NCT06087276

Essential 3 - Decentralized, Phase 3 Study Evaluating the Safety and Efficacy of Ulixacaltamide in Essential Tremor (ET)

Phase 3, Decentralized, Randomized, Double-Blind, Placebo Controlled, Parallel Design, Randomized Withdrawal, and Long-term Safety Study to Evaluate the Efficacy and Safety of Ulixacaltamide (PRAX-944) in Adults With Essential Tremor

• Status: Enrolling By Invitation
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 1,000 (estimated)
• Sponsor: Praxis Precision Medicines · Industry
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

The goal of this clinical study is to compare ulixacaltamide and placebo treatment in essential tremor. The main question it aims to answer is: • Is ulixacaltamide a safe and efficacious treatment for patients with essential tremor? Participants will be asked to participate in one of two clinical studies where they will be treated with either ulixacaltamide or placebo for a period of up to 12 weeks. After the controlled study completion, they will be eligible to participate in a long-term, open-label safety study (LTSS) and be treated with ulixacaltamide. Participants are eligible to enroll directly into the LTSS if they previously participated in an essential tremor trial, received sponsor invitation after being deemed ineligible for the controlled study, or following enrollment closure of the controlled study.

Detailed description

PRAX-944-321 is a decentralized, Phase 3, multi-study, clinical trial evaluating the safety and efficacy of ulixacaltamide in essential tremor (ET). The study includes 3 separate and simultaneous phase 3 pivotal studies where all participants undergo one screening process. The first study is a parallel design (PD) enrolling approximately 400 patients, the second is a randomized withdrawal (RW) enrolling approximately 200 patients and the last is a long-term safety study (LTSS) comprising up to 1000 patients. A total of 4 key efficacy hypotheses are being tested prospectively in the study. The analysis will be conducted simultaneously, with unblinding only occurring once. Hypotheses - 1) how do patients compare between ulixacaltamide and placebo after 56 days of intervention in the PD study, 2) for patients exposed to ulixacaltamide in the RW study who improved by at least 3 points in the mADL11 scale, which proportion maintains response after randomization staying on ulixacaltamide compared to placebo after re-randomization at day 56, 3) how does the pooled group of patients receiving ulixacaltamide in both studies (PD and RW) compare to placebo patients from the parallel design study after 56 days of intervention, 4) how do patients receiving ulixacaltamide in the RW study compare to placebo patients from the PD study after 56 days of intervention.

Conditions

• Essential Tremor

Interventions

Type	Name	Description
DRUG	60 mg ulixacaltamide	Once daily oral treatment with titration
DRUG	Placebo	Once daily oral treatment

Timeline

• Start date: 2023-11-02
• Primary completion: 2025-09-01
• Completion: 2026-12-01
• First posted: 2023-10-17
• Last updated: 2025-09-30

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study