Clinical trial NCT02679079: Safety and Efficacy Study of NBI-98854 in Children and Adolescents With Tourette Syndrome
Official title: A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of NBI-98854 in 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: 2016-02-10
Enrollment: 98
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: Phase 2, double-blind, placebo-controlled study to assess the safety and efficacy of NBI-98854 administered once daily (qd) for a total of 6 weeks of treatment. This study will enroll approximately 90 male and female pediatric subjects clinically diagnosed with Tourette Syndrome.


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 6 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS)
timeFrame: Baseline, Week 6
Secondary outcomes:
measure: Clinical Global Impression of Tourette Syndrome (CGI-TS) - Improvement Score at Week 6
timeFrame: Week 6
measure: Participants Who Are a YGTSS TTS Responder at Week 6
timeFrame: Baseline, Week 6
measure: Change From Baseline to Week 6 in the YGTSS Impairment Score
timeFrame: Baseline, Week 6
measure: Change From Baseline to Week 6 in the YGTSS Global Tic Severity Score
timeFrame: Baseline, Week 6
measure: Change From Baseline to Week 6 in the Rush Video-based Tic Rating Scale (RTRS) Total Score
timeFrame: Baseline, Week 6
measure: Change From Baseline to Week 6 in the Premonitory Urge for Tics Scale (PUTS) Total Score
timeFrame: Baseline, Week 6
measure: Change From Baseline to Week 6 in the Clinical Global Impression of Tics (CGI-Tics) - Severity Score
timeFrame: Baseline, Week 6
measure: Participants Who Are a CGI-TS-Improvement Responder at Week 6
timeFrame: Week 6
Other outcomes:
Locations:
city: Sun City
country: United States
state: Arizona
city: Springdale
country: United States
state: Arkansas
city: Long Beach
country: United States
state: California
city: San Bernardino
country: United States
state: California
city: San Diego
country: United States
state: California
city: Upland
country: United States
state: California
city: Bradenton
country: United States
state: Florida
city: Gainesville
country: United States
state: Florida
city: Hialeah
country: United States
state: Florida
city: Loxahatchee Groves
country: United States
state: Florida
city: Melbourne
country: United States
state: Florida
city: Orlando
country: United States
state: Florida
city: Saint Petersburg
country: United States
state: Florida
city: Tampa
country: United States
state: Florida
city: Atlanta
country: United States
state: Georgia
city: Chicago
country: United States
state: Illinois
city: Naperville
country: United States
state: Illinois
city: Wichita
country: United States
state: Kansas
city: Boston
country: United States
state: Massachusetts
city: Natick
country: United States
state: Massachusetts
city: Ann Arbor
country: United States
state: Michigan
city: Bloomfield Hills
country: United States
state: Michigan
city: Lincoln
country: United States
state: Nebraska
city: Nashua
country: United States
state: New Hampshire
city: Marlton
country: United States
state: New Jersey
city: Summit
country: United States
state: New Jersey
city: Manhasset
country: United States
state: New York
city: New York
country: United States
state: New York
city: Rochester
country: United States
state: New York
city: Cincinnati
country: United States
state: Ohio
city: Middleburg Heights
country: United States
state: Ohio
city: Oklahoma City
country: United States
state: Oklahoma
city: Franklin
country: United States
state: Tennessee
city: Nashville
country: United States
state: Tennessee
city: Dallas
country: United States
state: Texas
city: DeSoto
country: United States
state: Texas
city: Fort Worth
country: United States
state: Texas
city: Houston
country: United States
state: Texas
city: Irving
country: United States
state: Texas
city: Orem
country: United States
state: Utah
city: Salt Lake City
country: United States
state: Utah
city: Kirkland
country: United States
state: Washington
city: Seattle
country: United States
state: Washington
city: Spokane
country: United States
state: Washington
Study officials:
References: