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

Launched by ENCODED THERAPEUTICS · Oct 30, 2023

Keywords

ClinConnect Summary

The WAYFINDER trial is a study happening in Australia that aims to test a new gene therapy called ETX101 for children with Dravet syndrome, a severe form of epilepsy. This trial is specifically for children aged 6 months to just under 7 years who have a certain genetic change (an SCN1A variant) that is linked to this condition. To be eligible, children must have had their first seizure between 3 and 15 months of age, be diagnosed with Dravet syndrome, and be taking at least one medication to help prevent seizures.

Participants in this study will receive the ETX101 treatment, and researchers will closely monitor their health to see if the therapy is safe and effective. This means that doctors will check for any side effects and also see if the treatment helps reduce seizures. It’s important to note that children with certain other health issues or who have previously received gene therapy may not be eligible for this study. Overall, this trial offers an opportunity to explore a new potential treatment for a challenging condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Participant must have a predicted loss of function pathogenic or likely pathogenic SCN1A variant.
• • Participant must have experienced their first seizure between the ages of 3 and 15 months.
• • Participant must have a clinical diagnosis of Dravet syndrome or the treating clinician must have a 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 6-month 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.