Clinical trial NCT03325010: Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome
Official title: A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Dose Optimization Study to Assess the Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Tourette Syndrome
Lead sponsor: Neurocrine Biosciences
Sponsor class: INDUSTRY
First posted: 2017-10-30
Enrollment: 127
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a Phase 2b, randomized, double-blind, placebo-controlled, dose-optimization study to evaluate the efficacy, safety, and tolerability of NBI-98854 titrated to the subject's optimal dose administered once daily (qd) for a total of 12 weeks of treatment in pediatric subjects with TS.


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

Primary outcomes:
measure: Change From Baseline to Week 12 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS)
timeFrame: Baseline, Week 12
Secondary outcomes:
measure: Change From Baseline to Week 12 in the Clinical Global Impression of Tics Severity (CGI-Tics-Severity) Score
timeFrame: Baseline, Week 12
measure: Participants Who Are a Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) Responder at Week 12
timeFrame: Baseline, Week 12
measure: Participants Who Are a Clinical Global Impression of Tourette Syndrome Improvement (CGI-TS-Improvement) Responder at Week 12
timeFrame: Week 12
Other outcomes:
Locations:
city: Sun City
country: United States
facility: Neurocrine Clinical Site
state: Arizona
city: Rogers
country: United States
facility: Neurocrine Clinical Site
state: Arkansas
city: Anaheim
country: United States
facility: Neurocrine Clinical Site
state: California
city: San Diego
country: United States
facility: Neurocrine Clinical Site
state: California
city: Santa Clarita
country: United States
facility: Neurocrine Clinical Site
state: California
city: New Haven
country: United States
facility: Neurocrine Clinical Site
state: Connecticut
city: Hialeah
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Hialeah
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Hialeah
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Loxahatchee Groves
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Orange City
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Orlando
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Orlando
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Tampa
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Chicago
country: United States
facility: Neurocrine Clinical Site
state: Illinois
city: Chicago
country: United States
facility: Neurocrine Clinical Site
state: Illinois
city: Naperville
country: United States
facility: Neurocrine Clinical Site
state: Illinois
city: Iowa City
country: United States
facility: Neurocrine Clinical Site
state: Iowa
city: Leawood
country: United States
facility: Neurocrine Clinical Site
state: Kansas
city: Boston
country: United States
facility: Neurocrine Clinical Site
state: Massachusetts
city: Lincoln
country: United States
facility: Neurocrine Clinical Site
state: Nebraska
city: Nashua
country: United States
facility: Neurocrine Clinical Site
state: New Hampshire
city: Mount Arlington
country: United States
facility: Neurocrine Clinical Site
state: New Jersey
city: Voorhees
country: United States
facility: Neurocrine Clinical Site
state: New Jersey
city: Bronx
country: United States
facility: Neurocrine Clinical Site
state: New York
city: New York
country: United States
facility: Neurocrine Clinical Site
state: New York
city: Durham
country: United States
facility: Neurocrine Clinical Site
state: North Carolina
city: Memphis
country: United States
facility: Neurocrine Clinical Site
state: Tennessee
city: Dallas
country: United States
facility: Neurocrine Clinical Site
state: Texas
city: Houston
country: United States
facility: Neurocrine Clinical Site
state: Texas
city: Irving
country: United States
facility: Neurocrine Clinical Site
state: Texas
city: Everett
country: United States
facility: Neurocrine Clinical Site
state: Washington
city: Seattle
country: United States
facility: Neurocrine Clinical Site
state: Washington
city: Spokane
country: United States
facility: Neurocrine Clinical Site
state: Washington
city: San Juan
country: Puerto Rico
facility: Neurocrine Clinical Site
Study officials:
References: