Phase 2 N=98 Randomized Quadruple-blind Treatment

Safety and Efficacy Study of NBI-98854 in Children and Adolescents With Tourette Syndrome

Tourette Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT02679079 ↗

Enrolled (actual)

Serious AEs

1.0%

Results posted

Jan 2021

Primary outcome: Primary: Change From Baseline to Week 6 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS) — -8.8; -7.3; -9.1 Score on scale — p=0.89

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: NBI-98854 (Drug); Placebo (Drug)
Age: Pediatric · 6+ yrs
Sex: All
Sponsor: Neurocrine Biosciences
Primary completion: Apr 2017

Outcome Measures

Outcome	Result	p-value
PRIMARY Change From Baseline to Week 6 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS)	-8.8; -7.3; -9.1	0.89
SECONDARY Clinical Global Impression of Tourette Syndrome (CGI-TS) - Improvement Score at Week 6	3.4; 3.5; 3.0	0.21
SECONDARY Participants Who Are a YGTSS TTS Responder at Week 6	10; 9; 15	0.24
SECONDARY Change From Baseline to Week 6 in the YGTSS Impairment Score	-8.5; -7.1; -10.4	0.52
SECONDARY Change From Baseline to Week 6 in the YGTSS Global Tic Severity Score	-17.2; -14.4; -19.5	0.60
SECONDARY Change From Baseline to Week 6 in the Rush Video-based Tic Rating Scale (RTRS) Total Score	-1.2; -1.6; -1.7	0.64
SECONDARY Change From Baseline to Week 6 in the Premonitory Urge for Tics Scale (PUTS) Total Score	-0.6; -1.0; 0.3	0.45
SECONDARY Change From Baseline to Week 6 in the Clinical Global Impression of Tics (CGI-Tics) - Severity Score	-1.1; -0.5; -0.9	0.41
SECONDARY Participants Who Are a CGI-TS-Improvement Responder at Week 6	6; 6; 11	0.18

Summary

Phase 2, double-blind, placebo-controlled study to assess the safety and efficacy of NBI-98854 administered once daily (qd) for a total of 6 weeks of treatment. This study will enroll approximately 90 male and female pediatric subjects clinically diagnosed with Tourette Syndrome.

Eligibility Criteria

Inclusion Criteria

Have a clinical diagnosis of Tourette Syndrome (TS)
Have at least moderate tic severity
Have TS symptoms that impair school, occupational, and/or social function
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
Be in good general health
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
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

Have an active, clinically significant unstable medical condition within 1 month prior to screening
Have a known history of long QT syndrome or cardiac arrhythmia
Have a known history of neuroleptic malignant syndrome
Have a cancer diagnosis within 3 years prior to screening (some exceptions allowed)
Have an allergy, hypersensitivity, or intolerance to VMAT2 inhibitors
Have a blood loss ≥250 mL or donated blood within 30 days prior to screening
Have a known history of substance dependence, substance (drug) or alcohol abuse
Have a significant risk of suicidal or violent behavior
Have initiated Comprehensive Behavioral Intervention for Tics (CBIT) during the screening period or at baseline or plan to initiate CBIT during the study
Have received an investigational drug within 30 days before screening or plan to use an investigational drug (other than NBI-98854) during the study

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02679079). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.