Developmental and Epileptic Encephalopathy 1
Conditions
Keywords
DEE, Developmental and Epileptic Encephalopathy, epilepsy
Brief summary
A Phase 3, Randomized, Multi-Center, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Relutrigine in Participants with Developmental and Epileptic Encephalopathies Followed by an Open-Label Extension
Interventions
DRUG1.0mg/kg/day PRAX-562
Once daily orally or gastronomy/jejunostomy
DRUG1.5mg/kg/day PRAX-562
Once daily orally or gastronomy/jejunostomy
DRUGPlacebo
Once daily orally or gastronomy/jejunostomy
Sponsors
Praxis Precision Medicines
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
2 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Has a documented diagnosis of a developmental and epileptic encephalopathy. * Onset of seizures \<12 years old. * Has a weight \>7 kg at the time of signing consent/assent.
Exclusion criteria
* Has a history of left bundle branch block, arrhythmias, Brugada syndrome, congenital heart disease, familial short QT syndrome, or family history of sudden death or ventricular arrhythmias, including idiopathic ventricular fibrillation. * Had 2 or more episodes of convulsive status epilepticus requiring hospitalization and intubation in the 6 months prior to Screening. * Has an abnormal ECG reading, including a QT interval corrected for heart rate using Bazett's method (QTcB) \<350 and \>450 ms (males), or \<360 and \>460 ms (females) at Screening and/or on Day 1. * Any nerve stimulation must have been placed at least 3 months prior to Screening with at least 1 month of stable settings prior to Screening. * 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, including any prior use of gene therapy. * Is currently pregnant or breastfeeding or is planning to become pregnant during the clinical trial or within 5 half-lives of the last study drug dose.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To assess the effect of relutrigine on seizure frequency in participants with DEEs compared to placebo | 16 weeks | Change from baseline in monthly motor seizure frequency |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Achieve >50% reduction in monthly seizure frequency from baseline | 16 weeks | Proportion of patients achieving \>50% reduction in monthly seizure frequency from baseline |
| Change in seizure-free days | 16 weeks | Change in seizure-free days from baseline |
| Clinical Global Impression-Severity questionnaire | 16 weeks | CGI-S at baseline after 16 weeks |
| Clinical Global Impression-Improvement questionnaire | 16 weeks | CGI-I subdomains scores after 16 weeks |
| Caregiver Global Impression-Severity questionnaire | 16 weeks | CgGI-S at baseline after 16 weeks |
| Caregiver Global Impression-Improvement questionnaire | 16 weeks | CgGI-I subdomains scores after 16 weeks |
| To evaluate the safety and tolerability of relutrigine in participants with DEEs | 16 weeks | Incidence and severity of TEAEs |
| Columbia-Suicide Severity Rating Scale questionnaire | 16 weeks | Incidence of suicidal ideation or behavior as measured by the C-SSRS |
| Evaluate the safety and tolerability of relutrigine in participants with DEEs | 16 weeks | Incidence of clinically significant ECG abnormalities |
| To evaluate the safety labs and tolerability of relutrigine in participants with DEEs | 16 weeks | The principal investigator (PI) or sub investigator will review the laboratory report and document this review. Any clinically significant adverse changes occurring during the clinical trial will be documented as adverse events. |
| To evaluate the change in respiratory rate and tolerability of relutrigine in participants with DEEs | 16 weeks | Change in respiratory rate in breaths per minute |
| To evaluate the change in blood pressure and tolerability of relutrigine in participants with DEEs | 16 weeks | Change in blood pressure in mm/Hg |
| To evaluate the change in pulse and tolerability of relutrigine in participants with DEEs | 16 weeks | Change in heart rate in beats per minute |
| To evaluate the change in body temperature and tolerability of relutrigine in participants with DEEs | 16 weeks | Change in tympanic temperature in Celsius |
Countries
Australia, Brazil, United States
Contacts
STUDY_DIRECTORMedical Director
Praxis Precision Medicines
Outcome results
None listed