Open-Label Safety and Tolerability Study of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome

Tourette Syndrome Clinical Trial

Official title:

Open-Label Safety and Tolerability Study of Optimized Doses of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03444038
• Other study ID #: NBI-98854-TS2004
• Status: Completed
• Phase: Phase 2
• Start date: February 8, 2018
• Est. completion date: July 12, 2019

Study information

• Verified date: January 2022
• Source: Neurocrine Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 85
• Est. completion date: July 12, 2019
• Est. primary completion date: July 12, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Syndrome
• Tourette Syndrome

Intervention

Drug:
• Valbenazine
vesicular monoamine transporter 2 (VMAT2) inhibitor

Locations

Country	Name	City	State
Puerto Rico	Neurocrine Clinical Site	San Juan
United States	Neurocrine Clinical Site	Anaheim	California
United States	Neurocrine Clinical Site	Ann Arbor	Michigan
United States	Neurocrine Clinical Site	Bloomfield Hills	Michigan
United States	Neurocrine Clinical Site	Boca Raton	Florida
United States	Neurocrine Clinical Site	Bronx	New York
United States	Neurocrine Clinical Site	Charleston	South Carolina
United States	Neurocrine Clinical Site	Chicago	Illinois
United States	Neurocrine Clinical Site	Dallas	Texas
United States	Neurocrine Clinical Site	Durham	North Carolina
United States	Neurocrine Clinical Site	Everett	Washington
United States	Neurocrine Clinical Site	Gulf Breeze	Florida
United States	Neurocrine Clinical Site	Hialeah	Florida
United States	Neurocrine Clinical Site	Houston	Texas
United States	Neurocrine Clinical Site	Houston	Texas
United States	Neurocrine Clinical Site	Iowa City	Iowa
United States	Neurocrine Clinical Site	Leawood	Kansas
United States	Neurocrine Clinical Site	Lincoln	Nebraska
United States	Neurocrine Clinical Site	Mason	Ohio
United States	Neurocrine Clinical Site	Mount Arlington	New Jersey
United States	Neurocrine Clinical Site	Naperville	Illinois
United States	Neurocrine Clinical Site	Nashua	New Hampshire
United States	Neurocrine Clinical Site	New Haven	Connecticut
United States	Neurocrine Clinical Site	New York	New York
United States	Neurocrine Clinical Site	Oklahoma City	Oklahoma
United States	Neurocrine Clinical Site	Orlando	Florida
United States	Neurocrine Clinical Site	Orlando	Florida
United States	Neurocrine Clinical Site	Saint Louis	Missouri
United States	Neurocrine Clinical Site	Saint Petersburg	Florida
United States	Neurocrine Clinical Site	San Antonio	Texas
United States	Neurocrine Clinical Site	San Diego	California
United States	Neurocrine Clinical Site	Santa Ana	California
United States	Neurocrine Clinical Site	Santa Clarita	California
United States	Neurocrine Clinical Site	Sun City	Arizona
United States	Neurocrine Clinical Site	Tacoma	Washington
United States	Neurocrine Clinical Site	Tampa	Florida
United States	Neurocrine Clinical Site	Voorhees	New Jersey

Sponsors (1)

Lead Sponsor	Collaborator
Neurocrine Biosciences

Countries where clinical trial is conducted

United States,  Puerto Rico, 

References & Publications (1)

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. Review. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Treatment-emergent Adverse Events (TEAEs)	A TEAE is an adverse event not present prior to the initiation of study drug dosing, or is an already present event that worsens either in intensity or frequency following the initiation of study drug dosing. All qualifying TEAE are reported regardless of threshold.	Baseline through Week 24