PRAX-628 for Epilepsy

Recruiting at 10 trial locations

Overseen By Head of Pharmacovigilance

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Praxis Precision Medicines

Must be taking: ASMs

Must not be taking: Vigabatrin, Felbamate

Disqualifiers: Schizophrenia, OCD, Cardiac disease, 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: Unknown

Duration: 8 weeks

50 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, a potential anti-seizure medication, for people with specific types of epilepsy. It focuses on evaluating the treatment's effectiveness and safety. The study targets individuals who experience focal onset seizures (seizures starting in one part of the brain) or primary generalized tonic-clonic seizures (seizures affecting the whole brain) and have frequent episodes despite treatment with up to three anti-seizure medications. Participants will take 30mg of PRAX-628 daily for 8 weeks. Those with frequent seizures as described and stable medication doses might find this trial suitable. As a Phase 2 trial, it measures PRAX-628's effectiveness in an initial, smaller group, offering a chance to contribute to important research.

Will I have to stop taking my current medications?

The trial does not specify that you need to stop taking your current medications. In fact, you must be on stable doses of 1 to 3 allowable anti-seizure medications (ASMs) to participate.

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

Research has shown that PRAX-628 was well-tolerated in earlier studies. In one study, researchers safely administered it to healthy individuals at doses more than 15 times higher than the expected regular use. This indicates a strong safety profile. Another study found that high doses of PRAX-628 significantly improved seizure-related events without causing serious side effects. Overall, these findings suggest that PRAX-628 is generally safe for humans. ^{1 2 3 4 5}

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

Researchers are excited about PRAX-628 for epilepsy because it offers a fresh approach to managing seizures. Unlike standard treatments like valproate and lamotrigine, which often work by stabilizing electrical activity in the brain, PRAX-628 targets specific neuronal pathways with a novel mechanism aimed at reducing hyperexcitability more precisely. This targeted action could potentially lead to fewer side effects and improved seizure control, making it a promising option for patients who haven't found relief with current medications.

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

Research has shown that PRAX-628, the investigational treatment in this trial, may help treat seizures. In earlier studies, patients who took PRAX-628 experienced a noticeable drop in seizure activity. One study even found that it completely stopped certain brain reactions to light that can cause seizures. PRAX-628 has also reduced various types of seizures, such as those affecting movement and muscle control. These results suggest that PRAX-628 could be a strong treatment option for people with specific types of seizures, like focal onset or primary generalized tonic-clonic seizures. ^{1 2 3 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 or tonic-clonic seizures who have had at least 2 seizures per month and are on stable epilepsy medication can join. Those pregnant, planning pregnancy, with serious mental health issues, recent neurosurgery, cardiac problems, cancer history within 5 years, or recent experimental therapy use cannot participate.

Inclusion Criteria

Evidence by computed tomography (CT) or magnetic resonance imaging (MRI) ruling out a progressive cause of epilepsy

I have been diagnosed with focal onset or generalized tonic-clonic seizures.

I am on 1 to 3 stable seizure medications.

Exclusion Criteria

Pregnant or breastfeeding at Screening, positive serum pregnancy test at Screening, or planning pregnancy during the trial or within 14 days of the last study drug dose

Schizophrenia, obsessive-compulsive disorder, or other serious mental health disorders

I have had specific types of seizures or seizure emergencies in the last year.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 30mg/day of PRAX-628 for 8 weeks

8 weeks

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 trial is testing the effectiveness and safety of a new medication called PRAX-628 (30mg) for adults with certain types of seizures. It's an open label study where all participants know they're receiving the actual drug being tested.

How Is the Trial Designed?

Group I: Open-label 30mg/day PRAX-628 for 8 weeks Experimental Treatment 1 Intervention

Participants who meet all eligibility criteria will receive 30mg of PRAX-628 for 8 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 ...

Praxis Precision Medicines reports positive results of PRAX-628 study evaluating photo paroxysmal response (PPR) achieving 100% response in treated patients.

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 ...

PRAX-628-321 (POWER1) is a double-blind, randomized, multicenter, trial to evaluate the efficacy and safety of PRAX-628 in adults who can attest to ...

4.

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 ...

5.

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.

6.

neurology.org

neurology.org/doi/10.1212/WNL.0000000000203078

PRAX-628 is a Novel, Well-Tolerated, Activity Dependent ...

The profile of PRAX-628 may translate into well-tolerated efficacy in epilepsy as well as other indications caused by neuronal hyperexcitability. Disclosure: Dr ...

7.

investors.praxismedicines.com

investors.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-provides-update-advancing-clinical

Praxis Precision Medicines Provides Update on Advancing ...

Preclinical data demonstrates PRAX-628 is differentiated from standard of care, with the potential to be best-in-class for focal epilepsy. In ...