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Completed

NCT Number: NCT02679079

Safety and Efficacy Study of NBI-98854 in Children and Adolescents 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.

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Key information

Conditions

Tourette Syndrome Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Mental Disorders Movement Disorders Nervous System Diseases Neurodegenerative Diseases Neurodevelopmental Disorders Tic Disorders

Age range

6 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Sun City, Arizona, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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
  • 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

Treatment and study plan

NBI-98854

Drug

Placebo

Drug

Primary outcomes

  1. Change From Baseline to Week 6 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS)

    Time frame: Baseline, Week 6

    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

Secondary outcomes

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

    Time frame: 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.

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

    Time frame: Baseline, 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.

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

    Time frame: Baseline, Week 6

    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.

  4. Change From Baseline to Week 6 in the YGTSS Global Tic Severity Score

    Time frame: Baseline, Week 6

    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.

  5. Change From Baseline to Week 6 in the Rush Video-based Tic Rating Scale (RTRS) Total Score

    Time frame: Baseline, Week 6

    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.

  6. Change From Baseline to Week 6 in the Premonitory Urge for Tics Scale (PUTS) Total Score

    Time frame: Baseline, Week 6

    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.

  7. Change From Baseline to Week 6 in the Clinical Global Impression of Tics (CGI-Tics) - Severity Score

    Time frame: Baseline, Week 6

    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.

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

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Neurocrine Biosciences

Industry

Registry information

Official study title

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

Important dates

Study start
2016
Primary completion
2017
Study completion
2017
First posted
Feb 10, 2016
Registry last updated
Jan 15, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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