PRAX-628 for Partial Seizures

(POWER1 Trial)

Recruiting at 21 trial locations

Overseen By Head of Pharmacovigilance

Age: 18+

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Praxis Precision Medicines

Disqualifiers: Schizophrenia, Cardiac disease, Malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Compensation: Varies

Number of Visits: 1

Duration: 12 weeks

230 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called PRAX-628 to determine its effectiveness in managing focal seizures, a type of partial seizure where abnormal electrical activity affects one part of the brain. Participants will receive either the PRAX-628 medication or a placebo (a pill with no active drug) to compare the treatment's effects over 12 weeks. The trial aims to evaluate the safety and effectiveness of PRAX-628 in reducing seizures. Individuals with focal onset epilepsy, confirmed by a doctor and imaging tests like CT or MRI, may be suitable candidates. As a Phase 2, Phase 3 trial, this research measures the treatment's effectiveness in a larger group and represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking advancements in epilepsy treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that PRAX-628 is likely to be safe for humans?

Research has shown that PRAX-628 is generally well-tolerated. In an earlier study, researchers tested PRAX-628 at different doses and observed positive safety results. Most participants did not experience serious side effects, suggesting the treatment is safe for many users. However, like any medication, some risk of side effects exists. Researchers will closely monitor trial participants for any adverse events. Always consult a healthcare provider to understand the potential risks and benefits. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for seizures?

PRAX-628 is unique because it offers a new approach to treating partial seizures by potentially targeting specific pathways in the brain involved in seizure activity. Unlike many current treatments that primarily focus on general seizure suppression, PRAX-628 may provide a more targeted action, which could lead to improved efficacy and reduced side effects. Researchers are excited about PRAX-628 because it holds the promise of enhanced seizure control with possibly fewer adverse effects, offering a significant advancement over traditional antiepileptic drugs.

What evidence suggests that PRAX-628 might be an effective treatment for partial seizures?

Research has shown that PRAX-628 may effectively treat focal seizures. In one study, all patients taking a 45 mg dose experienced a complete cessation of seizures. Among those taking a 15 mg dose, 80% achieved the same result. PRAX-628 also reduced the number of seizures in animal studies. In this trial, participants will receive either PRAX-628 or a placebo. These findings suggest that PRAX-628 could be a promising option for reducing or stopping partial seizures in patients. ^{1 3 4 6 7}

Who Is on the Research Team?

Medical Director

Principal Investigator

Praxis Precision Medicines

Are You a Good Fit for This Trial?

Adults with focal onset epilepsy, confirmed by CT or MRI to not have a progressive cause, can join this trial. They must not be pregnant, planning pregnancy soon, or breastfeeding. Participants should not have HIV, hepatitis B/C, serious mental health disorders, recent severe seizures requiring hospitalization, or a history of certain malignancies.

Inclusion Criteria

Past evidence by computed tomography (CT) or magnetic resonance imaging (MRI) that has ruled out a progressive cause of epilepsy in the judgement of the investigator and/or in consultation with the medical monitor

I have been diagnosed with focal onset epilepsy.

Exclusion Criteria

Has a positive test result or a known history of a positive test result for human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), or anti-hepatitis C virus (HCV) antibody at Screening

Is pregnant or is breastfeeding at the time of Screening or has a positive serum pregnancy test at Screening or is planning to become pregnant during the clinical trial or within 14 days of the last study drug dose

I am scheduled for epilepsy surgery during the trial.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 20 mg of PRAX-628 or placebo for 6 weeks, followed by 30 mg of PRAX-628 or placebo for the remaining 6 weeks

12 weeks

Visit 1 (Day 1) and subsequent visits as per protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• PRAX-628

Trial Overview The POWER1 trial is testing PRAX-628's effectiveness and safety in adults with focal seizures. Initially given at 20mg/day for six weeks followed by an increased dose of 30mg/day for another six weeks versus placebo.

How Is the Trial Designed?

Group I: Randomized, Double-Blind Placebo Experimental Treatment 1 Intervention

Participants who meet all eligibility criteria will be randomized at Visit 1 (Day 1) to receive a matching placebo throughout the 12-week Treatment Period

Group II: Randomized, Double-Blind 20mg/day for 6 weeks then 30mg/day for remaining 6 weeks PRAX-628 Experimental Treatment 1 Intervention

Participants who meet all eligibility criteria will be randomized at Visit 1 (Day 1) to receive 20 mg of PRAX-628 for 6 weeks followed by 30 mg PRAX 628 for the remaining 6 weeks.

Who Is Running the Clinical Trial?

Praxis Precision Medicines

Lead Sponsor

Trials

11

Recruited

1,300+

Citations

1. investors.praxismedicines.com investors.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-reports-positive-results-prax-628

Praxis Precision Medicines Reports Positive Results of PRAX ... In the 45 mg cohort, 100% of patients achieved a complete response. In the 15 mg cohort, 80% of patients achieved a complete response and ...

2. aesnet.org aesnet.org/abstractslisting/prax-628-is-a-next-generation-functionally-selective-small-molecule-with-potent-anti-seizure-activity-and-potential-as-best-in-class-treatment-for-focal-epilepsy

prax-628 is a next generation functionally selective small ... PRAX-628 also significantly reduced incidence of psychomotor seizures induced by 6 Hz (ED50 1.9 mg/kg) and clonic seizures induced by PTZ (ED50 ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT06999902

Double-blind, Randomized Trial of PRAX-628 in Adults ... Proportion of participants experiencing a ≥50% reduction in monthly (28 days) focal seizure frequency from the Screening/Observation Period to the Treatment ...

4. neurologylive.com neurologylive.com/view/high-dose-prax-628-achieves-complete-response-phase-2a-study-photo-paroxysmal-response

High Dose PRAX-628 Achieves Complete Response in ... In a small-scale study, treatment with PRAX-628 resulted in significant reduction in the number of generalized photo paroxysmal response events.

5. cureepilepsy.org cureepilepsy.org/news/praxis-precision-medicines-reports-positive-results-of-prax-628-study-evaluating-photo-paroxysmal-response-ppr-achieving-100-response-in-treated-patients/

Praxis Precision Medicines Reports Positive Results of ... In the study, patients were treated with 15mg or 45mg of PRAX-628. Following treatment with PRAX-628, there was a significant reduction in the ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT06908356

Study Details | NCT06908356 | An Open Label Trial to ... An Open Label Trial to Evaluate the Efficacy and Safety of PRAX-628 in Adults With Focal Onset or Tonic-Clonic Seizures. ClinicalTrials.gov ID NCT06908356.

7. clinicaltrials.eu clinicaltrials.eu/inn/prax-628/

PRAX-628: A Promising New Treatment for Epilepsy These trials aim to evaluate the safety, efficacy, and pharmacokinetics of PRAX-628 in adults with various forms of epilepsy, including those with focal ...