Abstract Details

Title PRAX-944-222: A Phase 2 Randomized, Double-blind, Placebo-controlled Trial of PRAX-944 for the Treatment of Essential Tremor

Topic Movement Disorders

Presentation(s) P1 - Poster Session 1 (9:00 AM-5:00 PM)

Poster/Presentation
Number 257

Objective The PRAX-944-222 trial will evaluate the tolerability, safety and efficacy of PRAX-944, a novel selective T-type calcium channel blocker, for treatment of essential tremor (ET).

Background

ET is the most common movement disorder with clear need for novel therapies. Approximately 50% of patients seeking pharmacological therapy discontinue medications due to limited efficacy and poor tolerability. We previously demonstrated tolerability of pharmacodynamically-active doses of PRAX-944, up to 120 mg, in healthy participants (ACTRN12620000675921), as well as preliminary evidence of tremor reduction following 20-40 mg PRAX-944 in adults with ET (NCT05021978). The current PRAX-944-222 trial (NCT05021991) further explores the safety and efficacy of 20, 60 and 120 mg once-daily (QD) PRAX-944 in adults with ET.

Design/Methods

This 8-week, double-blind, placebo-controlled, randomized, parallel-group study will recruit 112 adults with moderate-to-severe ET defined by upper limb tremor quantified with The Essential Tremor Rating Assessment Scale (TETRAS-UL, score ≥10). Eligible participants must be off ET medications or stable on their medication for 1 month prior to screening. Participants will be randomized (1:1:1:1) to titrated doses of 20, 60, or 120 mg PRAX-944 QD or placebo.

Results The primary endpoint will be incidence and severity of adverse events. Secondary efficacy endpoints will include baseline to week 8 change in the TETRAS-performance and activities of daily living (ADL) subscales, wearable accelerometry measures and an ET performance-based scale combining clinician scoring of participants as they conduct ADL-related tasks. This trial will inform power calculations and dose selection for later phase trials. Enrolment will commence in late-2021.

Conclusions Expanding on preliminary findings, the PRAX-944-222 trial will further examine the safety, tolerability and efficacy of PRAX-944 in adults with ET, and will determine optimal doses and endpoints for evaluation in later phase studies. Importantly, the unique trial design seeks to address the sub-optimal benefit-risk profile of current therapies and associated unmet needs in ET.

Authors/Disclosures
Bernard M. Ravina, MD, MSCE (Voyager Therapeutics, Inc.) PRESENTER	Dr. Ravina has received personal compensation for serving as an employee of Praxis Precision Medicines. Dr. Ravina has stock in Praxis.
Gabriel M. Belfort, MD (BlueRock Therapeutics)	Dr. Belfort has received personal compensation for serving as an employee of Praxis Precision Medicines. Dr. Belfort has received stock or an ownership interest from Praxis Precision Medicines. Dr. Belfort has received intellectual property interests from a discovery or technology relating to health care.
Shane Raines	Shane Raines has received personal compensation for serving as an employee of 2b Analytics, LLC.
	No disclosure on file

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