Chorea, HuntingtonPhase 3KINECT-HDNCT04102579

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

A Phase 3 study testing Valbenazine in people with Chorea, Huntington, run by Neurocrine Biosciences at 46 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages 18 to 75 years
Conditions the study is for: Chorea, Huntington
What is being tested?: Valbenazine
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 128 participants
Where: 46 sitesin 2 countries
Study dates: 13 Nov 2019to 26 Oct 2021
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 75 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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

Who cannot join

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

Show all 12Show fewer

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

Trial locations and contacts

46 sites in 2 countries.

Country

City, postcode or hospital

46 sites in 2 countries

United States42 sites

Neurocrine Clinical Site
Ann Arbor, Michigan, 48109
Neurocrine Clinical Site
Atlanta, Georgia, 30329
Neurocrine Clinical Site
Aurora, Colorado, 80045
Neurocrine Clinical Site
Birmingham, Alabama, 35233
Neurocrine Clinical Site
Boston, Massachusetts, 02118
Neurocrine Clinical Site
Boston, Massachusetts, 02215
Neurocrine Clinical Site
Burlington, Vermont, 05401
Neurocrine Clinical Site
Charleston, South Carolina, 29425
Neurocrine Clinical Site
Charlestown, Massachusetts, 02129
Neurocrine Clinical Site
Charlottesville, Virginia, 22908
Neurocrine Clinical Site
Chicago, Illinois, 60611
Neurocrine Clinical Site
Chicago, Illinois, 60612
Neurocrine Clinical Site
Cleveland, Ohio, 44195
Neurocrine Clinical Site
Columbia, South Carolina, 29203
Neurocrine Clinical Site
Columbus, Ohio, 43210
Neurocrine Clinical Site
Durham, North Carolina, 27705
Neurocrine Clinical Site
Englewood, Colorado, 80113
Neurocrine Clinical Site
Fargo, North Dakota, 58103
Neurocrine Clinical Site
Gainesville, Florida, 32608
Neurocrine Clinical Site
Greenville, South Carolina, 29615
Neurocrine Clinical Site
Houston, Texas, 77054
Neurocrine Clinical Site
Indianapolis, Indiana, 46202
Neurocrine Clinical Site
Iowa City, Iowa, 52242
Neurocrine Clinical Site
Kansas City, Kansas, 66160
Neurocrine Clinical Site
La Jolla, California, 92037
Neurocrine Clinical Site
Little Rock, Arkansas, 72205
Neurocrine Clinical Site
Louisville, Kentucky, 40202
Neurocrine Clinical Site
Miami, Florida, 33136
Neurocrine Clinical Site
Nashville, Tennessee, 37212
Neurocrine Clinical Site
New Orleans, Louisiana, 70121
Neurocrine Clinical Site
Omaha, Nebraska, 68198
Neurocrine Clinical Site
Pittsburgh, Pennsylvania, 15213
Neurocrine Clinical Site
Rochester, New York, 14618
Neurocrine Clinical Site
Sacramento, California, 95817
Neurocrine Clinical Site
Salt Lake City, Utah, 84108
Neurocrine Clinical Site
Seattle, Washington, 98195
Neurocrine Clinical Site
Spokane, Washington, 99202
Neurocrine Clinical Site
Toledo, Ohio, 43614
Neurocrine Clinical Site
Washington D.C., District of Columbia, 20007
Neurocrine Clinical Site
West Bloomfield, Michigan, 48322
Neurocrine Clinical Site
Wichita, Kansas, 67226
Neurocrine Clinical Site
Williamsville, New York, 14221

Other trials in United States

Canada4 sites

Neurocrine Clinical Site
Ottawa, Ontario, K1Y 4E9
Neurocrine Clinical Site
Toronto, Ontario, M2K 1E1
Neurocrine Clinical Site
Toronto, Ontario, M3B 2S7
Neurocrine Clinical Site
Vancouver, British Columbia, V6T 2B5

Other trials in Canada

About this study

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

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
Being tested: Valbenazine, Placebo
Keywords: Huntington's, Huntington, Chorea, Movement disorder, Valbenazine, HD, Tetrabenazine, VMAT2-Inhibitor
Medical subject headings: Huntington Disease, Chorea, Movement Disorders

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

ValbenazineDrug
Also called NBI-98854
vesicular monoamine transporter 2 (VMAT2) inhibitor
PlaceboDrug
non-active dosage form

Study groups in NCT04102579
Study group	Role	What they receive
Valbenazine	Experimental	Capsule, administered orally once daily for 12 weeks. Receives: Drug: Valbenazine
Placebo	Placebo comparator	Capsule, administered orally once daily for 12 weeks. Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: 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
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.
Measured: Week 12
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.
Measured: Week 12
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.
Measured: Baseline, Week 12
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.
Measured: Baseline, Week 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

13 Nov 201926 Oct 2021

First posted25 Sep 2019
Study start13 Nov 2019
Primary completion15 Oct 2021
Study completion26 Oct 2021

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Chorea, HuntingtonStudies of the same condition, open and under way.
More trials run by Neurocrine BiosciencesThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 128 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04102579
Sponsor's protocol ID: NBI-98854-HD3005
Acronym: KINECT-HD
Status last verified: Sep 2023
First submitted: 23 Sep 2019
Last updated: 11 Oct 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Publications

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.
PubMed 37210099
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.
PubMed 32250312

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Chorea, Huntington trials

Study at a glance

From the registry record, last updated by the sponsor on 11 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT04102579
Acronym / sponsor ID: KINECT-HD / NBI-98854-HD3005
Conditions: Chorea, Huntington
Keywords: Huntington's, Huntington, Chorea, Movement disorder, Valbenazine, HD, Tetrabenazine, VMAT2-Inhibitor
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 128 participants (actual)
Lead sponsor: Neurocrine BiosciencesIndustry (pharma, biotech, devices)
Collaborators: Huntington Study Group
Study start: 13 Nov 2019 (actual)
Primary completion: 15 Oct 2021 (actual)
Study completion: 26 Oct 2021 (actual)
First posted: 25 Sep 2019 (actual)
Results: Posted 11 Oct 2023

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 2

Arm	Type	Receives
Valbenazine	Experimental	Drug: ValbenazineCapsule, administered orally once daily for 12 weeks.
Placebo	Placebo comparator	Drug: PlaceboCapsule, administered orally once daily for 12 weeks.

Who may qualify

Ages: 18 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

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

Key exclusion criteria (12 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
ValbenazineAlso: NBI-98854vesicular monoamine transporter 2 (VMAT2) inhibitor	Drug	Valbenazine
Placebonon-active dosage form	Drug	Placebo

Primary outcome

Change From Screening Period Baseline to Maintenance Period in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score.
Time frame: Baseline (average of screening and Day -1), maintenance (average of Weeks 10 and 12)
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.

4 secondary outcomes

Percent of Clinical Global Impression of Change (CGI-C) Responders at Week 12
Time frame: Week 12
Percent of Patient Global Impression of Change (PGI-C) Responders at Week 12
Time frame: Week 12
Change From Baseline to Week 12 in the Quality of Life in Neurological Disorders (Neuro-QoL) Upper Extremity Function T-Score
Time frame: Baseline, Week 12
Change From Baseline to Week 12 in the Neuro-QoL Lower Extremity Function T-Score
Time frame: Baseline, Week 12

Results and safety

Results posted to the registry on 11 Oct 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Valbenazine	64	57
Placebo	64	54

Primary outcome: Change From Screening Period Baseline to Maintenance Period in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score.

Time frame: Baseline (average of screening and Day -1), maintenance (average of Weeks 10 and 12). Least squares mean (Standard Error). Unit: units on a scale

Measure	Valbenazine n = 64	Placebo n = 61
Value	-4.60 (0.43)	-1.44 (0.44)

Valbenazine vs Placebo: Least Squares (LS) Means Difference: -3.16 (95% CI -4.37 to -1.95), p <0.0001, MMRM

Population analysed: All participants who are randomly assigned to a treatment group and who had at least 1 evaluable TMC change from baseline score during the 12-week double-blind treatment period.

4 secondary outcomes are reported in the full results.

Adverse events

Time frame: Day 1 (after dosing) through Week 14. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Valbenazine	1 of 64 (1.6%)	35 of 64 (54.7%)	0 of 64
Placebo	2 of 63 (3.2%)	17 of 63 (27%)	1 of 63

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 11 Oct 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 37210099Furr 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.
Derived from this study: PubMed 32250312Rodrigues FB, Wild EJ. Huntington's Disease Clinical Trials Corner: April 2020. J Huntingtons Dis. 2020;9(2):185-197. doi: 10.3233/JHD-200002.
Study websiteLink provided by the sponsor.
Individual participant dataSharing planned: No

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Chief Medical Officer study director

Chief Medical Officer

46 sites in 2 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Valbenazine in people with Chorea, Huntington, run by Neurocrine Biosciences at 46 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 11 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 46 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 11 Oct 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Screening Period Baseline to Maintenance Period in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score. (time frame: Baseline (average of screening and Day -1), maintenance (average of Weeks 10 and 12)). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04102579, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Oct 2023.