A Phase 3 interventional study of Valbenazine and Placebo in Chorea, Huntington, sponsored by Neurocrine Biosciences. Completed at 46 sites in 2 countries. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2023-10-11.
Sponsored by Neurocrine Biosciences · Phase 3, Interventional, and Treatment
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.
293 studies on the registry are indexed under Huntington Disease; 54 are open to participants now.
This study's enrollment of 128 is above the median of 40 across 207 interventional studies indexed under Huntington Disease.
Browse Huntington Disease studies →Neurocrine Biosciences is the lead sponsor of 86 studies on the registry; 16 are open to participants now.
Of its 35 completed or terminated interventional studies of FDA-regulated products, 23 (66%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Capsule, administered orally once daily for 12 weeks.
Drug: Valbenazine
Capsule, administered orally once daily for 12 weeks.
Drug: Placebo
vesicular monoamine transporter 2 (VMAT2) inhibitor
Also known as: NBI-98854
non-active dosage form
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.
Time frame: Baseline (average of screening and Day -1), maintenance (average of Weeks 10 and 12)
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.
Time frame: 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.
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
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
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.
Time frame: Baseline, Week 12
| Milestone | Valbenazine | Placebo |
|---|---|---|
| Started | 64 | 64 |
| Received at least 1 dose of study drug | 64 | 63 |
| Completed | 57 | 54 |
| Not completed | 7 | 10 |
| Withdrew: Withdrawal by subject | 3 | 4 |
| Withdrew: Lost to follow-up | 0 | 1 |
| Withdrew: Discontinued due to covid-19-related study pause | 3 | 4 |
| Withdrew: Other than specified | 1 | 0 |
| Withdrew: Not dosed with study drug | 0 | 1 |
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.
| units on a scale | Valbenazine | Placebo |
|---|---|---|
| Change From Screening Period Baseline to Maintenance Period in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score. | -4.60 ± 0.43 | -1.44 ± 0.44 |
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.
| percentage of responders | Valbenazine | Placebo |
|---|---|---|
| Percent of Clinical Global Impression of Change (CGI-C) Responders at Week 12 | 42.9 | 13.2 |
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.
| percentage of responders | Valbenazine | Placebo |
|---|---|---|
| Percent of Patient Global Impression of Change (PGI-C) Responders at Week 12 | 52.7 | 26.4 |
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.
| units on a scale | Valbenazine | Placebo |
|---|---|---|
| Change From Baseline to Week 12 in the Quality of Life in Neurological Disorders (Neuro-QoL) Upper Extremity Function T-Score | -1.58 ± 1.01 | -3.00 ± 1.04 |
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.
| units on a scale | Valbenazine | Placebo |
|---|---|---|
| Change From Baseline to Week 12 in the Neuro-QoL Lower Extremity Function T-Score | -0.27 ± 0.82 | 0.61 ± 0.84 |
Collected over Day 1 (after dosing) through Week 14. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Valbenazine | 0/64 (0%) | 1/64 (1.6%) | 35/64 (54.7%) |
| Placebo | 1/63 (1.6%) | 2/63 (3.2%) | 17/63 (27%) |
| Event | Valbenazine | Placebo |
|---|---|---|
| Psychotic disorderPsychiatric disorders | 0/64 | 1/63 |
| Colon cancerCardiac disorders | 0/64 | 1/63 |
| AngioedemaSkin and subcutaneous tissue disorders | 1/64 | 0/63 |
| Event | Valbenazine | Placebo |
|---|---|---|
| SomnolenceNervous system disorders | 10/64 | 2/63 |
| FatigueGeneral disorders | 9/64 | 6/63 |
| FallInjury, poisoning and procedural complications | 8/64 | 8/63 |
| RashSkin and subcutaneous tissue disorders | 5/64 | 0/63 |
| UrticariaSkin and subcutaneous tissue disorders | 5/64 | 0/63 |
| AkathisiaNervous system disorders | 4/64 | 3/63 |
All participants who received at least 1 dose of study drug.
| Age, Continuous(years) | Valbenazine | Placebo | Total |
|---|---|---|---|
| Mean | 54.1 ± 10.1 | 53.6 ± 11.6 | 53.8 ± 10.8 |
| Sex: Female, Male(Participants) | Valbenazine | Placebo | Total |
|---|---|---|---|
| Female | 33 | 36 | 69 |
| Male | 31 | 27 | 58 |
| Ethnicity (NIH/OMB)(Participants) | Valbenazine | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 5 | 3 | 8 |
| Not Hispanic or Latino | 59 | 60 | 119 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Valbenazine | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 1 | 0 | 1 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 1 | 0 | 1 |
| White | 60 | 62 | 122 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 2 | 1 | 3 |
| Region of Enrollment(participants) | Valbenazine | Placebo | Total |
|---|---|---|---|
| Canada | 2 | 7 | 9 |
| United States | 62 | 56 | 118 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Nov 2019 to primary completion in Oct 2021, 23 months.
Results were first posted in Oct 2023, 1 year 11 months after primary completion. A results delay was requested in Sep 2022.
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Studies of the drug tested here: Valbenazine