Brief Title
Safety and Efficacy of NBI-98854 in Pediatric Subjects With Tourette Syndrome
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
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.
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
Time to Loss of Treatment Response
Secondary Outcome
Change From Randomization Baseline to the 8 Weeks Post-randomization Timepoint in the YGTSS TTS
Condition
Tourette Syndrome
Intervention
Valbenazine
Study Arms / Comparison Groups
Pre-randomization Valbenazine
Description: Participants received valbenazine once daily for up to 12 weeks, depending on if and when randomization occured. The starting dose was 20 mg for participants <50 kg at baseline and 40 mg for participants ≥50 kg at baseline, and could be escalated in increments of 20 mg every 2 weeks to a maximum of 60 mg for participants <50 kg and 80 mg for participants ≥50 kg to achieve an optimal dose of valbenazine for each participant.
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
81
Start Date
April 17, 2018
Completion Date
July 16, 2019
Primary Completion Date
July 16, 2019
Eligibility 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
Gender
All
Ages
6 Years - 17 Years
Accepts Healthy Volunteers
No
Contacts
Clinical Development Lead, ,
Location Countries
Puerto Rico
Location Countries
Puerto Rico
Administrative Informations
NCT ID
NCT03530293
Organization ID
NBI-98854-TS2005
Responsible Party
Sponsor
Study Sponsor
Neurocrine Biosciences
Study Sponsor
Clinical Development Lead, Study Director, Neurocrine Biosciences
Verification Date
April 2022