Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington Disease

NCT04400331 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: NBI-98854-HD3006
• Study Type: interventional
• Target: 154
• Locations: 2 countries
• Sites: 34

Brief Summary

This is a Phase 3, open-label study to evaluate the long-term safety and tolerability of valbenazine, and to provide participants continued access to valbenazine for the treatment of chorea associated with Huntington disease.

Conditions

Chorea, Huntington

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Treatment-Emergent Adverse Events (TEAEs)

  Up to 262 weeks

Secondary Outcomes (1)

• Change from Baseline in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score

  Up to 262 weeks

Study Arms (1)

Valbenazine

EXPERIMENTAL

Capsule, administered orally once daily.

Drug: Valbenazine

Interventions

Valbenazine DRUG

vesicular monoamine transporter 2 (VMAT2) inhibitor

Also known as: NBI-98854

Valbenazine

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 participated in Study NBI-98854-HD3005 and
• a. Study drug dosing completion of Study NBI-98854-HD3005, as demonstrated by completed study drug dosing through the follow-up visit or early terminated Study NBI-98854-HD3005 for administrative reasons due to COVID-19 (for example, site closure related to COVID-19)
• Did not participate in Study NBI-98854-HD3005 and
• Have a clinical and genetic diagnosis of Huntington Disease (HD) with chorea
• Be able to walk, with or without the assistance of a person or device
• Be able to read and understand English and capable of providing consent to study participation or have a legally authorized representative providing consent and the participant providing assent
• Participants of childbearing potential must agree to use contraception consistently while participating in the study until 30 days after last dose of the study treatment

You may not qualify if:

• Have difficulty swallowing
• Are currently pregnant or breastfeeding
• Have a known history of long QT syndrome, cardiac tachyarrhythmia, left bundle-branch block, atrioventricular (AV) 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
• Have received gene therapy at any time
• Have received an investigational drug in a clinical study (other than valbenazine) within 30 days before the baseline visit or plan to use such investigational drug (other than valbenazine) during the study
• Have had a blood loss ≥550 mL or donated blood within 30 days before the baseline visit
• Have a history of severe hepatic impairment or history of protocol specified hematologic abnormalities during the course of the NBI-98854-HD3005 study
• Had a medically significant illness within 30 days before baseline, or any history of neuroleptic malignant syndrome
• Have a known hypersensitivity to any component of the formulation of valbenazine
• For participants who did not participate in NBI-98854-HD3005: have a history of VMAT2 inhibitor use within 30 days of baseline

Sponsors & Collaborators

• Neurocrine Biosciences: lead
• Huntington Study Group: collaborator

Study Sites (34)

Neurocrine Clinical Site

Birmingham, Alabama, 35294, 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

Aurora, Colorado, 80045, United States

Location

Neurocrine Clinical Site

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

Location

Neurocrine Clinical Site

Boca Raton, Florida, 33486, United States

Location

Neurocrine Clinical Site

Gainesville, Florida, 32608, 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

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, 02215, United States

Location

Neurocrine Clinical Site

Charlestown, Massachusetts, 02129, United States

Location

Neurocrine Clinical Site

Ann Arbor, Michigan, 48105, United States

Location

Neurocrine Clinical Site

Omaha, Nebraska, 68105, 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

Columbus, Ohio, 43221, 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

Burlington, Vermont, 05401, 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, MK2 1E1, Canada

Location

Related Links

• Study website

MeSH Terms

Conditions

Huntington Disease

Interventions

valbenazine

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Chorea; Dyskinesias; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Study Officials

• Chief Medical Officer

  Chief Medical Officer

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 19, 2020
• First Posted: May 22, 2020
• Study Start: September 18, 2020
• Primary Completion: March 1, 2026
• Study Completion: March 1, 2026
• Last Updated: December 24, 2024

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (31); CA (3)