Phase 3 N=128 Randomized Quadruple-blind Treatment

Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated With Huntington Disease

Chorea, Huntington

Bottom Line

View on ClinicalTrials.gov: NCT04102579 ↗

Enrolled (actual)

128

Serious AEs

2.4%

Results posted

Oct 2023

Primary outcome: Primary: Change From Screening Period Baseline to Maintenance Period in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score. — -4.60; -1.44 units on a scale — p=<0.0001

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Valbenazine (Drug); Placebo (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Neurocrine Biosciences
Primary completion: Oct 2021

Outcome Measures

Outcome	Result	p-value
PRIMARY Change From Screening Period Baseline to Maintenance Period in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score.	-4.60; -1.44	<0.0001 sig
SECONDARY Percent of Clinical Global Impression of Change (CGI-C) Responders at Week 12	42.9; 13.2	0.0007 sig
SECONDARY Percent of Patient Global Impression of Change (PGI-C) Responders at Week 12	52.7; 26.4	0.0062 sig
SECONDARY Change From Baseline to Week 12 in the Quality of Life in Neurological Disorders (Neuro-QoL) Upper Extremity Function T-Score	-1.58; -3.00	0.3304
SECONDARY Change From Baseline to Week 12 in the Neuro-QoL Lower Extremity Function T-Score	-0.27; 0.61	—

Summary

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.

Eligibility Criteria

Inclusion Criteria

Have a clinical diagnosis of Huntington Disease (HD) with chorea
Be able to walk, with or without the assistance of a person or device
Participants of childbearing potential who do not practice total abstinence must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) consistently while participating in the study until 30 days (females) or 90 days (males) after the last dose of the study drug
Be able to read and understand English

Exclusion Criteria

Have a history of previously established therapy with a VMAT2 inhibitor, in the judgement of the investigator
Have difficulty swallowing
Are currently pregnant or breastfeeding
Have a known history of long QT syndrome, cardiac tachyarrhythmia, left bundle-branch block, atrioventricular block, uncontrolled bradyarrhythmia, or heart failure
Have an unstable or serious medical or psychiatric illness
Have a significant risk of suicidal behavior
Have a history of substance dependence or substance (drug) or alcohol abuse, within 1 year of screening
If taking antidepressant therapy, be on a stable regimen
Have received gene therapy at any time
Have received an investigational drug in a clinical study within 30 days of the baseline visit or plan to use such investigational drug (other than valbenazine) during the study
Have had a blood loss ≥550 milliliters (mL) or donated blood within 30 days before the baseline visit
Had a medically significant illness within 30 days before baseline, or any history of neuroleptic malignant syndrome

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04102579). 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.