This is a Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of valbenazine to treat chorea in participants with Huntington disease.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
128
vesicular monoamine transporter 2 (VMAT2) inhibitor
non-active dosage form
Change From Screening Period Baseline to Maintenance Period in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score.
The TMC is part of the motor assessment of the UHDRS and measures chorea in 7 different body parts including the face, oral-buccal-lingual region, trunk and each limb independently. The TMC score is the sum of the individual scores and ranges from 0 to 28. A decrease in TMC scores indicates improvement in chorea symptoms.
Time frame: Baseline (average of screening and Day -1), maintenance (average of Weeks 10 and 12)
Percent of Clinical Global Impression of Change (CGI-C) Responders at Week 12
The CGI-C is a 7-point scale that rates the overall global change in chorea symptoms since the initiation of study drug dosing, ranging from 1 (very much improved) to 7 (very much worse), as assessed by the investigator or qualified clinician designee. Participants whose CGI-C score was either a 1 (very much improved) or a 2 (much improved) were classified as responders.
Time frame: Week 12
Percent of Patient Global Impression of Change (PGI-C) Responders at Week 12
The PGI-C is a 7-point scale that rates the overall global change in chorea symptoms since the initiation of study drug dosing, ranging from 1 (very much improved) to 7 (very much worse), as assessed by the participant. Participants whose PGI-C score was either a 1 (very much improved) or a 2 (much improved) were classified as responders.
Time frame: Week 12
Change From Baseline to Week 12 in the Quality of Life in Neurological Disorders (Neuro-QoL) Upper Extremity Function T-Score
The Neuro-QoL Upper Extremity Function Short Form consists of 8 questions about physical abilities, rated from 1 (unable to do) to 5 (without any difficulty). The Neuro-QoL scores were standardized as T-scores with a mean of 50 and standard deviation of 10. Scores below 50 indicated below average upper extremity function. The change from baseline to Week 12 in the Neuro-QoL Upper Extremity Function T-score are presented here. An increase in score indicates increased function.
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Neurocrine Clinical Site
Birmingham, Alabama, United States
Neurocrine Clinical Site
Little Rock, Arkansas, United States
Neurocrine Clinical Site
La Jolla, California, United States
Neurocrine Clinical Site
Sacramento, California, United States
Neurocrine Clinical Site
Aurora, Colorado, United States
Neurocrine Clinical Site
Englewood, Colorado, United States
Neurocrine Clinical Site
Washington D.C., District of Columbia, United States
Neurocrine Clinical Site
Gainesville, Florida, United States
Neurocrine Clinical Site
Miami, Florida, United States
Neurocrine Clinical Site
Atlanta, Georgia, United States
...and 36 more locations
Time frame: Baseline, Week 12
Change From Baseline to Week 12 in the Neuro-QoL Lower Extremity Function T-Score
The Neuro-QoL Lower Extremity Function Short Form consists of 8 questions about physical abilities, rated from 1 (unable to do) to 5 (without any difficulty). The Neuro-QoL scores were standardized as T-scores with a mean of 50 and standard deviation of 10. Scores below 50 indicated below average upper extremity function. The change from baseline to Week 12 in the Neuro-QoL Upper Extremity Function T-score are presented here. An increase in score indicates better function.
Time frame: Baseline, Week 12