Chorea, Huntington's Disease
Conditions
Brief summary
The Primary Objective: To evaluate the real-world effectiveness of deutetrabenazine in adult participants with chorea associated with Huntington's disease in China. The Secondary Objectives: To evaluate the real-world safety of deutetrabenazine in adult participants with chorea associated with Huntington's disease in China.
Interventions
DRUGDeutetrabenazine
deutetrabenazine tablets
Sponsors
Teva Branded Pharmaceutical Products R&D LLC
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participants with clinically confirmed diagnosis of chorea associated with Huntington's Disease (HD) * Participants whose baseline total maximal chorea (TMC) score ≥ 8 * Participants who are deutetrabenazine-naïve before study entry or who did not receive deutetrabenazine within 30 days of study entry, and who are about to be treated with deutetrabenazine for chorea associated with HD * Participants who have provided written consent for the use of personal and medical information for study purposes
Exclusion criteria
* Participants who have an unstable or serious medical or psychiatric illness at baseline * Participants with any history of suicidality, untreated or inadequately treated depression * Participants with certain comorbidities, including hepatic impairment, congenital long QT syndrome, and clinically significant cardiac arrhythmias. * Participants who received reserpine within 20 days of deutetrabenazine treatment initiation * Participants who received monoamine oxidase inhibitors within 14 days of deutetrabenazine treatment initiation * Participants who received vesicular monoamine transporter 2 (VMAT2) inhibitors, e.g., tetrabenazine or valbenazine, within 30 days of deutetrabenazine treatment initiation * Participants unable to provide a written consent for the study. * Participants who are participating in another study that includes treatment with an investigational drug and/or intervention at the same time as enrolment in the current study
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in TMC Score in Participants Receiving ≥24 mg/Day | Baseline, Week 16 | The TMC score was determined from Item 12 of the motor assessment (Part 1) of the UHDRS and quantifies chorea (range, 0-28, lower score indicated less chorea). It was the sum of maximal chorea scores for 7 body regions (face, buccal-oral-lingual, trunk, and 4 extremities), each of which was scored on a scale from 0 to 4 (0, absent; 1, slight or intermittent; 2, mild and common or moderate and intermittent; 3, moderate and common; and 4, marked and prolonged). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Any Adverse Events (AEs) By Severity Grade | Baseline up to Week 16 | AE: any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of AEs recorded as one of following Common Terminology Criteria for Adverse Events (CTCAE) criteria: -Grade 1: Mild; asymptomatic or mild symptoms; clinical/diagnostic observations only; intervention not indicated. -Grade 2: Moderate; local or noninvasive intervention indicated; limiting age-appropriate instrumental Activities of Daily Living (ADL) (for example, preparing meals, shopping for groceries or clothes, using telephone). -Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization; disabling; limiting self-care ADL (bathing, dressing, feeding self, using toilet, taking medications, and not bedridden). -Grade 4: Life-threatening; urgent intervention indicated. -Grade 5: Death related to AE. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section. |
| Number of Participants With Serious Adverse Events (SAEs) | Baseline up to Week 16 | An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. The SAEs were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in the Reported AE section. |
| Number of Participants With Treatment-related AEs | Baseline up to Week 16 | An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Number of participants with treatment-related AEs is reported in this outcome measure. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in the Reported AE section. |
| Number of Participants With AEs Leading to Dose Reduction, Interruption, Treatment Discontinuation, and Study Withdrawal | Baseline up to Week 16 | An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Number of participants with AEs leading to dose reduction, interruption, treatment discontinuation, and study withdrawal is reported in this outcome measure. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in the Reported AE section. |
| Number of Participants With AEs in Titration Phase | Baseline up to Week 16 | An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs lasted from titration phase to maintenance phase were considered an AE that occurred in the titration phase. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in the Reported AE section. |
| Number of Participants With AEs in Maintenance Phase | Baseline up to Week 16 | An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in the Reported AE section. |
| Number of Participants With AEs of Special Interest | Baseline up to Week 16 | An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs of special interest included suicidality, depression, somnolence, QTc prolongation, akathisia, and parkinsonism. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in the Reported AE section. |
| Change From Baseline in TMC Score in All Participants Regardless of Study Drug Dose | Baseline, Week 16 | The TMC score was determined from Item 12 of the motor assessment (Part 1) of the UHDRS and quantifies chorea (range, 0-28, lower score indicated less chorea). It was the sum of maximal chorea scores for 7 body regions (face, buccal-oral-lingual, trunk, and 4 extremities), each of which was scored on a scale from 0 to 4 (0, absent; 1, slight or intermittent; 2, mild and common or moderate and intermittent; 3, moderate and common; and 4, marked and prolonged). |
Countries
China
Contacts
STUDY_DIRECTORTeva Medical Expert, MD
Teva Branded Pharmaceutical Products R&D LLC
Participant flow
Pre-assignment details
A total of 50 participants were screened and enrolled in the study. Per prespecified analysis, data were collected for single arm regardless of the dose received.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 48.9 years STANDARD_DEVIATION 10.8 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 50 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 0 Participants |
| Sex: Female, Male Female | 25 Participants |
| Sex: Female, Male Male | 25 Participants |
| Total Maximal Chorea (TMC) Score in Participants Who Received Deutetrabenazine Treatment ≥24 mg/day | 12.8 score on a scale STANDARD_DEVIATION 4.3 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 50 |
| other Total, other adverse events | 20 / 50 |
| serious Total, serious adverse events | 2 / 50 |
Outcome results
None listed