Tourette SyndromePhase 2NCT03325010
Safety, Tolerability, and Efficacy 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 35 sites in 2 countries.
Trial summary
This is a Phase 2b, randomized, double-blind, placebo-controlled, dose-optimization study to evaluate the efficacy, safety, and tolerability of NBI-98854 titrated to the subject's optimal dose administered once daily (qd) for a total of 12 weeks of treatment 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
- 127 participants
- Where
- 35 sitesin 2 countries
- Study dates
- 5 Oct 2017to 16 Nov 2018
- Phase
- Phase 2
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 10Show 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
Trial locations and contacts
35 sites in 2 countries.
35 sites in 2 countries
United States34 sites
Neurocrine Clinical Site
Anaheim, California, 92805
Neurocrine Clinical Site
Boston, Massachusetts, 02114
Neurocrine Clinical Site
Chicago, Illinois, 60634
Neurocrine Clinical Site
Chicago, Illinois, 60637
Neurocrine Clinical Site
Dallas, Texas, 75243
Neurocrine Clinical Site
Durham, North Carolina, 27705
Neurocrine Clinical Site
Everett, Washington, 98201
Neurocrine Clinical Site
Hialeah, Florida, 33012
Neurocrine Clinical Site
Hialeah, Florida, 33013
Neurocrine Clinical Site
Hialeah, Florida, 33018
Neurocrine Clinical Site
Houston, Texas, 77058
Neurocrine Clinical Site
Iowa City, Iowa, 52242
Neurocrine Clinical Site
Irving, Texas, 75062
Neurocrine Clinical Site
Leawood, Kansas, 66206
Neurocrine Clinical Site
Lincoln, Nebraska, 68526
Neurocrine Clinical Site
Loxahatchee Groves, Florida, 33470
Neurocrine Clinical Site
Memphis, Tennessee, 38119
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, 06519
Neurocrine Clinical Site
New York, New York, 10036
Neurocrine Clinical Site
Orange City, Florida, 32763
Neurocrine Clinical Site
Orlando, Florida, 32801
Neurocrine Clinical Site
Orlando, Florida, 32803
Neurocrine Clinical Site
Rogers, Arkansas, 72758
Neurocrine Clinical Site
San Diego, California, 92108
Neurocrine Clinical Site
Santa Clarita, California, 91321
Neurocrine Clinical Site
Seattle, Washington, 98105
Neurocrine Clinical Site
Spokane, Washington, 99204
Neurocrine Clinical Site
Sun City, Arizona, 85351
Neurocrine Clinical Site
Tampa, Florida, 33609
Neurocrine Clinical Site
The Bronx, New York, 10467
Neurocrine Clinical Site
Voorhees Township, New Jersey, 08043
About this study
Official title: A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Dose Optimization Study to Assess the Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome
This is a Phase 2b, randomized, double-blind, placebo-controlled, dose-optimization study to evaluate the efficacy, safety, and tolerability of NBI-98854 titrated to the subject's optimal dose administered once daily (qd) for a total of 12 weeks of treatment 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 Ingrezza, NBI-98854
vesicular monoamine transporter 2 (VMAT2) inhibitor
Placebo oral capsuleDrug
non-active dosage form
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Participants received placebo (matching valbenazine) once daily for 12 weeks. Receives: Drug: Placebo oral capsule |
| Valbenazine | Experimental | Participants received valbenazine once daily for 12 weeks. 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 subjects <50 kg and 80 mg for subjects ≥50 kg to achieve an optimal dose of valbenazine for each participant. Receives: Drug: Valbenazine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline to Week 12 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (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 12
Secondary outcomes (3)
Change From Baseline to Week 12 in the Clinical Global Impression of Tics Severity (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 12
Participants Who Are a Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) Responder at Week 12
A TTS responder is defined, on a per-visit basis, as a participant whose TTS value is reduced by at least 30% from baseline at the specified postbaseline visit.
Measured: Baseline, Week 12
Participants Who Are a Clinical Global Impression of Tourette Syndrome Improvement (CGI-TS-Improvement) Responder at Week 12
The CGI-TS-Improvement scale is used to assess overall improvement since the initiation of study drug dosing on a 7-point scale. A CGI-TS-Improvement responder is defined, on a per-visit basis, as a participant whose CGI-TS-Improvement score is 1 ("Very much improved") or 2 ("Much improved") at the specified postbaseline visit.
Measured: Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Oct 2017
- Study start5 Oct 2017
- Primary completion1 Nov 2018
- Study completion16 Nov 2018
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
- 127 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurocrine Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03325010
- Sponsor's protocol ID
- NBI-98854-TS2003
- Status last verified
- Jun 2021
- First submitted
- 25 Oct 2017
- Last updated
- 28 Jun 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No