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Tourette SyndromePhase 2NCT02679079

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

A Phase 2 study testing NBI-98854 in people with Tourette Syndrome, run by Neurocrine Biosciences at 44 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 6 weeks of treatment. This study will enroll approximately 90 male and female pediatric subjects clinically diagnosed with Tourette Syndrome. 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?
NBI-98854
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
98 participants
Where
44 sitesUnited States
Study dates
23 Mar 2016to 14 Apr 2017
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 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

44 sites in 1 country.

44 sites in 1 country

United States44 sites

  • Study site

    Ann Arbor, Michigan

  • Study site

    Atlanta, Georgia

  • Study site

    Bloomfield Hills, Michigan

  • Study site

    Boston, Massachusetts

  • Study site

    Bradenton, Florida

  • Study site

    Chicago, Illinois

  • Study site

    Cincinnati, Ohio

  • Study site

    Dallas, Texas

  • Study site

    DeSoto, Texas

  • Study site

    Fort Worth, Texas

  • Study site

    Franklin, Tennessee

  • Study site

    Gainesville, Florida

  • Study site

    Hialeah, Florida

  • Study site

    Houston, Texas

  • Study site

    Irving, Texas

  • Study site

    Kirkland, Washington

  • Study site

    Lincoln, Nebraska

  • Study site

    Long Beach, California

  • Study site

    Loxahatchee Groves, Florida

  • Study site

    Manhasset, New York

  • Study site

    Marlton, New Jersey

  • Study site

    Melbourne, Florida

  • Study site

    Middleburg Heights, Ohio

  • Study site

    Naperville, Illinois

  • Study site

    Nashua, New Hampshire

  • Study site

    Nashville, Tennessee

  • Study site

    Natick, Massachusetts

  • Study site

    New York, New York

  • Study site

    Oklahoma City, Oklahoma

  • Study site

    Orem, Utah

  • Study site

    Orlando, Florida

  • Study site

    Rochester, New York

  • Study site

    Salt Lake City, Utah

  • Study site

    San Bernardino, California

  • Study site

    San Diego, California

  • Study site

    Seattle, Washington

  • Study site

    Spokane, Washington

  • Study site

    Springdale, Arkansas

  • Study site

    St. Petersburg, Florida

  • Study site

    Summit, New Jersey

  • Study site

    Sun City, Arizona

  • Study site

    Tampa, Florida

  • Study site

    Upland, California

  • Study site

    Wichita, Kansas

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 Pediatric Subjects With Tourette Syndrome

Phase 2, double-blind, placebo-controlled study to assess the safety and efficacy of NBI-98854 administered once daily (qd) for a total of 6 weeks of treatment. This study will enroll approximately 90 male and female pediatric subjects clinically diagnosed with 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 NCT02679079
Study groupRoleWhat they receive
PlaceboPlacebo comparatorAdministered once daily for 6 weeks
Receives: Drug: Placebo
Dose Group 1ExperimentalAdministered once daily for 6 weeks
Receives: Drug: NBI-98854
Dose Group 2ExperimentalAdministered once daily for 6 weeks
Receives: Drug: NBI-98854

What is being measured

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

  1. Change From Baseline to Week 6 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 6

Secondary outcomes (8)

  1. Clinical Global Impression of Tourette Syndrome (CGI-TS) - Improvement Score at Week 6

    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 6

  2. Participants Who Are a YGTSS TTS Responder at Week 6

    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 6

  3. Change From Baseline to Week 6 in the YGTSS Impairment Score

    The YGTSS Impairment item is used to rate impairment due to tics using the following 50-point anchored scale: 0 = None; 10 = Minimal; 20 = Mild; 30 = Moderate; 40 = Marked; 50 = Severe.

    Measured: Baseline, Week 6

  4. Change From Baseline to Week 6 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 6

  5. Change From Baseline to Week 6 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 participants who discontinued prior to Week 6.

    Measured: Baseline, Week 6

  6. Change From Baseline to Week 6 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 total score ranges from 9 to 36, with higher scores indicating a worse outcome.

    Measured: Baseline, Week 6

  7. Change From Baseline to Week 6 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 6

  8. Participants Who Are a CGI-TS-Improvement Responder at Week 6

    A participant is classified as a CGI-TS-Improvement responder at a given visit if their CGI-TS-Improvement score is either a "1" ("very much improved") or a "2" ("much improved") at the visit.

    Measured: Week 6

Timeline

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

  1. First posted10 Feb 2016 (estimated)
  2. Study start23 Mar 2016
  3. Primary completion14 Apr 2017
  4. Study completion14 Apr 2017

Resources

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
98 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02679079
Sponsor's protocol ID
NBI-98854-1501
Status last verified
Dec 2020
First submitted
5 Feb 2016
Last updated
15 Jan 2021
Data monitoring committee
Yes

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 44 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 10 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 15 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 44 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 15 Jan 2021). 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 6 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) (time frame: Baseline, Week 6). 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 NCT02679079.

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 15 Jan 2021.

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