Elsunersen for Epileptic Encephalopathy
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called Elsunersen for children with a rare condition that affects brain development and causes epilepsy. The goal is to determine if Elsunersen can safely reduce seizures in children who began experiencing them before three months of age and have a specific genetic issue called SCN2A. The trial includes different groups, with some receiving the treatment and others not, to compare results. Eligible children have experienced at least four seizures in the past month and have the SCN2A gene variant confirmed by genetic testing. As a Phase 3 trial, this study represents the final step before FDA approval, offering hope for an effective new treatment.
Do I need to stop my current medications for the trial?
The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is there any evidence suggesting that Elsunersen is likely to be safe for humans?
Research has shown that Elsunersen, a treatment under study for a rare form of epilepsy, has some early safety data. In one study, doctors examined how Elsunersen affected a young infant, providing initial insights into the treatment's tolerability. Although more research is needed, these early findings contribute to understanding its safety. Since this trial is in a later stage, earlier studies found Elsunersen to be fairly well-tolerated.
Clinical trial phases are designed to ensure a treatment's safety before reaching this stage, providing some confidence in its safety. However, individual experiences can vary, and discussing any concerns with the medical team involved in the trial is important.12345Why do researchers think this study treatment might be promising?
Researchers are excited about Elsunersen for epileptic encephalopathy because it offers a fresh approach to managing this challenging condition. Unlike standard treatments, which often include anticonvulsants like valproate or lamotrigine, Elsunersen works by targeting specific genetic pathways involved in epilepsy. This new mechanism of action could potentially address the root causes of seizures rather than just managing symptoms. Additionally, the option for both double-blind and open-label treatment periods in trials suggests flexibility in understanding its effectiveness and safety, which could lead to more tailored treatment strategies.
What evidence suggests that Elsunersen might be an effective treatment for epileptic encephalopathy?
Research has shown that elsunersen can significantly reduce seizures in patients with early-onset SCN2A developmental and epileptic encephalopathy (DEE). One study found that a premature baby treated with an antisense oligonucleotide, a type of genetic therapy, experienced over a 60% decrease in seizures, with this improvement persisting over time. Additionally, other trials have found that higher doses of elsunersen reduce seizures more effectively. This treatment also improved behavior and movement in some cases. These findings suggest that elsunersen could be promising for managing symptoms of SCN2A-DEE. Participants in this trial may receive elsunersen in either a double-blind or open-label treatment period, depending on their assigned cohort.13678
Who Is on the Research Team?
Medical Director
Principal Investigator
Praxis Precision Medicines
Are You a Good Fit for This Trial?
This trial is for children with early onset SCN2A Developmental and Epileptic Encephalopathy (SCN2A-DEE), a severe form of epilepsy. Specific eligibility criteria are not provided, but typically include age range, seizure frequency, and ability to undergo procedures.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Double-Blind Treatment
Participants receive either elsunersen or a sham procedure in a double-blind manner
Open-Label Treatment
Participants receive open-label elsunersen
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Elsunersen
Find a Clinic Near You
Who Is Running the Clinical Trial?
Praxis Precision Medicines
Lead Sponsor