Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated With Huntington Disease (KINECT-HD)
Primary Purpose
Chorea, Huntington
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Valbenazine
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Chorea, Huntington focused on measuring Huntington's, Huntington, Chorea, Movement disorder, Valbenazine, HD, Tetrabenazine, VMAT2-Inhibitor
Eligibility Criteria
Inclusion Criteria:
- Have a clinical diagnosis of Huntington Disease (HD) with chorea
- Be able to walk, with or without the assistance of a person or device
- Subjects 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
Exclusion Criteria:
- 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 (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
- 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 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
Sites / Locations
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Valbenazine
Placebo
Arm Description
Capsule, administered orally once daily for 12 weeks.
Capsule, administered orally once daily for 12 weeks.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Percent of Clinical Global Impression of Change (CGI-C) Responders at Week 12
The CGI-C is a 7-point scale that rates the overall global change in chorea symptoms since the initiation of study drug dosing, ranging from 1 (very much improved) to 7 (very much worse), as assessed by the investigator or qualified clinician designee.
Participants whose CGI-C score was either a 1 (very much improved) or a 2 (much improved) were classified as responders.
Percent of Patient Global Impression of Change (PGI-C) Responders at Week 12
The PGI-C is a 7-point scale that rates the overall global change in chorea symptoms since the initiation of study drug dosing, ranging from 1 (very much improved) to 7 (very much worse), as assessed by the participant.
Participants whose PGI-C score was either a 1 (very much improved) or a 2 (much improved) were classified as responders.
Change From Baseline to Week 12 in the Quality of Life in Neurological Disorders (Neuro-QoL) Upper Extremity Function T-Score
The Neuro-QoL Upper Extremity Function Short Form consists of 8 questions about physical abilities, rated from 1 (unable to do) to 5 (without any difficulty). The Neuro-QoL scores were standardized as T-scores with a mean of 50 and standard deviation of 10. Scores below 50 indicated below average upper extremity function. The change from baseline to Week 12 in the Neuro-QoL Upper Extremity Function T-score are presented here. An increase in score indicates increased function.
Change From Baseline to Week 12 in the Neuro-QoL Lower Extremity Function T-Score
The Neuro-QoL Lower Extremity Function Short Form consists of 8 questions about physical abilities, rated from 1 (unable to do) to 5 (without any difficulty). The Neuro-QoL scores were standardized as T-scores with a mean of 50 and standard deviation of 10. Scores below 50 indicated below average upper extremity function. The change from baseline to Week 12 in the Neuro-QoL Upper Extremity Function T-score are presented here. An increase in score indicates better function.
Full Information
NCT ID
NCT04102579
First Posted
September 23, 2019
Last Updated
September 15, 2023
Sponsor
Neurocrine Biosciences
Collaborators
Huntington Study Group
1. Study Identification
Unique Protocol Identification Number
NCT04102579
Brief Title
Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated With Huntington Disease
Acronym
KINECT-HD
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
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Completed
Study Start Date
November 13, 2019 (Actual)
Primary Completion Date
October 15, 2021 (Actual)
Study Completion Date
October 26, 2021 (Actual)
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, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and tolerability of valbenazine to treat chorea in participants with Huntington disease.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Chorea, Huntington
Keywords
Huntington's, Huntington, Chorea, Movement disorder, Valbenazine, HD, Tetrabenazine, VMAT2-Inhibitor
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
128 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Valbenazine
Arm Type
Experimental
Arm Description
Capsule, administered orally once daily for 12 weeks.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Capsule, administered orally once daily for 12 weeks.
Intervention Type
Drug
Intervention Name(s)
Valbenazine
Other Intervention Name(s)
NBI-98854
Intervention Description
vesicular monoamine transporter 2 (VMAT2) inhibitor
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
non-active dosage form
Primary Outcome Measure Information:
Title
Change From Screening Period Baseline to Maintenance Period 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 TMC scores indicates improvement in chorea symptoms.
Time Frame
Baseline (average of screening and Day -1), maintenance (average of Weeks 10 and 12)
Secondary Outcome Measure Information:
Title
Percent of Clinical Global Impression of Change (CGI-C) Responders at Week 12
Description
The CGI-C is a 7-point scale that rates the overall global change in chorea symptoms since the initiation of study drug dosing, ranging from 1 (very much improved) to 7 (very much worse), as assessed by the investigator or qualified clinician designee.
Participants whose CGI-C score was either a 1 (very much improved) or a 2 (much improved) were classified as responders.
Time Frame
Week 12
Title
Percent of Patient Global Impression of Change (PGI-C) Responders at Week 12
Description
The PGI-C is a 7-point scale that rates the overall global change in chorea symptoms since the initiation of study drug dosing, ranging from 1 (very much improved) to 7 (very much worse), as assessed by the participant.
Participants whose PGI-C score was either a 1 (very much improved) or a 2 (much improved) were classified as responders.
Time Frame
Week 12
Title
Change From Baseline to Week 12 in the Quality of Life in Neurological Disorders (Neuro-QoL) Upper Extremity Function T-Score
Description
The Neuro-QoL Upper Extremity Function Short Form consists of 8 questions about physical abilities, rated from 1 (unable to do) to 5 (without any difficulty). The Neuro-QoL scores were standardized as T-scores with a mean of 50 and standard deviation of 10. Scores below 50 indicated below average upper extremity function. The change from baseline to Week 12 in the Neuro-QoL Upper Extremity Function T-score are presented here. An increase in score indicates increased function.
Time Frame
Baseline, Week 12
Title
Change From Baseline to Week 12 in the Neuro-QoL Lower Extremity Function T-Score
Description
The Neuro-QoL Lower Extremity Function Short Form consists of 8 questions about physical abilities, rated from 1 (unable to do) to 5 (without any difficulty). The Neuro-QoL scores were standardized as T-scores with a mean of 50 and standard deviation of 10. Scores below 50 indicated below average upper extremity function. The change from baseline to Week 12 in the Neuro-QoL Upper Extremity Function T-score are presented here. An increase in score indicates better function.
Time Frame
Baseline, Week 12
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:
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
Exclusion Criteria:
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
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
35233
Country
United States
Facility Name
Neurocrine Clinical Site
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72205
Country
United States
Facility Name
Neurocrine Clinical Site
City
La Jolla
State/Province
California
ZIP/Postal Code
92037
Country
United States
Facility Name
Neurocrine Clinical Site
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Neurocrine Clinical Site
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Neurocrine Clinical Site
City
Englewood
State/Province
Colorado
ZIP/Postal Code
80113
Country
United States
Facility Name
Neurocrine Clinical Site
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007
Country
United States
Facility Name
Neurocrine Clinical Site
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32608
Country
United States
Facility Name
Neurocrine Clinical Site
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Neurocrine Clinical Site
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Facility Name
Neurocrine Clinical Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Neurocrine Clinical Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Neurocrine Clinical Site
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Neurocrine Clinical Site
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
Neurocrine Clinical Site
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Neurocrine Clinical Site
City
Wichita
State/Province
Kansas
ZIP/Postal Code
67226
Country
United States
Facility Name
Neurocrine Clinical Site
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Facility Name
Neurocrine Clinical Site
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70121
Country
United States
Facility Name
Neurocrine Clinical Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02118
Country
United States
Facility Name
Neurocrine Clinical Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
Neurocrine Clinical Site
City
Charlestown
State/Province
Massachusetts
ZIP/Postal Code
02129
Country
United States
Facility Name
Neurocrine Clinical Site
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Neurocrine Clinical Site
City
West Bloomfield
State/Province
Michigan
ZIP/Postal Code
48322
Country
United States
Facility Name
Neurocrine Clinical Site
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68198
Country
United States
Facility Name
Neurocrine Clinical Site
City
Rochester
State/Province
New York
ZIP/Postal Code
14618
Country
United States
Facility Name
Neurocrine Clinical Site
City
Williamsville
State/Province
New York
ZIP/Postal Code
14221
Country
United States
Facility Name
Neurocrine Clinical Site
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705
Country
United States
Facility Name
Neurocrine Clinical Site
City
Fargo
State/Province
North Dakota
ZIP/Postal Code
58103
Country
United States
Facility Name
Neurocrine Clinical Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Neurocrine Clinical Site
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
Neurocrine Clinical Site
City
Toledo
State/Province
Ohio
ZIP/Postal Code
43614
Country
United States
Facility Name
Neurocrine Clinical Site
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Neurocrine Clinical Site
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Neurocrine Clinical Site
City
Columbia
State/Province
South Carolina
ZIP/Postal Code
29203
Country
United States
Facility Name
Neurocrine Clinical Site
City
Greenville
State/Province
South Carolina
ZIP/Postal Code
29615
Country
United States
Facility Name
Neurocrine Clinical Site
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37212
Country
United States
Facility Name
Neurocrine Clinical Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77054
Country
United States
Facility Name
Neurocrine Clinical Site
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Facility Name
Neurocrine Clinical Site
City
Burlington
State/Province
Vermont
ZIP/Postal Code
05401
Country
United States
Facility Name
Neurocrine Clinical Site
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
Neurocrine Clinical Site
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Facility Name
Neurocrine Clinical Site
City
Spokane
State/Province
Washington
ZIP/Postal Code
99202
Country
United States
Facility Name
Neurocrine Clinical Site
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V6T 2B5
Country
Canada
Facility Name
Neurocrine Clinical Site
City
Ottawa
State/Province
Ontario
ZIP/Postal Code
K1Y 4E9
Country
Canada
Facility Name
Neurocrine Clinical Site
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M2K 1E1
Country
Canada
Facility Name
Neurocrine Clinical Site
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M3B 2S7
Country
Canada
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
37210099
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. Erratum In: Lancet Neurol. 2023 Sep;22(9):e10. Lancet Neurol. 2023 Sep;22(9):e10.
Results Reference
result
PubMed Identifier
32250312
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.
Results Reference
derived
Links:
URL
http://www.kinect-hd.org
Description
Study website
Learn more about this trial
Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated With Huntington Disease
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