Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington Disease
Primary Purpose
Chorea, Huntington
Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Valbenazine
Sponsored by
About this trial
This is an interventional treatment trial for Chorea, Huntington
Eligibility Criteria
Inclusion Criteria:
Either #1 or #2 must be met for inclusion eligibility.
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
Exclusion Criteria:
- 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
Sites / Locations
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
- Neurocrine Clinical SiteRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Valbenazine
Arm Description
Capsule, administered orally once daily.
Outcomes
Primary Outcome Measures
Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
Secondary Outcome Measures
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.
Full Information
NCT ID
NCT04400331
First Posted
May 19, 2020
Last Updated
October 31, 2022
Sponsor
Neurocrine Biosciences
Collaborators
Huntington Study Group
1. Study Identification
Unique Protocol Identification Number
NCT04400331
Brief Title
Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington Disease
Official Title
Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington Disease
Study Type
Interventional
2. Study Status
Record Verification Date
October 2022
Overall Recruitment Status
Recruiting
Study Start Date
September 18, 2020 (Actual)
Primary Completion Date
December 2023 (Anticipated)
Study Completion Date
December 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neurocrine Biosciences
Collaborators
Huntington Study Group
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Chorea, Huntington
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
150 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Valbenazine
Arm Type
Experimental
Arm Description
Capsule, administered orally once daily.
Intervention Type
Drug
Intervention Name(s)
Valbenazine
Other Intervention Name(s)
NBI-98854
Intervention Description
vesicular monoamine transporter 2 (VMAT2) inhibitor
Primary Outcome Measure Information:
Title
Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
Time Frame
Up to 156 weeks
Secondary Outcome Measure Information:
Title
Change from Baseline in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score
Description
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.
Time Frame
Up to 156 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Either #1 or #2 must be met for inclusion eligibility.
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
Exclusion Criteria:
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Neurocrine Medical Information Call Center
Phone
877-641-3461
Email
medinfo@neurocrine.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Chief Medical Officer
Organizational Affiliation
Chief Medical Officer
Official's Role
Study Director
Facility Information:
Facility Name
Neurocrine Clinical Site
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72205
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
La Jolla
State/Province
California
ZIP/Postal Code
92037
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Boca Raton
State/Province
Florida
ZIP/Postal Code
33431
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32608
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Wichita
State/Province
Kansas
ZIP/Postal Code
67226
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70121
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Charlestown
State/Province
Massachusetts
ZIP/Postal Code
02129
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48105
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68105
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Rochester
State/Province
New York
ZIP/Postal Code
14618
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Williamsville
State/Province
New York
ZIP/Postal Code
14221
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43221
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Toledo
State/Province
Ohio
ZIP/Postal Code
43614
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Columbia
State/Province
South Carolina
ZIP/Postal Code
29203
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Greenville
State/Province
South Carolina
ZIP/Postal Code
29615
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37212
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77054
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Burlington
State/Province
Vermont
ZIP/Postal Code
05401
Country
United States
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6T 2B5
Country
Canada
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1Y 4E9
Country
Canada
Individual Site Status
Recruiting
Facility Name
Neurocrine Clinical Site
City
Toronto
State/Province
Ontario
ZIP/Postal Code
MK2 1E1
Country
Canada
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
No
Links:
URL
https://huntingtonstudygroup.org/current-clinical-trials/kinect-hd2/
Description
Study website
Learn more about this trial
Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington Disease
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