Objective: To describe the design and baseline characteristics of a real-world study evaluating the safety and effectiveness of deutetrabenazine (DTBZ) in South Korean patients with HD-associated chorea.

Background: HD is an autosomal dominant neurodegenerative disorder characterized by progressive motor, cognitive, and behavioral impairment, with chorea as the predominant motor manifestation [1]. DTBZ, a vesicular monoamine transporter 2 inhibitor, has demonstrated efficacy and safety in patients with HD-associated chorea [2,3]; however, patients from South Korea were not included.

Method: This ongoing Phase 4 observational study with prospective and retrospective components includes adults aged ≥18 years in South Korea with HD-associated chorea receiving DTBZ per local prescribing information. Participants initiating DTBZ undergo baseline assessment (Visit 1), including demographics, clinical characteristics, and total maximal chorea (TMC) score, with safety evaluation and repeat TMC scoring ≥4 weeks after reaching optimal dose (Visit 2). For those on DTBZ at enrollment, treatment history and safety data are collected retrospectively, with prospective safety follow-up as applicable. The primary endpoint is the number and proportion of participants with serious and non-serious adverse events and adverse drug reactions during the study period; exposure-adjusted incidence rates will also be calculated. The secondary endpoint is change in TMC score between Visits 1–2 in those initiating DTBZ.

Results: This analysis included 32 participants (mean [SD] age, 57.8 [13.1] years; 25.0% male) with a mean (SD) disease duration of 3.5 (3.0) years, CAG repeat length of 43.1 (4.0), and body mass index of 21.4 (3.0) kg/m². Overall, 43.8% had ≥1 concomitant medical condition, most commonly psychiatric (21.9%), metabolic (18.8%), neurologic (18.8%), and vascular disorders (15.6%). Prior medication use (≤6 months before first dose) was reported in 34.4% and consisted exclusively of nervous system agents. Concomitant medication use was reported in 46.9%, most commonly nervous system drugs (37.5%) including antiepileptics (28.1%), antidepressants (25.0%), and antipsychotics (15.6%).

Conclusion: This study will address an unmet need for real-world data on DTBZ safety and effectiveness in South Korean patients with HD-associated chorea.

References: 1. McColgan P, Tabrizi SJ. Huntington’s disease: A clinical review. Eur J Neurol. 2018;25(1):24–34.
2. Frank S, et al. Effect of deutetrabenazine on chorea among patients with Huntington disease: A randomized clinical trial. JAMA. 2016;316(1):40–50.
3. Frank S, et al. The safety of deutetrabenazine for chorea in Huntington Disease: An open-label extension study. CNS Drugs. 2022;36(11):1207–1216.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/real-world-evaluation-of-deutetrabenazine-for-huntington-disease-associated-chorea-in-south-korea-study-design-and-baseline-characteristics/