Double-blind, Randomized Trial of PRAX-628 in Adults With Focal Seizures to Evaluate Efficacy and Safety (POWER1)

Phase 2

Recruiting

• Conditions: Focal Seizure
• Interventions: Drug: 20mg/day PRAX-628 for 6 weeks then 30mg/day PRAX-628 for remaining 6 weeks; Drug: Placebo

• Registration Number: NCT06999902
• Lead Sponsor: Praxis Precision Medicines

Brief Summary

A Double-Blind, Randomized, Multicenter, Trial Evaluating the Efficacy and Safety of PRAX-628 in Adults With Focal Seizures (POWER1)

Detailed Description

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 concurrently taking at least 1, but no more than 3 acceptable anti-seizure medications.

Study Timeline

• Study Start: 2024-12-11
• Study First Submitted: 2025-03-14
• Status Verified: 2025-05
• Study First Submitted QC: 2025-05-23
• Last Update Submitted: 2025-05-23
• Study First Posted: 2025-05-31
• Last Update Posted: 2025-05-31
• Primary Completion: 2025-11-24
• Study Completion: 2025-12-08

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 230

Inclusion Criteria

1. A diagnosis of focal onset epilepsy according to the International League Against Epilepsy (ILAE) Classification of Epilepsy.
2. 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.

Exclusion Criteria

1. Subject has had any of the of the following within the 12-month period preceding trial entry: History of pseudo or psychogenic seizures, cluster seizures where the individual seizures cannot be counted, an episode of convulsive status epilepticus requiring hospitalization and intubation, or subject only has focal seizures with awareness that do not have motor activity.
2. Planned epilepsy surgery during the course of the clinical trial.
3. History of neurosurgery for seizures <1 year prior to enrollment, or radiosurgery <2 years prior to enrollment or vagus nerve stimulation (VNS) implantation.
4. History of schizophrenia, obsessive-compulsive disorder, or other serious mental health disorders.
5. History of malignancy, myeloproliferative or lymphoproliferative disorders within the past 5 years are excluded.
6. History of cardiac disease(s)/cardiac conduction disorders/or cardiac structural abnormality(ies).
7. 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.
8. 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.
9. Has received any other experimental or investigational drug, device or other therapy within 30 days or 5 half-lives (whichever is longer) prior to Screening, or any prior use of gene therapy.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Randomized, Double-Blind 20mg/day for 6 weeks then 30mg/day for remaining 6 weeks PRAX-628	20mg/day PRAX-628 for 6 weeks then 30mg/day PRAX-628 for remaining 6 weeks	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.
Randomized, Double-Blind Placebo	Placebo	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

Primary Outcome Measures

Name	Time	Method
To evaluate the efficacy of PRAX-628 compared to placebo on focal seizure frequency in adults currently taking 1 to 3 ASMs	12 weeks	Median percent change in monthly (28 days) focal seizure frequency from the Screening/Observation Period to the Treatment Period for PRAX-628 compared to placebo.

Secondary Outcome Measures

Name	Time	Method
To assess the safety and tolerability of PRAX-628 in adults with focal seizures	12 weeks	Changes in suicidality, as assessed by C-SSRS
To evaluate the efficacy of PRAX 628 compared to placebo on focal seizure frequency in adults currently taking 1 to 3 ASMs	12 weeks	Number of days post-randomization to reach the same number of monthly focal seizures as the Screening/Observation Period through the end of the Treatment Period; Proportion of participants experiencing seizure freedom, 100% reduction in monthly (28 days) focal seizure frequency from the Screening/Observation Period to the Treatment Period for PRAX-628 compared with placebo; Impact of PRAX-628 compared to placebo on PGI-S and CGI-S
To assess trends over time in efficacy of PRAX 628 on focal seizure frequency	12 weeks	Percent change in monthly focal seizure frequency from the Screening/Observation Period to the Treatment Period for PRAX-628 compared with placebo.

Trial Locations

Locations (1)

Praxis Research Site; 🇪🇸 Valencia, Spain