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Safety and Efficacy of NBI-98854 in Pediatric Subjects With Tourette Syndrome

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ClinicalTrials.gov Identifier: NCT03530293
Recruitment Status : Recruiting
First Posted : May 21, 2018
Last Update Posted : November 30, 2018
Sponsor:
Information provided by (Responsible Party):
Neurocrine Biosciences

Brief 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.

Condition or disease Intervention/treatment Phase
Tourette Syndrome Drug: Valbenazine Drug: Placebo oral capsule Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 180 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description: open-label study drug treatment period followed by blinded randomization into treatment arm or placebo arm
Primary Purpose: Treatment
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
Actual Study Start Date : April 16, 2018
Estimated Primary Completion Date : January 2020
Estimated Study Completion Date : February 2020

Resource links provided by the National Library of Medicine

Drug Information available for: Valbenazine

Arm Intervention/treatment
Experimental: Valbenazine
Capsule, administered once daily. Randomization into this arm occurs after open-label treatment with valbenazine once daily for up to 12 weeks. Total treatment up to 36 weeks.
Drug: Valbenazine
vesicular monoamine transporter 2 (VMAT2) inhibitor
Other Name: Ingrezza, NBI-98854

Placebo Comparator: Placebo
Capsule, administered once daily. Randomization into this arm occurs after open-label treatment with valbenazine once daily for up to 12 weeks. Total treatment up to 36 weeks.
Drug: Placebo oral capsule
non-active dosage form




Primary Outcome Measures :
  1. Severity of tic symptoms assessed by Yale Global Tic Severity Scale (YGTSS) [ Time Frame: 40 weeks ]
    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 scale also includes an impairment assessment. The YGTSS total tic score (TTS) will be the primary measure for this study and represents the sum of the 5 subcategories (number, frequency, intensity, complexity, and interference) for both motor and phonic tics, each scored from 0 to 5, with higher values representing a worsening assessment.


Secondary Outcome Measures :
  1. Overall improvement of Tourette Syndrome with Clinical Global Impression-Tourette Syndrome (CGI-TS-Improvement) [ Time Frame: 40 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Have a clinical diagnosis of Tourette Syndrome (TS)
  2. Have at least moderate tic severity
  3. Have TS symptoms that impair school, occupational, and/or social function
  4. 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
  5. Be in good general health
  6. 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
  7. 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:

  1. Have an active, clinically significant unstable medical condition within 1 month prior to screening
  2. Have a known history of long QT syndrome or cardiac arrhythmia
  3. Have a known history of neuroleptic malignant syndrome
  4. Have a cancer diagnosis within 3 years prior to screening (some exceptions allowed)
  5. Have an allergy, hypersensitivity, or intolerance to VMAT2 inhibitors
  6. Have a blood loss ≥250 mL or donated blood within 30 days prior to screening
  7. Have a known history of substance dependence, substance (drug) or alcohol abuse
  8. Have a significant risk of suicidal or violent behavior
  9. Have initiated Comprehensive Behavioral Intervention for Tics (CBIT) during the screening period or at baseline or plan to initiate CBIT during the study
  10. Have received an investigational drug within 30 days before screening or plan to use an investigational drug (other than NBI-98854) during the study
  11. Have previously participated in an NBI-98854 clinical study, except for NBI-98854-1403 or NBI-98854-1501.
  12. Have HIV, hepatitis B, or hepatitis C

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03530293


Contacts
Contact: Cheryl Chen 858-617-7744 cechen@neurocrine.com

  Show 44 Study Locations
Sponsors and Collaborators
Neurocrine Biosciences
Investigators
Study Director: Chief Medical Officer Chief Medical Officer

Additional Information:
Responsible Party: Neurocrine Biosciences
ClinicalTrials.gov Identifier: NCT03530293     History of Changes
Other Study ID Numbers: NBI-98854-TS2005
First Posted: May 21, 2018    Key Record Dates
Last Update Posted: November 30, 2018
Last Verified: November 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Syndrome
Tourette Syndrome
Disease
Pathologic Processes
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Tic Disorders
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Neurodevelopmental Disorders
Mental Disorders
Tetrabenazine
Adrenergic Uptake Inhibitors
Neurotransmitter Uptake Inhibitors
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Adrenergic Agents
Neurotransmitter Agents
Physiological Effects of Drugs