Zorevunersen for Dravet Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial examines the effectiveness of a new treatment, zorevunersen, for individuals with Dravet syndrome, a severe form of epilepsy characterized by frequent seizures. Researchers aim to determine if zorevunersen is effective and safe in reducing these seizures. The trial includes two groups: one receiving zorevunersen and the other a placebo (an inactive treatment for comparison). Individuals diagnosed with Dravet syndrome, experiencing specific types of seizures, and who have tried at least two different treatments may be suitable candidates for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to potentially groundbreaking treatment advancements.
Do I need to stop my current medications for the trial?
You can continue taking your current anti-seizure medications as long as they are not sodium channel blockers like phenytoin or carbamazepine. Your medications should be stable during the baseline period.
Is there any evidence suggesting that zorevunersen is likely to be safe for humans?
A previous study monitored 81 patients who took zorevunersen for safety. The results showed that patients generally tolerated the treatment well. While some experienced side effects, these were mostly mild, with serious issues being rare. This treatment is being tested for Dravet syndrome, a type of epilepsy that begins in childhood. As zorevunersen is in a later stage of testing, there is increased confidence in its safety for humans. However, participating in a trial always carries the possibility of unknown risks.12345
Why do researchers think this study treatment might be promising for Dravet syndrome?
Researchers are excited about zorevunersen for Dravet Syndrome because it offers a novel approach compared to current treatments like valproic acid and stiripentol. Unlike these standard anti-seizure medications, zorevunersen is designed to target the underlying genetic cause of Dravet Syndrome, focusing on modifying the expression of the SCN1A gene. This targeted genetic approach has the potential to address the root cause of the condition rather than merely managing symptoms, which could lead to more effective and long-lasting results for patients.
What evidence suggests that zorevunersen might be an effective treatment for Dravet syndrome?
Research has shown that zorevunersen, which participants in this trial may receive, may help treat Dravet syndrome. Studies have found that it can significantly reduce the number of major seizures in patients. Additionally, patients demonstrated better communication skills and improved daily living abilities. These results suggest that zorevunersen could be a good option for managing Dravet syndrome symptoms. Overall, the evidence is promising for those considering this treatment.12346
Are You a Good Fit for This Trial?
This trial is for patients with Dravet Syndrome who've tried at least two seizure treatments without success or stopped due to side effects. They must be on one anti-seizure medication and have had a certain number of major motor seizures recently. Their treatment regimen should be stable, and they need a specific gene variant linked to the syndrome.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive zorevunersen or sham treatment to evaluate efficacy, safety, and tolerability
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension (optional)
Participants may opt into continuation of treatment long-term with zorevunersen
What Are the Treatments Tested in This Trial?
Interventions
- Zorevunersen
Find a Clinic Near You
Who Is Running the Clinical Trial?
Stoke Therapeutics, Inc
Lead Sponsor