RecruitingPhase 2ACTRN12622000408785

A Clinical Trial to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of PRAX-944 in Essential Tremor - Part B

A Phase 2 Clinical Trial Evaluating the Efficacy, Safety, Tolerability, and Pharmacokinetics of PRAX-944 in Adults with Essential Tremor - Part B

Sponsor

Praxis Precision Medicines

Enrollment

12 participants

Start Date

Aug 5, 2021

Study Type

Interventional

Conditions

Essential Tremor

Summary

This is Part B of a 2-part, Phase 2, Clinical Trial Evaluating the Efficacy, Safety, Tolerability, and Pharmacokinetics of PRAX-944 in Adults with Essential Tremor. Each participant will complete 3 study periods: Screening, Treatment Period (21 or 28 days) and Safety Follow-up.

Eligibility

Sex: Both males and femalesMin Age: 18 Yearss

Plain Language Summary

Simplified for easier understanding

Essential tremor is one of the most common movement disorders, causing uncontrollable shaking — most often of the hands — that can make everyday tasks like writing, eating, and drinking very difficult. Current medications help some people but are not effective enough for many others, leaving them with significant disability. PRAX-944 is a new investigational drug being tested as a potential treatment for essential tremor. This Phase 2 study is the second part of a two-part trial that will evaluate whether PRAX-944 effectively reduces tremor severity, how safe it is, and how it is processed by the body. Participants take the medication for 21–28 days and are closely monitored. You may be eligible if you are 18 or older, have a clinical diagnosis of essential tremor, and are either not taking any tremor medication or are on a single stable tremor medication. People with other neurological conditions causing tremor, those who have had brain surgery or deep brain stimulation for tremor, or those who cannot stop taking primidone are not eligible.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

Open-label Titration Phase - Titration to 120mg PRAX-944 orally according to the following titration schedule: PRAX-944 20mg orally once a day for 3 days, 40mg orally once a day for 4 days, 60mg oral

Open-label Titration Phase - Titration to 120mg PRAX-944 orally according to the following titration schedule: PRAX-944 20mg orally once a day for 3 days, 40mg orally once a day for 4 days, 60mg orally once a day for 7 days, 80mg orally once a day for 7 days, 100mg orally once a day for 7 days, and 120mg orally for 7 days. If at any point during the open-label titration phase a participant does not tolerate escalation (based on AEs and safety assessments) and the Investigator returns the participant to a lower dose level, the participant will continue according to the schedule outlined above, but no further dose changes will be allowed. Once participants complete the open label phase, they will immediately start the double-blind phase. They will be randomly assigned to continue with PRAX-944 or placebo for the double-blind phase. Double-blind Phase: PRAX-944 or Placebo orally once a day for 14 days after the final dose of the open-label phase. Total duration of participation in the trial will be 70 days (56 on treatment plus a 14 day follow-up period). Study drug adherence will be monitored by a compliance application and verified by unused product return.