Objective: To assess the real-world utilization of deutetrabenazine (DTBZ) for the treatment of tardive dyskinesia (TD) or chorea associated with Huntington disease (HD) initiated by a 4-week patient titration kit.

Background: DTBZ is a vesicular monoamine transporter type 2 inhibitor. Separate randomized, double-blind, placebo-controlled pivotal studies established efficacy and safety of DTBZ for the treatment of TD or chorea associated with HD. The optimal dose of DTBZ is determined individually for each patient based on efficacy and tolerability. However, titration observed in real-world prescription data is often inconsistent with labeling and suggests patients may not reach optimal doses needed for treatment success.

Method: The START study is a 15-month, prospective, non-interventional, single-arm, 2-cohort (TD and HD) study. Participants will be provided a newly introduced 4-week DBTZ patient titration kit. Participants with TD will start at DTBZ 6 mg twice daily and participants with HD will start at DTBZ 6 mg once daily. Increases will occur weekly for 4 weeks up to daily doses of 30 mg (TD) and 24 mg (HD). Further titration may occur based on efficacy and tolerability. The suggested visit interval is every 4 weeks for a total of 7 visits. Assessments included in the study are the Clinical Global Impression of Change (CGIC), and either Abnormal Involuntary Movement Scale for TD participants or Total Maximal Chorea score for HD participants. Outcomes relevant to care include quality of life, real-world effectiveness, participant satisfaction with the titration kit, and observed safety, including reports of depression and suicidality.

Results: The primary endpoint is the proportion of participants who achieve treatment success at week 12, defined as improvement on CGIC. Secondary endpoints include treatment success measured by the Patient Global Impression of Change and the evaluation of dosing and treatment patterns with DTBZ in the real world. Exploratory endpoints will describe the changes in the Unified Huntington’s Disease Rating Scale–Motor Component (Total Motor Score), Hospital Anxiety and Depression Scale, and Columbia–Suicidality Severity Rating Scale.

Conclusion: This study will generate critical real-world data on the utilization of a 4-week DTBZ titration kit for treatment of TD or chorea associated with HD.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/the-start-study-a-phase-4-study-on-the-real-world-utilization-of-deutetrabenazine-initiated-by-a-4-week-patient-titration-kit/