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Safety, Pharmacokinetics, and Pharmacodynamics of NBI-98854 in Children and Adolescents With Tourette Syndrome (T-FORCE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02256475
Recruitment Status : Completed
First Posted : October 3, 2014
Last Update Posted : January 7, 2016
Sponsor:
Information provided by (Responsible Party):
Neurocrine Biosciences

Brief Summary:
This is a Phase 1b, open-label, multiple-dose study of the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-98854 in a total of 36 pediatric subjects with Tourette syndrome (TS). The study will be conducted in approximately 18 male and female children (6 to 11 years of age) and approximately 18 male and female adolescents (12 to 18 years of age). Both age groups will be divided equally into 3 dosing cohorts with 6 subjects each. Ascending doses will be evaluated as part of a staggered-cohort design. Study drug will be administered in each cohort for 14 consecutive days.

Condition or disease Intervention/treatment Phase
Tourette Syndrome Drug: NBI-98854 Phase 1

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 36 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Basic Science
Official Title: A Phase 1b, Open-Label, Multiple-Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-98854 in Children and Adolescents With Tourette Syndrome
Study Start Date : September 2014
Actual Primary Completion Date : November 2015
Actual Study Completion Date : December 2015

Resource links provided by the National Library of Medicine

Drug Information available for: Valbenazine

Arm Intervention/treatment
Experimental: Adolescents Dose Group 1
Fixed dose of NBI-98854 administered once daily at 0800 for 14 days.
Drug: NBI-98854
Experimental: Adolescents Dose Group 2
Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Dosing will not commence until all safety and PK results from the adolescent dose group 1 have been reviewed to ensure there are no safety concerns and that maximum tolerated dose (MTD) has not been reached.
Drug: NBI-98854
Experimental: Adolescents Dose Group 3
Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Dosing will not commence until all safety and PK results from the adolescent dose group 2 have been reviewed to ensure there are no safety concerns and that MTD has not been reached.
Drug: NBI-98854
Experimental: Children Dose Group 1
Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Dosing will not commence until all safety and PK results from the adolescent dose group 1 have been reviewed to ensure there are no safety concerns and that MTD has not been reached.
Drug: NBI-98854
Experimental: Children Dose Group 2
Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Dosing will not commence until all safety and PK results from the children dose group 1 have been reviewed to ensure there are no safety concerns and that MTD has not been reached.
Drug: NBI-98854
Experimental: Children Dose Group 3
Fixed dose of NBI-98854 administered once daily at 0800 for 14 days. Dosing will not commence until all safety and PK results from the children dose group 2 have been reviewed to ensure there are no safety concerns and that MTD has not been reached.
Drug: NBI-98854



Primary Outcome Measures :
  1. Number of participants with adverse events following dosing with NBI-98854 [ Time Frame: Up to 21 days ]
  2. Area Under Curve (AUC) of NBI-98854 and its metabolites following repeated daily doses of NBI-98854 [ Time Frame: Day 1: predose, 0.5, 1, 2, 4, 6, 8, 12, 24 hours post-dose; Day 14: predose, 0.5, 1, 2, 4, 6, 8, 12, 24, 72, 120, 168 hours postdose ]
  3. Assessment of tic behaviors associated with TS using the Yale Global Tic Severity Scale (YGTSS) [ Time Frame: Days 1, 7, 14, and 21 ]

Secondary Outcome Measures :
  1. Assessment of tic behaviors associated with TS using the Rush Video-based Tic Rating Scale (RTRS) [ Time Frame: Days 1, 7, 14, and 21 ]
  2. Quantification of the premonitory urge phenomena associated with tics using the Premonitory Urge for Tics Scale (PUTS) [ Time Frame: Days 1, 7, 14, and 21 ]
  3. Clinical Global Impression of Tourette Syndrome (CGI-TS) [ Time Frame: Days 1, 7, 14, and 21 ]
  4. CogState computerized test to assess cognitive function [ Time Frame: Day 1 at predose, 2.5 and 8 hours postdose; Day 14 at 8 hours postdose ]


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Ages Eligible for Study:   6 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Have documentation of written informed consent, or written and witnessed assent from the subject and written informed consent from the subject's parent or legal guardian.
  2. Be in good general health.
  3. Have a Diagnostic and Statistical Manual of Mental Disorders diagnosis of Tourette Syndrome (DSM-IV or -V).
  4. Have TS symptoms that impair school, occupational, and/or social function.
  5. If medications are being used to treat TS symptoms, must be on stable doses of these medications for a minimum of 30 days before baseline (Day -1), and the medication regimen is expected to remain stable throughout the study period. The use of tetrabenazine to treat TS symptoms is prohibited.
  6. Subjects of childbearing potential who do not practice total abstinence must be instructed on the proper use of barrier methods of contraception and agree to use hormonal or two forms of nonhormonal contraception consistently from screening until 30 days after the last dose of study drug.
  7. Adolescent subjects (12 to 18 years of age) must have a negative urine drug screen for amphetamines, barbiturates, benzodiazepine, phencyclidine, cocaine, opiates, or cannabinoids. Subjects who are on stable doses of prescribed benzodiazepines, opiates, or psychostimulants are allowed to participate in the study.
  8. Adolescent subjects (12 to 18 years of age) must have a negative alcohol breath test.
  9. Be willing and able to adhere to the study regimen and study procedures described in the protocol and informed consent/assent forms, including all requirements at the study center and return for the follow-up visit.

Exclusion Criteria:

  1. Have an unstable medical condition or chronic disease.
  2. Had a medically significant illness within 30 days of screening.
  3. Excessive use of tobacco and/or nicotine-containing products.
  4. Have a history of substance (drug) dependence or substance or alcohol abuse.
  5. Are currently pregnant or lactating.
  6. Have a known history of neuroleptic malignant syndrome.
  7. Have a known history of long QT syndrome or cardiac tachy-arrhythmia.
  8. Have a cancer diagnosis within 3 years prior to screening, with the exception of localized skin cancer or carcinoma in situ of the cervix.
  9. Have received an investigational drug within 30 days before screening or plan to use an investigational drug (other than NBI-98854) during the study.
  10. Have a significant risk of suicidal or violent behavior.

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): NCT02256475


Locations
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United States, California
San Diego, California, United States
United States, Florida
Kissimmee, Florida, United States
St. Petersburg, Florida, United States
United States, Illinois
Naperville, Illinois, United States
United States, Missouri
St. Louis, Missouri, United States
United States, Nebraska
Lincoln, Nebraska, United States
United States, New York
New York, New York, United States
Rochester, New York, United States
United States, Virginia
Petersburg, Virginia, United States
United States, Washington
Kirkland, Washington, United States
Sponsors and Collaborators
Neurocrine Biosciences
Investigators
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Principal Investigator: Chris O'Brien, MD Neurocrine Biosciences

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Responsible Party: Neurocrine Biosciences
ClinicalTrials.gov Identifier: NCT02256475     History of Changes
Other Study ID Numbers: NBI-98854-1403
First Posted: October 3, 2014    Key Record Dates
Last Update Posted: January 7, 2016
Last Verified: January 2016

Additional relevant MeSH terms:
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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