Clinical trial NCT03444038: Open-Label Safety and Tolerability Study of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome
Official title: Open-Label Safety and Tolerability Study of Optimized Doses 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: 2018-02-23
Enrollment: 85
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a Phase 2b, multicenter, open-label study to evaluate the safety and tolerability of optimized doses of NBI-98854 administered once daily for 24 weeks in pediatric subjects with Tourette Syndrome.


Eligibility criteria: Inclusion Criteria:

1. Have participated in and completed Study NBI-98854-TS2003
2. Have a clinical diagnosis of Tourette Syndrome (TS)
3. 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
4. Be in good general health
5. Adolescent subjects (12 to 18 years of age) must have a negative urine drug screen for amphetamines, barbiturates, benzodiazepine, phencyclidine, cocaine, opiates, or cannabinoids and a negative alcohol screen
6. 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 vesicular monoamine transporter 2 (VMAT2) inhibitors
6. Have a blood loss ≥250 mL or donated blood within 56 days prior to baseline
7. Have a known history of substance (drug) dependence, or substance or alcohol abuse
8. Have a significant risk of suicidal or violent behavior
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

Primary outcomes:
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
timeFrame: Baseline through Week 24
Secondary outcomes:
Other outcomes:
Locations:
city: Sun City
country: United States
facility: Neurocrine Clinical Site
state: Arizona
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 Ana
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: Boca Raton
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Gulf Breeze
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Hialeah
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: Saint Petersburg
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: 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: Ann Arbor
country: United States
facility: Neurocrine Clinical Site
state: Michigan
city: Bloomfield Hills
country: United States
facility: Neurocrine Clinical Site
state: Michigan
city: Saint Louis
country: United States
facility: Neurocrine Clinical Site
state: Missouri
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: Mason
country: United States
facility: Neurocrine Clinical Site
state: Ohio
city: Oklahoma City
country: United States
facility: Neurocrine Clinical Site
state: Oklahoma
city: Charleston
country: United States
facility: Neurocrine Clinical Site
state: South Carolina
city: Dallas
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: San Antonio
country: United States
facility: Neurocrine Clinical Site
state: Texas
city: Everett
country: United States
facility: Neurocrine Clinical Site
state: Washington
city: Tacoma
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:
citation: Farber RH, Angelov A, Kim K, Carmack T, Thai-Cuarto D, Roberts E. Clinical development of valbenazine for tics associated with Tourette syndrome. Expert Rev Neurother. 2021 Apr;21(4):393-404. doi: 10.1080/14737175.2021.1898948. Epub 2021 Apr 1.