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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Chorea, Huntington
• Intervention / Treatment: Drug: Valbenazine

Detailed Description

After completion of Week 156/early termination visit, participants in the US will be given the option to continue into an extended maintenance period for up to 104 weeks and participants in Canada will have the option to participate in a separate open-label study (Study NBI-98854-HD3022).

• Study Type: Interventional
• Enrollment (Actual): 154
• Phase: Phase 3

Contacts and Locations

Study Locations

• Canada
  • British Columbia
    • Vancouver, British Columbia, Canada, V6T 2B5
      • Neurocrine Clinical Site
  • Ontario
    • Ottawa, Ontario, Canada, K1Y 4E9
      • Neurocrine Clinical Site
    • Toronto, Ontario, Canada, MK2 1E1
      • Neurocrine Clinical Site
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35294
      • Neurocrine Clinical Site
  • Arkansas
    • Little Rock, Arkansas, United States, 72205
      • Neurocrine Clinical Site
  • California
    • La Jolla, California, United States, 92037
      • Neurocrine Clinical Site
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Neurocrine Clinical Site
  • District of Columbia
    • Washington, District of Columbia, United States, 20007
      • Neurocrine Clinical Site
  • Florida
    • Boca Raton, Florida, United States, 33486
      • Neurocrine Clinical Site
    • Gainesville, Florida, United States, 32608
      • Neurocrine Clinical Site
  • Georgia
    • Atlanta, Georgia, United States, 30329
      • Neurocrine Clinical Site
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Neurocrine Clinical Site
    • Chicago, Illinois, United States, 60612
      • Neurocrine Clinical Site
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Neurocrine Clinical Site
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • Neurocrine Clinical Site
  • Kansas
    • Wichita, Kansas, United States, 67226
      • Neurocrine Clinical Site
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • Neurocrine Clinical Site
  • Louisiana
    • New Orleans, Louisiana, United States, 70121
      • Neurocrine Clinical Site
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Neurocrine Clinical Site
    • Charlestown, Massachusetts, United States, 02129
      • Neurocrine Clinical Site
  • Michigan
    • Ann Arbor, Michigan, United States, 48105
      • Neurocrine Clinical Site
  • Nebraska
    • Omaha, Nebraska, United States, 68105
      • Neurocrine Clinical Site
  • New York
    • Rochester, New York, United States, 14618
      • Neurocrine Clinical Site
    • Williamsville, New York, United States, 14221
      • Neurocrine Clinical Site
  • North Carolina
    • Durham, North Carolina, United States, 27705
      • Neurocrine Clinical Site
  • Ohio
    • Columbus, Ohio, United States, 43221
      • Neurocrine Clinical Site
    • Toledo, Ohio, United States, 43614
      • Neurocrine Clinical Site
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15213
      • Neurocrine Clinical Site
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Neurocrine Clinical Site
    • Columbia, South Carolina, United States, 29203
      • Neurocrine Clinical Site
    • Greenville, South Carolina, United States, 29615
      • Neurocrine Clinical Site
  • Tennessee
    • Nashville, Tennessee, United States, 37212
      • Neurocrine Clinical Site
  • Texas
    • Houston, Texas, United States, 77054
      • Neurocrine Clinical Site
  • Vermont
    • Burlington, Vermont, United States, 05401
      • Neurocrine Clinical Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 71 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

Either #1 or #2 must be met for inclusion eligibility.

1. 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)

2. Did not participate in Study NBI-98854-HD3005 and

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

Exclusion Criteria:

1. Have difficulty swallowing
2. Are currently pregnant or breastfeeding
3. Have a known history of long QT syndrome, cardiac tachyarrhythmia, left bundle-branch block, atrioventricular (AV) block, uncontrolled bradyarrhythmia, or heart failure
4. Have an unstable or serious medical or psychiatric illness
5. Have a significant risk of suicidal behavior
6. Have a history of substance dependence or substance (drug) or alcohol abuse, within 1 year of screening
7. Have received gene therapy at any time
8. 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
9. Have had a blood loss ≥550 mL or donated blood within 30 days before the baseline visit
10. Have a history of severe hepatic impairment or history of protocol specified hematologic abnormalities during the course of the NBI-98854-HD3005 study
11. Had a medically significant illness within 30 days before baseline, or any history of neuroleptic malignant syndrome
12. Have a known hypersensitivity to any component of the formulation of valbenazine
13. For participants who did not participate in NBI-98854-HD3005: have a history of VMAT2 inhibitor use within 30 days of baseline

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Valbenazine; Capsule, administered orally once daily.	Drug: Valbenazine; vesicular monoamine transporter 2 (VMAT2) inhibitor; Other Names: NBI-98854

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Participants with Treatment-Emergent Adverse Events (TEAEs)	Up to 262 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from Baseline 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 score indicates improvement in chorea.	Up to 262 weeks

Collaborators and Investigators

• Sponsor: Neurocrine Biosciences
• Collaborators: Huntington Study Group
• Investigators: Study Director: Chief Medical Officer, Chief Medical Officer

Publications and helpful links

Helpful Links

• Study website

Study record dates

Study Major Dates

• Study Start (Actual): September 18, 2020
• Primary Completion (Estimated): March 1, 2026
• Study Completion (Estimated): March 1, 2026

Study Registration Dates

• First Submitted: May 19, 2020
• First Submitted That Met QC Criteria: May 19, 2020
• First Posted (Actual): May 22, 2020

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: December 19, 2024
• Last Verified: December 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neurologic Manifestations; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Mental Disorders; Genetic Diseases, Inborn; Neurocognitive Disorders; Cognition Disorders; Dementia; Neurodegenerative Diseases; Movement Disorders; Heredodegenerative Disorders, Nervous System; Basal Ganglia Diseases; Dyskinesias; Huntington Disease; Chorea
• Other Study ID Numbers: NBI-98854-HD3006

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No