A Clinical Trial of PRAX-944 in Participants With Essential Tremor

NCT ID: NCT05021978

Last Updated: 2024-03-01

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-03-03
• Study Completion Date: 2022-03-24

Brief Summary

This is a 2-part clinical trial to evaluate the efficacy, safety, and tolerability of 40 and 120 mg oral PRAX-944 compared to placebo in the treatment of adults with essential tremor. Part A is designed to study the dose titration from 20 to 40 mg every morning (QAM) (ie, 2 weeks with 7 days at each dose level). Part B is designed to study the dose titration from 20 to up to 120 mg QAM with at least 14 days of dosing at the highest tolerated dose for each participant. Blood levels of PRAX-944 will also be measured throughout the trial and pharmacokinetics will be evaluated.

Conditions

Essential Tremor

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Part A: Open-label 20 and 40 mg PRAX-944

Once daily, oral dosing with 7 days of 20 mg and 7 days of 40 mg

Group Type: EXPERIMENTAL

Part A: 20 and 40 mg PRAX-944

Intervention Type: DRUG

Once daily oral treatment

Part B: Open-label titration PRAX-944 (120 mg) followed by blinded PRAX-944

Once daily, oral dosing with titration to 120 mg: 3 days of 20 mg, 4 days of 40 mg, 7 days of 60 mg, 7 days of 80 mg, 7 days of 100 mg, 28 days of 120 mg

Group Type: EXPERIMENTAL

Part B: 120 mg PRAX-944

Intervention Type: DRUG

Once daily oral treatment with titration

Part B: Open-label titration PRAX-944 (120 mg) followed by blinded placebo

Once daily, oral dosing with titration to 120 mg: 3 days of 20 mg, 4 days of 40 mg, 7 days of 60 mg, 7 days of 80 mg, 7 days of 100 mg, 14 days of 120 mg, 14 days placebo

Group Type: ACTIVE_COMPARATOR

Part B: 120 mg PRAX-944 and Placebo

Intervention Type: DRUG

Once daily oral treatment with titration followed by placebo

Interventions

Part A: 20 and 40 mg PRAX-944

Once daily oral treatment

Intervention Type: DRUG

Part B: 120 mg PRAX-944

Once daily oral treatment with titration

Intervention Type: DRUG

Part B: 120 mg PRAX-944 and Placebo

Once daily oral treatment with titration followed by placebo

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Clinical diagnosis of ET consistent with Movement Disorders Society Criteria, a duration of ET of at least 3 years and with onset before the age of 65

2. TETRAS upper limb score (ie, sum of bilateral upper limb items 4a, 4b, and 4c) of ≥10 as rated by the Investigator at Screening and Baseline OR A score of ≥2 on 2 or more of the following TETRAS activities of daily living (ADL) subscale items: (2) Feeding with a spoon, (3) Drinking from a glass, (5) Dressing, (6) Pouring, (9) Writing OR A score of ≥2 on 1 of the following TETRAS ADL subscale items: (2) Feeding with a spoon, (3) Drinking from a glass, (5) Dressing, (6) Pouring, or (9) Writing AND a score of ≥2 on both of the following TETRAS ADL subscale items: (10) Working and (12) Social Impact

3. If currently receiving any medication for ET, is on a stable dose of any of these medications for ET for 28 days prior to Screening and is willing to maintain stable doses throughout the trial. If receiving primidone for ET, is willing and able to discontinue 14 days prior to Day 1.

4. Body mass index (BMI) between 18 and 40 kg/m2 (inclusive).

Exclusion Criteria

1. Sporadically using a benzodiazepine, sleep medication, or anxiolytic that would confound the assessment of tremor.

2. Trauma to the nervous system within 3 months preceding the onset of tremor.

3. History of other medical, neurological or psychiatric condition that may explain or cause tremor, including but not limited to Parkinson's disease, dystonia, cerebellar disease, family history of Fragile X syndrome, traumatic brain injury, alcohol abuse or withdrawal, benzodiazepine abuse or withdrawal, multiple sclerosis, polyneuropathy, and endocrine states such as hyperthyroidism.

4. Prior magnetic resonance-guided focused ultrasound or surgical intervention for ET such as deep brain stimulation or thalamotomy.

5. Botulinum toxin injection for ET in the 6 months prior to Screening.

6. Unwilling or unable to refrain from alcohol 24 hours before and during clinical trial visits.

7. History of substance use disorder

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Praxis Precision Medicines

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

VP, Clinical Development

Role: STUDY_DIRECTOR

Praxis Precision Medicines

Locations

Praxis Research Site

Port Charlotte, Florida, United States

Praxis Research Site

Spokane, Washington, United States

Praxis Research Site

New Lambton Heights, New South Whales, Australia

Praxis Research Site

Southport, Queensland, Australia

Praxis Research Site

Fitzroy, Victoria, Australia

Praxis Research Site

Melbourne, Victoria, Australia

Praxis Research Site

Parkville, Victoria, Australia

Praxis Research Site

Grafton, Auckland, New Zealand

Praxis Research Site

Christchurch, , New Zealand

Countries

United States; Australia; New Zealand

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

PRAX-944-221

Identifier Type: -

Identifier Source: org_study_id