Clinical trial NCT03530293: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Tourette Syndrome
Lead sponsor: Neurocrine Biosciences
Sponsor class: INDUSTRY
First posted: 2018-05-21
Enrollment: 81
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 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.


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

Primary outcomes:
measure: Time to Loss of Treatment Response
timeFrame: Randomization (Week 8, 10 or 12) through Week 36
Secondary outcomes:
measure: Change From Randomization Baseline to the 8 Weeks Post-randomization Timepoint in the YGTSS TTS
timeFrame: Randomization Baseline (Week 8, 10 or 12); 8 weeks post-randomization
measure: Change From Randomization Baseline to the Week 36 Visit in the YGTSS TTS
timeFrame: Randomization Baseline (Week 8, 10 or 12); Week 36
measure: Change From Randomization Baseline to the 8 Weeks Post-randomization Timepoint in the CGI-Tics-Severity Score
timeFrame: Randomization Baseline (Week 8, 10 or 12); 8 weeks post-randomization
measure: Change From Randomization Baseline to the Week 36 Visit in the CGI-Tics-Severity Score
timeFrame: Randomization Baseline (Week 8, 10 or 12); Week 36
Other outcomes:
Locations:
city: Sun City
country: United States
facility: Neuricrine Clinical Site
state: Arizona
city: Little Rock
country: United States
facility: Neurocrine Clinical Site
state: Arkansas
city: Rogers
country: United States
facility: Neurocrine Clinical Site
state: Arkansas
city: Anaheim
country: United States
facility: Neurocrine Clinical Site
state: California
city: Fullerton
country: United States
facility: Neurocrine Clinical Site
state: California
city: San Diego
country: United States
facility: Neurocrine Clinical Site
state: California
city: Santa Ana
country: United States
facility: Neurocrine Clinical Site
state: California
city: Pueblo
country: United States
facility: Neurocrine Clinical Site
state: Colorado
city: Stamford
country: United States
facility: Neurocrine Clinical Site
state: Connecticut
city: Washington
country: United States
facility: Neurocrine Clinical Site
state: District of Columbia
city: Gulf Breeze
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Hialeah
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Miami
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Miami
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: North Miami
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Orlando
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Palmetto Bay
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Saint Petersburg
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Spring Hill
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Tallahassee
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Atlanta
country: United States
facility: Neurocrine Clinical Site
state: Georgia
city: Fayetteville
country: United States
facility: Neurocrine Clinical Site
state: Georgia
city: Savannah
country: United States
facility: Neurocrine Clinical Site
state: Georgia
city: Chicago
country: United States
facility: Neurocrine Clinical Site
state: Illinois
city: South Bend
country: United States
facility: Neurocrine Clinical Site
state: Indiana
city: Boston
country: United States
facility: Neurocrine Clinical Site
state: Massachusetts
city: Ann Arbor
country: United States
facility: Neurocrine Clinical Site
state: Michigan
city: Bloomfield Hills
country: United States
facility: Neurocrine Clinical Site
state: Michigan
city: West Bloomfield
country: United States
facility: Neurocrine Clinical Site
state: Michigan
city: Lincoln
country: United States
facility: Neurocrine Clinical Site
state: Nebraska
city: Nashua
country: United States
facility: Neurocrine Clinical Site
state: New Hampshire
city: Cherry Hill
country: United States
facility: Neurocrine Clinical Site
state: New Jersey
city: Mount Arlington
country: United States
facility: Neurocrine Clinical Site
state: New Jersey
city: New York
country: United States
facility: Neurocrine Clinical Site
state: New York
city: S. Setauket
country: United States
facility: Neurocrine Clinical Site
state: New York
city: Charlotte
country: United States
facility: Neurocrine Clinical Site
state: North Carolina
city: Mason
country: United States
facility: Neurocrine Clinical Site
state: Ohio
city: Oklahoma City
country: United States
facility: Neurocrine Clinical Site
state: Oklahoma
city: Dallas
country: United States
facility: Neurocrine Clinical Site
state: Texas
city: DeSoto
country: United States
facility: Neurocrine Clinical Site
state: Texas
city: Houston
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: San Antonio
country: United States
facility: Neurocrine Clinical Site
state: Texas
city: Charlottesville
country: United States
facility: Neurocrine Clinical Site
state: Virginia
city: Bothell
country: United States
facility: Neurocrine Clinical Site
state: Washington
city: Everett
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:
affiliation: Neurocrine Biosciences
name: Clinical Development Lead
role: STUDY_DIRECTOR
References: