An Open Label Trial to Evaluate the Efficacy and Safety of PRAX-628 in Adults With Focal Onset or Tonic-Clonic Seizures

An Open Label Clinical Trial to Evaluate the Efficacy and Safety of PRAX-628 in Adult Patients With Focal Onset or Primary Generalized Tonic-Clonic Seizures

An Open Label Clinical Trial to Evaluate the Efficacy and Safety of PRAX-628 in Adult Patients With Focal Onset or Primary Generalized Tonic-Clonic Seizures

Study Overview

• Status: Recruiting
• Conditions: Focal Onset Seizure; Primary Generalized Epilepsy
• Intervention / Treatment: Drug: 30mg PRAX-628

Detailed Description

An open label clinical trial to evaluate the efficacy and safety of PRAX-628 in adult patients with focal onset or primary generalized tonic-clonic seizures who can attest to concurrently taking at least 1, but no more than 3 acceptable anti-seizure medications.

• Study Type: Interventional
• Enrollment (Estimated): 50
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Head of Pharmacovigilance; Phone Number: 617-300-8460; Email: clinicaltrials@praxismedicines.com

Study Locations

• Australia
  • Heidelberg, Australia, 03084
    • Recruiting
    • Praxis Research Site
  • Melbourne, Australia, 03004
    • Recruiting
    • Praxis Research Site
• Spain
  • Madrid, Spain, 28010
    • Recruiting
    • Praxis Research Site
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85032
      • Recruiting
      • Praxis Research Site
  • Florida
    • Miami Lakes, Florida, United States, 30116
      • Recruiting
      • Praxis Research Site
  • Maryland
    • Bethesda, Maryland, United States, 20817
      • Recruiting
      • Praxis Research Site
  • Missouri
    • Chesterfield, Missouri, United States, 63005
      • Recruiting
      • Praxis Research Site
  • New Jersey
    • Hackensack, New Jersey, United States, 07601
      • Recruiting
      • Praxis Research Site
  • New York
    • Middletown, New York, United States, 10941
      • Recruiting
      • Praxis Research Site
  • Texas
    • Round Rock, Texas, United States, 78681
      • Recruiting
      • Praxis Research Site
    • Seabrook, Texas, United States, 77586
      • Recruiting
      • Praxis Research Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• A diagnosis of focal onset seizures or idiopathic generalized tonic clonic seizures.
• Evidence by computed tomography (CT) or magnetic resonance imaging (MRI) in the past that has ruled out a progressive cause of epilepsy.
• Participant must have been receiving stable doses of allowable ASMs (a minimum of 1 and a maximum of 3 ASMs).
• Participant and/or caregiver (if applicable) self-reports at least 2 countable focal onset seizures per month for focal onset patients, or 1 countable generalized tonic-clonic seizure per month in the 3 months immediately prior to the Screening/Observation Period for PGTCS patients.

Exclusion Criteria:

• History of pseudo or psychogenic seizures, or cluster seizures only, within the 12-month period preceding study entry where the individual seizures cannot be counted, or an episode of convulsive status epilepticus requiring hospitalization and intubation in the 12 months prior to Screening.
• Planned epilepsy surgery during the course of the clinical trial.
• History of neurosurgery for seizures <1 year prior to enrollment, or radiosurgery <2 years prior to enrollment.
• Schizophrenia and obsessive-compulsive disorder, or other serious mental health disorders.
• Active suicidal plan/intent in the past 6 months, or a history of suicide attempt in the last 2 years, or more than 1 lifetime suicide attempt.
• Participants with a history of malignancy, myeloproliferative or lymphoproliferative disorders within the past 5 years are excluded.
• History of cardiac disease(s)/cardiac conduction disorders/or cardiac structural abnormality(ies).
• Is pregnant or 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.
• 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.
• Vigabatrin: Use in the last 5 years without stable visual fields tested twice over the 12 months after the last dose of vigabatrin.
• Felbamate: If used as a concomitant ASM, patients must be on felbamate for at least 2 years, with a stable dose for 2 months prior to Screening. If a patient received felbamate in the past, it must have been discontinued 2 months prior to Screening.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Open-label 30mg/day PRAX-628 for 8 weeks; Participants who meet all eligibility criteria will receive 30mg of PRAX-628 for 8 weeks.	Drug: 30mg PRAX-628; Once daily oral

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the efficacy of PRAX-628 on seizure frequency in adults with FOS or PGTCS currently taking ASMs	Median percent change in monthly (28 days) seizure frequency from the Screening/Observation Period to the Treatment Period for PRAX-628	8 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the efficacy of PRAX 628 on seizure frequency in adults with FOS or PGTCS currently taking ASMs	• Proportion of participants experiencing a ≥50% reduction in monthly (28 days) seizure frequency from the Screening/Observation Period to the Treatment Period (Responder Rate) for PRAX-628.	8 weeks
To evaluate the efficacy of PRAX 628 on seizure frequency in adults with FOS or PGTCS currently taking ASMs	• Proportion of participants experiencing seizure freedom, 100% reduction in monthly (28 days) seizure frequency from the Screening/Observation Period to the Treatment Period for PRAX-628.	8 weeks
To evaluate the efficacy of PRAX 628 on seizure frequency in adults with FOS or PGTCS currently taking ASMs	• Number of days after the first administration of PRAX-628 to reach the same or higher number of monthly seizures from the Screening/Observation Period to the Treatment Period	8 weeks
CGI-S Change from Baseline	CGI-S assesses the clinician's impression of the participant's current illness state. The clinician should use his/her total clinical experience with this patient population and rate the current severity on a 7-point scale from 1 (normal, not at all ill) to 7 (among the most extremely ill patients).	8 weeks
PGI-S Change from Baseline	PGI-S assesses the participants' impression of their current illness state. The participant is required to assess their condition on a 7-point scale from 1 (Not present) to 7 (extremely severe).	8 weeks
To assess the safety and tolerability of PRAX-628 in adults with FOS or PGTCS currently taking ASMs	The number of participants with Adverse Events (AE) will be reported by preferred term	8 weeks

Collaborators and Investigators

• Sponsor: Praxis Precision Medicines
• Investigators: Study Director: Medical Director, Praxis Precision Medicines

Study record dates

Study Major Dates

• Study Start (Actual): January 10, 2025
• Primary Completion (Estimated): July 1, 2025
• Study Completion (Estimated): July 1, 2025

Study Registration Dates

• First Submitted: March 19, 2025
• First Submitted That Met QC Criteria: March 26, 2025
• First Posted (Actual): April 3, 2025

Study Record Updates

• Last Update Posted (Actual): April 3, 2025
• Last Update Submitted That Met QC Criteria: March 26, 2025
• Last Verified: March 1, 2025

More Information

Terms related to this study

• Keywords: Primary Generalized Epilepsy; Focal Onset Seizure; Tonic-Clonic
• Additional Relevant MeSH Terms: Neurologic Manifestations; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epilepsy; Seizures; Epilepsy, Generalized
• Other Study ID Numbers: PRAX-628-212

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No