Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome

Tourette Syndrome Clinical Trial

Official title:

A Phase 2b, Randomized, Double-Blind, Placebo-Controlled, Dose Optimization Study to Assess the Safety, Tolerability, and Efficacy of NBI-98854 for the Treatment of Pediatric Subjects With Tourette Syndrome

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03325010
• Other study ID #: NBI-98854-TS2003
• Status: Completed
• Phase: Phase 2
• Start date: October 5, 2017
• Est. completion date: November 16, 2018

Study information

• Verified date: June 2021
• Source: Neurocrine Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a Phase 2b, randomized, double-blind, placebo-controlled, dose-optimization study to evaluate the efficacy, safety, and tolerability of NBI-98854 titrated to the subject's optimal dose administered once daily (qd) for a total of 12 weeks of treatment in pediatric subjects with TS.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 127
• Est. completion date: November 16, 2018
• Est. primary completion date: November 1, 2018
• 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

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	Boston	Massachusetts
United States	Neurocrine Clinical Site	Bronx	New York
United States	Neurocrine Clinical Site	Chicago	Illinois
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	Hialeah	Florida
United States	Neurocrine Clinical Site	Hialeah	Florida
United States	Neurocrine Clinical Site	Hialeah	Florida
United States	Neurocrine Clinical Site	Houston	Texas
United States	Neurocrine Clinical Site	Iowa City	Iowa
United States	Neurocrine Clinical Site	Irving	Texas
United States	Neurocrine Clinical Site	Leawood	Kansas
United States	Neurocrine Clinical Site	Lincoln	Nebraska
United States	Neurocrine Clinical Site	Loxahatchee Groves	Florida
United States	Neurocrine Clinical Site	Memphis	Tennessee
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	Orange City	Florida
United States	Neurocrine Clinical Site	Orlando	Florida
United States	Neurocrine Clinical Site	Orlando	Florida
United States	Neurocrine Clinical Site	Rogers	Arkansas
United States	Neurocrine Clinical Site	San Diego	California
United States	Neurocrine Clinical Site	Santa Clarita	California
United States	Neurocrine Clinical Site	Seattle	Washington
United States	Neurocrine Clinical Site	Spokane	Washington
United States	Neurocrine Clinical Site	Sun City	Arizona
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, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline to Week 12 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (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.	Baseline, Week 12
Secondary	Change From Baseline to Week 12 in the Clinical Global Impression of Tics Severity (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.	Baseline, Week 12
Secondary	Participants Who Are a Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) Responder at Week 12	A TTS responder is defined, on a per-visit basis, as a participant whose TTS value is reduced by at least 30% from baseline at the specified postbaseline visit.	Baseline, Week 12
Secondary	Participants Who Are a Clinical Global Impression of Tourette Syndrome Improvement (CGI-TS-Improvement) Responder at Week 12	The CGI-TS-Improvement scale is used to assess overall improvement since the initiation of study drug dosing on a 7-point scale. A CGI-TS-Improvement responder is defined, on a per-visit basis, as a participant whose CGI-TS-Improvement score is 1 ("Very much improved") or 2 ("Much improved") at the specified postbaseline visit.	Week 12

External Links

•   Go to TForceGOLD.com to get information regarding the study and participating sites.