PRAX-944 for Essential Tremor

(Essential1 Trial)

Recruiting at 36 trial locations

Overseen By Head of Pharmacovigilance, Praxis Precision Medicines

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Praxis Precision Medicines

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

133 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing PRAX 944, a new medication, to see if it can help people with Essential Tremor, a condition that causes uncontrollable shaking. The study includes adults who have had this condition for a significant period. Researchers want to find out if PRAX 944 can reduce the symptoms by affecting certain brain pathways involved in movement.

Research Team

Director, Clinical Development

Principal Investigator

Praxis Precision Medicines

Eligibility Criteria

Inclusion Criteria

If currently receiving any medication for ET, is on a stable dose of any of these medications for ET for 1 month 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.

Your body mass index (BMI) is between 18 and 40.

Your body mass index (BMI) should be between 18 and 40 kg/m2.

Exclusion Criteria

You have a history of specific medical, neurological, or psychiatric conditions that can cause tremors, such as Parkinson's disease, Alzheimer's disease, or a family history of Fragile X syndrome.

You are not using certain types of medication that could affect the evaluation of tremors.

You have had a problem with drugs or alcohol in the past, but you have been doing well for at least 2 years.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral dosing of PRAX-944 with titration over 56 days

8 weeks

Multiple visits for titration and monitoring

Extension Double-blind Lead-in

Participants receive double-blind oral dosing of PRAX-944 for 43 days

6 weeks

Extension Open-label

Participants receive open-label flexible dosing of PRAX-944 for 469 days

67 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• PRAX-944

Participant Groups

Group I: Regimen 2 Experimental Treatment 4 Interventions

Double-blind Part: Oral dosing, once daily in the morning with titration over 56 days to 60 mg PRAX-944: 7 days of 5 mg, 7 days of 10 mg, 7 days of 20 mg, 7 days of 40 mg, 28 days of 60 mg Extension Part: Double-blind Lead-in: Oral dosing, once daily in the morning with titration over 43 days to 100 mg PRAX-944: 7 days of 80 mg, 36 days of 100 mg Extension Part: Open-label Flexible PRAX-944 Dosing: Oral dosing, once daily in the morning of 20 mg to 100 mg PRAX-944 for 469 days Crossover Part: Following double-blind lead-in/open-label: 1:1 randomization to placebo or stable dose of PRAX-944 for 21 days followed by cross-over to either placebo or PRAX-944 oral dosing, once daily in the morning with titration over 7 days (3 days at 20 mg, 4 days at 40 mg) to 60 mg (14 days) before returning to open-label part.

Group II: Regimen 1 Experimental Treatment 4 Interventions

Double-blind Part: Oral dosing, once daily in the morning with titration over 56 days to 100 mg PRAX-944: 7 days of 5 mg, 7 days of 10 mg, 7 days of 20 mg, 7 days of 40 mg, 7 days of 60 mg, 7 days of 80 mg, 14 days of 100 mg Extension Part: Double-blind Lead-in: Oral dosing, once daily in the morning over 43 days: 100 mg PRAX-944 Extension Part: Open-label Flexible PRAX-944 Dosing: Oral dosing, once daily in the morning of 20 mg to 100 mg PRAX-944 for 469 days Crossover Part: Following double-blind lead-in/open-label: 1:1 randomization to placebo or stable dose of PRAX-944 for 21 days followed by cross-over to either placebo or PRAX-944 oral dosing, once daily in the morning with titration over 7 days (3 days at 20 mg, 4 days at 40 mg) to 60 mg (14 days) before returning to open-label part.

Group III: Regimen 3 Placebo Group 4 Interventions

Double-blind Part: Oral dosing, once daily in the morning: 56 days of placebo Extension Part: Double-blind Lead-in: Oral dosing, once daily in the morning with titration over 43 days to 100 mg PRAX-944: 7 days of 5 mg, 7 days of 10 mg, 7 days of 20 mg, 7 days of 40 mg, 7 days of 60 mg, 7 days of 80 mg, 14 days of 100 mg Extension Part: Open-label Flexible PRAX-944 Dosing: Oral dosing, once daily in the morning of 20 mg to 100 mg PRAX-944 for 469 days Crossover Part: Following double-blind lead-in/open-label: 1:1 randomization to placebo or stable dose of PRAX-944 for 21 days followed by cross-over to either placebo or PRAX-944 oral dosing, once daily in the morning with titration over 7 days (3 days at 20 mg, 4 days at 40 mg) to 60 mg (14 days) before returning to open-label part.

Who Is Running the Clinical Trial?

Praxis Precision Medicines

Lead Sponsor

Trials

11

Recruited

1,300+