Characterization of essential tremor (ET) experience and unmet need in patients seeking participation in the Essential3 Phase 3 Program.

Despite affecting up to 7 million people in the US, ET is often underrecognized and inadequately treated by conventional medications, which are often ineffective against tremor or poorly tolerated. Ulixacaltamide is a novel, selective T-type calcium channel blocker in development for ET treatment. Essential3 is an ongoing, innovative, decentralized multi-study combining in-home and telehealth visits to assess efficacy and safety of ulixacaltamide in adults with ET.

Preliminary eligibility to participate in Essential3 was assessed via an online pre-screener capturing demographics, symptoms, and other clinical features. Assessment of qualifiers was based on (though not limited to): receiving an ET diagnosis; bilateral arm/hand tremor; symptoms >3 years; sufficient self-reported action tremor severity with higher likelihood of meeting inclusion criteria. Data are presented for 75,000 survey responders as of 08/2024.

Responders were of median age 71 (18-101) years. Of those with an ET diagnosis, 58% indicated being diagnosed/treated by a neurologist. Most (70%) were diagnosed in the last 10 years. Between 71%-87% reported difficulty completing daily tasks e.g. pouring, drinking from a glass, picking up/reaching for small items, using a smartphone. Conversely, 12%-29% indicated no problem completing tasks. Most had not received ultrasound/surgery, Cala Trio, or botulinum toxin intervention. Over 85% reported willingness to allow home study visits, with 91% willing to use a mobile app and video calls if deemed eligible to participate.

Findings from the largest ET survey to date show that patients seeking participation in a clinical trial report ET as a high-burden, inadequately managed disorder impacting several activities of daily living. Findings also suggest a degree of normalization of ET impact (likely necessitating ongoing patient and physician education) and emphasize the urgent need for innovation in developing effective, well-tolerated treatments.