Brief Title
Safety, Pharmacokinetics, and Pharmacodynamics of NBI-98854 in Children and Adolescents With Tourette Syndrome
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
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.
Study Phase
Phase 1
Study Type
Interventional
Primary Outcome
Number of participants with adverse events following dosing with NBI-98854
Secondary Outcome
Assessment of tic behaviors associated with TS using the Rush Video-based Tic Rating Scale (RTRS)
Condition
Tourette Syndrome
Intervention
NBI-98854
Study Arms / Comparison Groups
Adolescents Dose Group 1
Description: Fixed dose of NBI-98854 administered once daily at 0800 for 14 days.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
36
Start Date
September 2014
Completion Date
December 2015
Primary Completion Date
November 2015
Eligibility 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.
Gender
All
Ages
6 Years - 18 Years
Accepts Healthy Volunteers
No
Contacts
Chris O'Brien, MD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT02256475
Organization ID
NBI-98854-1403
Responsible Party
Sponsor
Study Sponsor
Neurocrine Biosciences
Study Sponsor
Chris O'Brien, MD, Principal Investigator, Neurocrine Biosciences
Verification Date
January 2016