A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome

Launched by ENCODED THERAPEUTICS · Feb 22, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new gene therapy called ETX101 for young children with a type of epilepsy known as Dravet Syndrome, which is linked to a specific genetic change (SCN1A-positive). The trial is currently recruiting participants aged between 6 months and 4 years old. To be eligible, children must have had their first seizure between 3 and 15 months old and must be receiving at least one medication to help prevent seizures. It's important that they have a confirmed diagnosis of Dravet Syndrome or that their doctor strongly suspects they have it.

Participants in the trial will receive ETX101 and will be monitored closely to see how safe and effective it is. The study is designed to increase the dose of the therapy gradually to find the best balance of safety and effectiveness. Parents and guardians can expect regular check-ups and assessments during the trial, and their child's health will be closely observed throughout the process. This trial could provide important information about a new treatment option for children who struggle with Dravet Syndrome.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Participant has a predicted loss of function pathogenic or likely pathogenic SCN1A variant
• • Participant must have experienced their first seizure between the age of 3 and 15 months
• • Participant must have a clinical diagnosis of Dravet syndrome or the treating clinician must have high clinical suspicion of a diagnosis of Dravet syndrome
• • Participant is receiving at least one prophylactic antiseizure medication
• Exclusion Criteria:
• • Participant has another genetic mutation or clinical comorbidity which could potentially confound the typical Dravet phenotype
• • Participant has a known central nervous system structural and/or vascular abnormality (indicated by an MRI or CT scan of the brain).
• • Participant has an abnormality that may interfere with CSF distribution and/or has an existing ventriculoperitoneal shunt.
• • Participant is currently taking or has taken antiseizure medications (ASMs) at a therapeutic dose that are contraindicated in Dravet syndrome, including sodium channel blockers.
• • Participant has experienced seizure freedom for a period of 4 consecutive weeks within the 90-day period prior to informed consent.
• • Participant has previously received gene or cell therapy.
• • Participant is currently enrolled in a clinical trial or receiving an investigational therapy.
• • Participant has clinically significant underlying liver disease.