Clinical trial NCT04102579: Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated With Huntington Disease
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated With Huntington Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Chorea, Huntington
Keywords: Huntington's
Huntington
Chorea
Movement disorder
Valbenazine
HD
Tetrabenazine
VMAT2-Inhibitor
Lead sponsor: Neurocrine Biosciences
Sponsor class: INDUSTRY
Collaborators: name: Huntington Study Group
First posted: 2019-09-25
Enrollment: 128
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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:

1. Have a clinical diagnosis of Huntington Disease (HD) with chorea
2. Be able to walk, with or without the assistance of a person or device
3. 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
4. Be able to read and understand English

Exclusion Criteria:

1. Have a history of previously established therapy with a VMAT2 inhibitor, in the judgement of the investigator
2. Have difficulty swallowing
3. Are currently pregnant or breastfeeding
4. Have a known history of long QT syndrome, cardiac tachyarrhythmia, left bundle-branch block, atrioventricular block, uncontrolled bradyarrhythmia, or heart failure
5. Have an unstable or serious medical or psychiatric illness
6. Have a significant risk of suicidal behavior
7. Have a history of substance dependence or substance (drug) or alcohol abuse, within 1 year of screening
8. If taking antidepressant therapy, be on a stable regimen
9. Have received gene therapy at any time
10. 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
11. Have had a blood loss ≥550 milliliters (mL) or donated blood within 30 days before the baseline visit
12. Had a medically significant illness within 30 days before baseline, or any history of neuroleptic malignant syndrome

Primary outcomes:
measure: Change From Screening Period Baseline to Maintenance Period in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score.
timeFrame: Baseline (average of screening and Day -1), maintenance (average of Weeks 10 and 12)
Secondary outcomes:
measure: Percent of Clinical Global Impression of Change (CGI-C) Responders at Week 12
timeFrame: Week 12
measure: Percent of Patient Global Impression of Change (PGI-C) Responders at Week 12
timeFrame: Week 12
measure: Change From Baseline to Week 12 in the Quality of Life in Neurological Disorders (Neuro-QoL) Upper Extremity Function T-Score
timeFrame: Baseline, Week 12
measure: Change From Baseline to Week 12 in the Neuro-QoL Lower Extremity Function T-Score
timeFrame: Baseline, Week 12
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Neurocrine Clinical Site
state: Alabama
city: Little Rock
country: United States
facility: Neurocrine Clinical Site
state: Arkansas
city: La Jolla
country: United States
facility: Neurocrine Clinical Site
state: California
city: Sacramento
country: United States
facility: Neurocrine Clinical Site
state: California
city: Aurora
country: United States
facility: Neurocrine Clinical Site
state: Colorado
city: Englewood
country: United States
facility: Neurocrine Clinical Site
state: Colorado
city: Washington
country: United States
facility: Neurocrine Clinical Site
state: District of Columbia
city: Gainesville
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Miami
country: United States
facility: Neurocrine Clinical Site
state: Florida
city: Atlanta
country: United States
facility: Neurocrine Clinical Site
state: Georgia
city: Chicago
country: United States
facility: Neurocrine Clinical Site
state: Illinois
city: Chicago
country: United States
facility: Neurocrine Clinical Site
state: Illinois
city: Indianapolis
country: United States
facility: Neurocrine Clinical Site
state: Indiana
city: Iowa City
country: United States
facility: Neurocrine Clinical Site
state: Iowa
city: Kansas City
country: United States
facility: Neurocrine Clinical Site
state: Kansas
city: Wichita
country: United States
facility: Neurocrine Clinical Site
state: Kansas
city: Louisville
country: United States
facility: Neurocrine Clinical Site
state: Kentucky
city: New Orleans
country: United States
facility: Neurocrine Clinical Site
state: Louisiana
city: Boston
country: United States
facility: Neurocrine Clinical Site
state: Massachusetts
city: Boston
country: United States
facility: Neurocrine Clinical Site
state: Massachusetts
city: Charlestown
country: United States
facility: Neurocrine Clinical Site
state: Massachusetts
city: Ann Arbor
country: United States
facility: Neurocrine Clinical Site
state: Michigan
city: West Bloomfield
country: United States
facility: Neurocrine Clinical Site
state: Michigan
city: Omaha
country: United States
facility: Neurocrine Clinical Site
state: Nebraska
city: Rochester
country: United States
facility: Neurocrine Clinical Site
state: New York
city: Williamsville
country: United States
facility: Neurocrine Clinical Site
state: New York
city: Durham
country: United States
facility: Neurocrine Clinical Site
state: North Carolina
city: Fargo
country: United States
facility: Neurocrine Clinical Site
state: North Dakota
city: Cleveland
country: United States
facility: Neurocrine Clinical Site
state: Ohio
city: Columbus
country: United States
facility: Neurocrine Clinical Site
state: Ohio
city: Toledo
country: United States
facility: Neurocrine Clinical Site
state: Ohio
city: Pittsburgh
country: United States
facility: Neurocrine Clinical Site
state: Pennsylvania
city: Charleston
country: United States
facility: Neurocrine Clinical Site
state: South Carolina
city: Columbia
country: United States
facility: Neurocrine Clinical Site
state: South Carolina
city: Greenville
country: United States
facility: Neurocrine Clinical Site
state: South Carolina
city: Nashville
country: United States
facility: Neurocrine Clinical Site
state: Tennessee
city: Houston
country: United States
facility: Neurocrine Clinical Site
state: Texas
city: Salt Lake City
country: United States
facility: Neurocrine Clinical Site
state: Utah
city: Burlington
country: United States
facility: Neurocrine Clinical Site
state: Vermont
city: Charlottesville
country: United States
facility: Neurocrine Clinical Site
state: Virginia
city: Seattle
country: United States
facility: Neurocrine Clinical Site
state: Washington
city: Spokane
country: United States
facility: Neurocrine Clinical Site
state: Washington
city: Vancouver
country: Canada
facility: Neurocrine Clinical Site
state: British Columbia
city: Ottawa
country: Canada
facility: Neurocrine Clinical Site
state: Ontario
city: Toronto
country: Canada
facility: Neurocrine Clinical Site
state: Ontario
city: Toronto
country: Canada
facility: Neurocrine Clinical Site
state: Ontario
Study officials:
affiliation: Chief Medical Officer
name: Chief Medical Officer
role: STUDY_DIRECTOR
References:
citation: Furr Stimming E, Claassen DO, Kayson E, Goldstein J, Mehanna R, Zhang H, Liang GS, Haubenberger D; Huntington Study Group KINECT-HD Collaborators. Safety and efficacy of valbenazine for the treatment of chorea associated with Huntington's disease (KINECT-HD): a phase 3, randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2023 Jun;22(6):494-504. doi: 10.1016/S1474-4422(23)00127-8.
citation: Rodrigues FB, Wild EJ. Huntington's Disease Clinical Trials Corner: April 2020. J Huntingtons Dis. 2020;9(2):185-197. doi: 10.3233/JHD-200002.