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)
Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Encoded Therapeutics
Age
0–6
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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.
You may not be if
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.
Clareo Health | 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)
