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

Phase 2

Recruiting

• Conditions: Focal Onset Seizure; Primary Generalized Epilepsy
• Interventions: Drug: 30mg PRAX-628

• Registration Number: NCT06908356
• Lead Sponsor: Praxis Precision Medicines

Brief Summary

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

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 Timeline

• Study Start: 2025-01-10
• Status Verified: 2025-03
• Study First Submitted: 2025-03-19
• Study First Submitted QC: 2025-03-26
• Last Update Submitted: 2025-03-26
• Study First Posted: 2025-04-03
• Last Update Posted: 2025-04-03
• Primary Completion: 2025-07
• Study Completion: 2025-07

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 50

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Open-label 30mg/day PRAX-628 for 8 weeks	30mg PRAX-628	Participants who meet all eligibility criteria will receive 30mg of PRAX-628 for 8 weeks.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
To evaluate the efficacy of PRAX 628 on seizure frequency in adults with FOS or PGTCS currently taking ASMs	8 weeks	• 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
CGI-S Change from Baseline	8 weeks	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).
PGI-S Change from Baseline	8 weeks	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).
To assess the safety and tolerability of PRAX-628 in adults with FOS or PGTCS currently taking ASMs	8 weeks	The number of participants with Adverse Events (AE) will be reported by preferred term

Trial Locations

Locations (1)

Praxis Research Site; 🇪🇸 Madrid, Spain