Safety and Efficacy of NBI-98854 in Pediatric Subjects With Tourette Syndrome

Tourette Syndrome Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03530293
• Other study ID #: NBI-98854-TS2005
• Status: Terminated
• Phase: Phase 2
• Start date: April 17, 2018
• Est. completion date: July 16, 2019

Study information

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

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 81
• Est. completion date: July 16, 2019
• Est. primary completion date: July 16, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Syndrome
• Tourette Syndrome

Intervention

Drug:
• Valbenazine
vesicular monoamine transporter 2 (VMAT2) inhibitor
• Placebo oral capsule
non-active dosage form

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	Atlanta	Georgia
United States	Neurocrine Clinical Site	Bloomfield Hills	Michigan
United States	Neurocrine Clinical Site	Boston	Massachusetts
United States	Neurocrine Clinical Site	Bothell	Washington
United States	Neurocrine Clinical Site	Charlotte	North Carolina
United States	Neurocrine Clinical Site	Charlottesville	Virginia
United States	Neurocrine Clinical Site	Cherry Hill	New Jersey
United States	Neurocrine Clinical Site	Chicago	Illinois
United States	Neurocrine Clinical Site	Dallas	Texas
United States	Neurocrine Clinical Site	DeSoto	Texas
United States	Neurocrine Clinical Site	Everett	Washington
United States	Neurocrine Clinical Site	Fayetteville	Georgia
United States	Neurocrine Clinical Site	Fullerton	California
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	Irving	Texas
United States	Neurocrine Clinical Site	Lincoln	Nebraska
United States	Neurocrine Clinical Site	Little Rock	Arkansas
United States	Neurocrine Clinical Site	Mason	Ohio
United States	Neurocrine Clinical Site	Miami	Florida
United States	Neurocrine Clinical Site	Miami	Florida
United States	Neurocrine Clinical Site	Mount Arlington	New Jersey
United States	Neurocrine Clinical Site	Nashua	New Hampshire
United States	Neurocrine Clinical Site	New York	New York
United States	Neurocrine Clinical Site	North Miami	Florida
United States	Neurocrine Clinical Site	Oklahoma City	Oklahoma
United States	Neurocrine Clinical Site	Orlando	Florida
United States	Neurocrine Clinical Site	Palmetto Bay	Florida
United States	Neurocrine Clinical Site	Pueblo	Colorado
United States	Neurocrine Clinical Site	Rogers	Arkansas
United States	Neurocrine Clinical Site	S. Setauket	New York
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	Savannah	Georgia
United States	Neurocrine Clinical Site	South Bend	Indiana
United States	Neurocrine Clinical Site	Spokane	Washington
United States	Neurocrine Clinical Site	Spring Hill	Florida
United States	Neurocrine Clinical Site	Stamford	Connecticut
United States	Neuricrine Clinical Site	Sun City	Arizona
United States	Neurocrine Clinical Site	Tallahassee	Florida
United States	Neurocrine Clinical Site	Washington	District of Columbia
United States	Neurocrine Clinical Site	West Bloomfield	Michigan

Sponsors (1)

Lead Sponsor	Collaborator
Neurocrine Biosciences

Countries where clinical trial is conducted

United States,  Puerto Rico, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Time to Loss of Treatment Response	Loss of treatment response during the withdrawal period was defined as: 2 consecutive visits with 1) an increase in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) of greater than 35% or 7 points from the randomized withdrawal period baseline and 2) an increase in CGI-Tics-Severity score of =2 points from the randomized withdrawal period baseline; or discontinuation due to lack of efficacy or a treatment-emergent adverse event (TEAE) of worsening of tics. Median (lower and upper quartiles) Kaplan-Meier estimates for the time to loss of treatment response were not able to be calculated because of the low incidence of loss of treatment response events.	Randomization (Week 8, 10 or 12) through Week 36
Secondary	Change From Randomization Baseline to the 8 Weeks Post-randomization Timepoint in the YGTSS TTS	The YGTSS is designed to rate the overall severity of motor and phonic tic symptoms across a range of dimensions: number, frequency, intensity, complexity, and interference. The YGTSS was administered by the investigator (or qualified designee) using a computer-based structured clinical interview. The TTS is the sum of the 5 motor tic items and the 5 phonic (vocal) tic items and ranges from 0 to 50, with higher scores representing greater severity. Least-squares mean were estimated using a mixed-effects model for repeated measures.	Randomization Baseline (Week 8, 10 or 12); 8 weeks post-randomization
Secondary	Change From Randomization Baseline to the Week 36 Visit in the YGTSS TTS	The YGTSS is designed to rate the overall severity of motor and phonic tic symptoms across a range of dimensions: number, frequency, intensity, complexity, and interference. The YGTSS was administered by the investigator (or qualified designee) using a computer-based structured clinical interview. The TTS is the sum of the 5 motor tic items and the 5 phonic (vocal) tic items and ranges from 0 to 50, with higher scores representing greater severity.	Randomization Baseline (Week 8, 10 or 12); Week 36
Secondary	Change From Randomization Baseline to the 8 Weeks Post-randomization Timepoint in the CGI-Tics-Severity Score	The CGI-Tics-Severity scale is used to assess overall severity on a 7-point scale. Each of the CGI-Tics-Severity response categories was assigned a numerical score as follows: 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; 7 = Among the most extremely ill patient.	Randomization Baseline (Week 8, 10 or 12); 8 weeks post-randomization
Secondary	Change From Randomization Baseline to the Week 36 Visit in the CGI-Tics-Severity Score	The CGI-Tics-Severity scale is used to assess overall severity on a 7-point scale. Each of the CGI-Tics-Severity response categories was assigned a numerical score as follows: 1 = Normal, not at all ill; 2 = Borderline ill; 3 = Mildly ill; 4 = Moderately ill; 5 = Markedly ill; 6 = Severely ill; 7 = Among the most extremely ill patient.	Randomization Baseline (Week 8, 10 or 12); Week 36