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

NCT04102579 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: NBI-98854-HD3005
• Study Type: interventional
• Target: 128
• Locations: 2 countries
• Sites: 46

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.

Conditions

Chorea, Huntington

Keywords

Huntington's; Huntington; Chorea; Movement disorder; Valbenazine; HD; Tetrabenazine; VMAT2-Inhibitor

Outcome Measures

Primary Outcomes (1)

• 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.

  Baseline (average of screening and Day -1), maintenance (average of Weeks 10 and 12)

Secondary Outcomes (4)

• Percent of Clinical Global Impression of Change (CGI-C) Responders at Week 12

  Week 12

• Percent of Patient Global Impression of Change (PGI-C) Responders at Week 12

  Week 12

• Change From Baseline to Week 12 in the Quality of Life in Neurological Disorders (Neuro-QoL) Upper Extremity Function T-Score

  Baseline, Week 12

• Change From Baseline to Week 12 in the Neuro-QoL Lower Extremity Function T-Score

  Baseline, Week 12

Study Arms (2)

Valbenazine

EXPERIMENTAL

Capsule, administered orally once daily for 12 weeks.

Drug: Valbenazine

Placebo

PLACEBO COMPARATOR

Capsule, administered orally once daily for 12 weeks.

Drug: Placebo

Interventions

Valbenazine DRUG

vesicular monoamine transporter 2 (VMAT2) inhibitor

Also known as: NBI-98854

Valbenazine

Placebo DRUG

non-active dosage form

Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Neurocrine Biosciences: lead
• Huntington Study Group: collaborator

Study Sites (46)

Neurocrine Clinical Site

Birmingham, Alabama, 35233, United States

Location

Neurocrine Clinical Site

Little Rock, Arkansas, 72205, United States

Location

Neurocrine Clinical Site

La Jolla, California, 92037, United States

Location

Neurocrine Clinical Site

Sacramento, California, 95817, United States

Location

Neurocrine Clinical Site

Aurora, Colorado, 80045, United States

Location

Neurocrine Clinical Site

Englewood, Colorado, 80113, United States

Location

Neurocrine Clinical Site

Washington D.C., District of Columbia, 20007, United States

Location

Neurocrine Clinical Site

Gainesville, Florida, 32608, United States

Location

Neurocrine Clinical Site

Miami, Florida, 33136, United States

Location

Neurocrine Clinical Site

Atlanta, Georgia, 30329, United States

Location

Neurocrine Clinical Site

Chicago, Illinois, 60611, United States

Location

Neurocrine Clinical Site

Chicago, Illinois, 60612, United States

Location

Neurocrine Clinical Site

Indianapolis, Indiana, 46202, United States

Location

Neurocrine Clinical Site

Iowa City, Iowa, 52242, United States

Location

Neurocrine Clinical Site

Kansas City, Kansas, 66160, United States

Location

Neurocrine Clinical Site

Wichita, Kansas, 67226, United States

Location

Neurocrine Clinical Site

Louisville, Kentucky, 40202, United States

Location

Neurocrine Clinical Site

New Orleans, Louisiana, 70121, United States

Location

Neurocrine Clinical Site

Boston, Massachusetts, 02118, United States

Location

Neurocrine Clinical Site

Boston, Massachusetts, 02215, United States

Location

Neurocrine Clinical Site

Charlestown, Massachusetts, 02129, United States

Location

Neurocrine Clinical Site

Ann Arbor, Michigan, 48109, United States

Location

Neurocrine Clinical Site

West Bloomfield, Michigan, 48322, United States

Location

Neurocrine Clinical Site

Omaha, Nebraska, 68198, United States

Location

Neurocrine Clinical Site

Rochester, New York, 14618, United States

Location

Neurocrine Clinical Site

Williamsville, New York, 14221, United States

Location

Neurocrine Clinical Site

Durham, North Carolina, 27705, United States

Location

Neurocrine Clinical Site

Fargo, North Dakota, 58103, United States

Location

Neurocrine Clinical Site

Cleveland, Ohio, 44195, United States

Location

Neurocrine Clinical Site

Columbus, Ohio, 43210, United States

Location

Neurocrine Clinical Site

Toledo, Ohio, 43614, United States

Location

Neurocrine Clinical Site

Pittsburgh, Pennsylvania, 15213, United States

Location

Neurocrine Clinical Site

Charleston, South Carolina, 29425, United States

Location

Neurocrine Clinical Site

Columbia, South Carolina, 29203, United States

Location

Neurocrine Clinical Site

Greenville, South Carolina, 29615, United States

Location

Neurocrine Clinical Site

Nashville, Tennessee, 37212, United States

Location

Neurocrine Clinical Site

Houston, Texas, 77054, United States

Location

Neurocrine Clinical Site

Salt Lake City, Utah, 84108, United States

Location

Neurocrine Clinical Site

Burlington, Vermont, 05401, United States

Location

Neurocrine Clinical Site

Charlottesville, Virginia, 22908, United States

Location

Neurocrine Clinical Site

Seattle, Washington, 98195, United States

Location

Neurocrine Clinical Site

Spokane, Washington, 99202, United States

Location

Neurocrine Clinical Site

Vancouver, British Columbia, V6T 2B5, Canada

Location

Neurocrine Clinical Site

Ottawa, Ontario, K1Y 4E9, Canada

Location

Neurocrine Clinical Site

Toronto, Ontario, M2K 1E1, Canada

Location

Neurocrine Clinical Site

Toronto, Ontario, M3B 2S7, Canada

Location

Related Publications (2)

• 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.

  PMID: 37210099 RESULT

• 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.

  PMID: 32250312 DERIVED

Related Links

• Study website

MeSH Terms

Conditions

Huntington Disease; Chorea; Movement Disorders

Interventions

valbenazine

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Dyskinesias; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Neurocrine Medical Information Call Center
• Organization: Neurocrine Biosciences

medinfo@neurocrine.com

877-641-3461

Study Officials

• Chief Medical Officer

  Chief Medical Officer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 23, 2019
• First Posted: September 25, 2019
• Study Start: November 13, 2019
• Primary Completion: October 15, 2021
• Study Completion: October 26, 2021
• Last Updated: October 11, 2023
• Results First Posted: October 11, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

CA (4); US (42)