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
Trial Overview
The study tests the efficacy and safety of Elsunersen at two different doses (0.5mg and 1mg) compared to a sham procedure in pediatric patients. It's randomized, meaning participants are assigned by chance, double-blind so neither researchers nor participants know who gets what treatment.
How Is the Trial Designed?
Open-label elsunersen
Open-label elsunersen
Double-blind treatment period elsunersen
Double-blind sham-procedure
Find a Clinic Near You
Who Is Running the Clinical Trial?
Praxis Precision Medicines
Lead Sponsor
Citations
Antisense oligonucleotide treatment in a preterm infant with ...
A >60% reduction in seizure frequency corresponding to five to seven seizures per hour was observed, which has been sustained during follow-up ...
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to ...
To assess secondary efficacy outcomes of elsunersen in participants with early-onset SCN2A DEE, Change in motor seizure-free days from baseline, 24 weeks ; CGI-S ...
3.
investors.praxismedicines.com
investors.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-provides-update-advancing-clinicalPraxis Precision Medicines Provides Update on Advancing ...
More than 70% of early-onset SCN2A-DEE patients live with uncontrolled seizures, and approximately 75% live with severe intellectual disability ...
4.
insideprecisionmedicine.com
insideprecisionmedicine.com/topics/precision-medicine/antisense-treatment-reduces-preterm-infants-seizures-by-more-than-half/Antisense Treatment Reduces Preterm Infant's Seizures by ...
A preterm infant with a genetic disease had the frequency of seizures reduced by over half after treatment with an antisense ...
Praxis Precision Medicines Provides Corporate Update ...
In vivo, elsunersen has demonstrated significant, dose-dependent reduction in seizures, improvement in behavioral and locomotor activity and ...
6.
investors.praxismedicines.com
investors.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-receives-prime-designation-emaPraxis Precision Medicines Receives PRIME Designation from ...
Elsunersen is an antisense oligonucleotide (ASO) designed to selectively decrease SCN2A gene expression, directly targeting the underlying cause ...
7.
ctv.veeva.com
ctv.veeva.com/study/randomized-double-blind-trial-of-elsunersen-in-pediatric-scn2a-dee-to-assess-efficacy-and-safetyA Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to ...
Has a documented Gain of Function SCN2A variant confirmed through genetic testing. Has onset of seizures prior to 3 months of age.
Elsunersen for Early Onset SCN2A Developmental and ...
This study is focused on understanding if a new treatment called Elsunersen can help children who have a rare condition known as early onset SCN2A ...
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