Focal Onset Seizure, Primary Generalized Epilepsy
Conditions
Keywords
Focal Onset Seizure, Primary Generalized Epilepsy, Tonic-Clonic
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.
Interventions
DRUG30mg PRAX-628
Once daily oral
Sponsors
Praxis Precision Medicines
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| To evaluate the efficacy of PRAX 628 on seizure frequency in adults with FOS or PGTCS currently taking ASMs | 8 weeks | • 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. |
| 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 |
Countries
Australia, Spain, United States
Contacts
Primary ContactHead of Pharmacovigilance
Outcome results
None listed