≥2 points from the randomized withdrawal period baseline; or discontinuation due to lack of efficacy or a treatment-emergent adverse event (TEAE) of worsening of tics. Median (lower and upper quartiles) Kaplan-Meier estimates for the time to loss of treatment response were not able to be calculated because of the low incidence of loss of treatment response events.
Measured: Randomization (Week 8, 10 or 12) through Week 36
Secondary outcomes (4)
Change From Randomization Baseline to the 8 Weeks Post-randomization Timepoint 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. Least-squares mean were estimated using a mixed-effects model for repeated measures.
Measured: Randomization Baseline (Week 8, 10 or 12); 8 weeks post-randomization
Change From Randomization Baseline to the Week 36 Visit 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: Randomization Baseline (Week 8, 10 or 12); Week 36
Change From Randomization Baseline to the 8 Weeks Post-randomization Timepoint in the 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: Randomization Baseline (Week 8, 10 or 12); 8 weeks post-randomization
Change From Randomization Baseline to the Week 36 Visit in the 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: Randomization Baseline (Week 8, 10 or 12); Week 36
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2018
- Study start17 Apr 2018
- Primary completion16 Jul 2019
- Study completion16 Jul 2019
Why it stopped: Futility
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
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 81 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurocrine Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Development Lead, Study director, Neurocrine Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03530293
- Sponsor's protocol ID
- NBI-98854-TS2005
- Status last verified
- Apr 2022
- First submitted
- 8 May 2018
- Last updated
- 17 May 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Double-Blind, Placebo-Controlled, Randomized Withdrawal Study to Evaluate the Safety and Efficacy of NBI-98854 in Pediatric Subjects With Tourette Syndrome
- ClinicalTrials.gov ID
- NCT03530293
- Acronym / sponsor ID
- NBI-98854-TS2005
- Conditions
- Tourette Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Futility
- Enrolment
- 81 participants (actual)
- Lead sponsor
- Neurocrine BiosciencesIndustry (pharma, biotech, devices)
- Study start
- 17 Apr 2018 (actual)
- Primary completion
- 16 Jul 2019 (actual)
- Study completion
- 16 Jul 2019 (actual)
- First posted
- 21 May 2018 (actual)
- Results
- Posted 17 May 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Pre-randomization Valbenazine | Experimental | Drug: ValbenazineParticipants 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. |
| Randomized Placebo | Placebo comparator | Drug: Placebo oral capsuleParticipants 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. |
| Randomized Valbenazine | Experimental | Drug: ValbenazineParticipants 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. |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
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 (12 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 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 | Pre-randomization Valbenazine, Randomized Valbenazine |
| Placebo oral capsulenon-active dosage form | Drug | Randomized Placebo |
Primary outcome
Time to Loss of Treatment Response
Time frame: Randomization (Week 8, 10 or 12) through Week 36
Loss of treatment response during the withdrawal period was defined as: 2 consecutive visits with 1) an increase in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) of greater than 35% or 7 points from the randomized withdrawal period baseline and 2) an increase in CGI-Tics-Severity score of ≥2 points from the randomized withdrawal period baseline; or discontinuation due to lack of efficacy or a treatment-emergent adverse event (TEAE) of worsening of tics. Median (lower and upper quartiles) Kaplan-Meier estimates for the time to loss of treatment response were not able to be calculated because of the low incidence of loss of treatment response events.
4 secondary outcomes
Change From Randomization Baseline to the 8 Weeks Post-randomization Timepoint in the YGTSS TTS
Time frame: Randomization Baseline (Week 8, 10 or 12); 8 weeks post-randomization
Change From Randomization Baseline to the Week 36 Visit in the YGTSS TTS
Time frame: Randomization Baseline (Week 8, 10 or 12); Week 36
Change From Randomization Baseline to the 8 Weeks Post-randomization Timepoint in the CGI-Tics-Severity Score
Time frame: Randomization Baseline (Week 8, 10 or 12); 8 weeks post-randomization
Change From Randomization Baseline to the Week 36 Visit in the CGI-Tics-Severity Score
Time frame: Randomization Baseline (Week 8, 10 or 12); Week 36
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 May 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Clinical Development Lead study director
Neurocrine Biosciences
49 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 49 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 7 inclusion and 12 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: stopped early, as of the sponsor's last update on 17 May 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 49 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. 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 17 May 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?
Time to Loss of Treatment Response (time frame: Randomization (Week 8, 10 or 12) through Week 36). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 May 2022.