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| Name | Class |
|---|---|
| Huntington Study Group | NETWORK |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Valbenazine | Experimental | Capsule, administered orally once daily for 12 weeks. |
|
| Placebo | Placebo Comparator | Capsule, administered orally once daily for 12 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Valbenazine | Drug | vesicular monoamine transporter 2 (VMAT2) inhibitor |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Baseline (average of screening and Day -1), maintenance (average of Weeks 10 and 12) |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Chief Medical Officer | Chief Medical Officer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Neurocrine Clinical Site | Birmingham | Alabama | 35233 | United States | ||
| Neurocrine Clinical Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37210099 | Result | 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. | |
| 32250312 |
| Label | URL |
|---|---|
| Study website | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | Valbenazine | Capsule, administered orally once daily for 12 weeks. |
| FG001 | Placebo | Capsule, administered orally once daily for 12 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 12, 2021 | Sep 8, 2023 |
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| Placebo | Drug | non-active dosage form |
|
| 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. | 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. | 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. | Baseline, Week 12 |
| Little Rock |
| Arkansas |
| 72205 |
| United States |
| Neurocrine Clinical Site | La Jolla | California | 92037 | United States |
| Neurocrine Clinical Site | Sacramento | California | 95817 | United States |
| Neurocrine Clinical Site | Aurora | Colorado | 80045 | United States |
| Neurocrine Clinical Site | Englewood | Colorado | 80113 | United States |
| Neurocrine Clinical Site | Washington D.C. | District of Columbia | 20007 | United States |
| Neurocrine Clinical Site | Gainesville | Florida | 32608 | United States |
| Neurocrine Clinical Site | Miami | Florida | 33136 | United States |
| Neurocrine Clinical Site | Atlanta | Georgia | 30329 | United States |
| Neurocrine Clinical Site | Chicago | Illinois | 60611 | United States |
| Neurocrine Clinical Site | Chicago | Illinois | 60612 | United States |
| Neurocrine Clinical Site | Indianapolis | Indiana | 46202 | United States |
| Neurocrine Clinical Site | Iowa City | Iowa | 52242 | United States |
| Neurocrine Clinical Site | Kansas City | Kansas | 66160 | United States |
| Neurocrine Clinical Site | Wichita | Kansas | 67226 | United States |
| Neurocrine Clinical Site | Louisville | Kentucky | 40202 | United States |
| Neurocrine Clinical Site | New Orleans | Louisiana | 70121 | United States |
| Neurocrine Clinical Site | Boston | Massachusetts | 02118 | United States |
| Neurocrine Clinical Site | Boston | Massachusetts | 02215 | United States |
| Neurocrine Clinical Site | Charlestown | Massachusetts | 02129 | United States |
| Neurocrine Clinical Site | Ann Arbor | Michigan | 48109 | United States |
| Neurocrine Clinical Site | West Bloomfield | Michigan | 48322 | United States |
| Neurocrine Clinical Site | Omaha | Nebraska | 68198 | United States |
| Neurocrine Clinical Site | Rochester | New York | 14618 | United States |
| Neurocrine Clinical Site | Williamsville | New York | 14221 | United States |
| Neurocrine Clinical Site | Durham | North Carolina | 27705 | United States |
| Neurocrine Clinical Site | Fargo | North Dakota | 58103 | United States |
| Neurocrine Clinical Site | Cleveland | Ohio | 44195 | United States |
| Neurocrine Clinical Site | Columbus | Ohio | 43210 | United States |
| Neurocrine Clinical Site | Toledo | Ohio | 43614 | United States |
| Neurocrine Clinical Site | Pittsburgh | Pennsylvania | 15213 | United States |
| Neurocrine Clinical Site | Charleston | South Carolina | 29425 | United States |
| Neurocrine Clinical Site | Columbia | South Carolina | 29203 | United States |
| Neurocrine Clinical Site | Greenville | South Carolina | 29615 | United States |
| Neurocrine Clinical Site | Nashville | Tennessee | 37212 | United States |
| Neurocrine Clinical Site | Houston | Texas | 77054 | United States |
| Neurocrine Clinical Site | Salt Lake City | Utah | 84108 | United States |
| Neurocrine Clinical Site | Burlington | Vermont | 05401 | United States |
| Neurocrine Clinical Site | Charlottesville | Virginia | 22908 | United States |
| Neurocrine Clinical Site | Seattle | Washington | 98195 | United States |
| Neurocrine Clinical Site | Spokane | Washington | 99202 | United States |
| Neurocrine Clinical Site | Vancouver | British Columbia | V6T 2B5 | Canada |
| Neurocrine Clinical Site | Ottawa | Ontario | K1Y 4E9 | Canada |
| Neurocrine Clinical Site | Toronto | Ontario | M2K 1E1 | Canada |
| Neurocrine Clinical Site | Toronto | Ontario | M3B 2S7 | Canada |
| Derived |
| 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. |
| Received at Least 1 Dose of Study Drug |
|
| COMPLETED |
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| NOT COMPLETED |
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All participants who received at least 1 dose of study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | Valbenazine | Capsule, administered orally once daily for 12 weeks. |
| BG001 | Placebo | Capsule, administered orally once daily for 12 weeks. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Region of Enrollment | Number | participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | 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. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline (average of screening and Day -1), maintenance (average of Weeks 10 and 12) |
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| Secondary | 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. | All participants who were randomly assigned to a treatment group, and who had at least 1 evaluable TMC change from baseline score and observed data at Week 12. | Posted | Number | percentage of responders | Week 12 |
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| Secondary | 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. | All participants who were randomly assigned to a treatment group, and who had at least 1 evaluable TMC change from baseline score and observed data at Week 12. | Posted | Number | percentage of responders | Week 12 |
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| Secondary | 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. | 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. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline, Week 12 |
|
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | 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. | 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. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline, Week 12 |
|
|
Day 1 (after dosing) through Week 14
All-cause mortality, serious adverse events, and other adverse events were assessed using the safety analysis population which included all participants who were randomly assigned to a treatment group, received at least 1 dose of study treatment, and had any postbaseline safety data. Treatment-emergent adverse events were those that occurred on or after first dose date of study treatment and up to 14 days after the last dose of study treatment.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Valbenazine | Capsule, administered orally once daily for 12 weeks. | 0 | 64 | 1 | 64 | 35 | 64 |
| EG001 | Placebo | Capsule, administered orally once daily for 12 weeks. | 1 | 63 | 2 | 63 | 17 | 63 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Psychotic disorder | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Colon cancer | Cardiac disorders | MedDRA 24.0 | Systematic Assessment | A serious adverse event of colon cancer in the placebo group resulted in death. |
|
| Angioedema | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fatigue | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Somnolence | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Akathisia | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment | Note: One additional participant in the valbenazine group had an event of urticaria that was coded under the system organ class of immune system disorders. |
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Generally, the PI has the right to publish results provided such publication does not violate confidentiality or IP provisions within the contract with the Sponsor. Prior to submission for publication or presentation of results, the PI must provide the Sponsor time for review. The Sponsor can request the PI to withhold or remove information from all publications. For a multi-center study, any publication of results by the PI shall not be made before the first multi-center publication.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Neurocrine Medical Information Call Center | Neurocrine Biosciences | 877-641-3461 | medinfo@neurocrine.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 19, 2021 | Sep 8, 2023 | SAP_001.pdf |
| ID | Term |
|---|---|
| D006816 | Huntington Disease |
| D002819 | Chorea |
| D009069 | Movement Disorders |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D003704 | Dementia |
| D020820 | Dyskinesias |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D003072 | Cognition Disorders |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| C000603978 | valbenazine |
Not provided
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Not provided
| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| United States |
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