Thinking of joining a study?

Register your interest

Become a volunteer?

NCT06112275 | RECRUITING | Dravet Syndrome

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)
Sponsor:

Encoded Therapeutics

Brief Summary:

WAYFINDER is a Phase 1/2 study in Australia to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged 6 to \<84 months. The study follows an open-label, dose-escalation design.

Condition or disease

Dravet syndrome

Intervention/treatment

ETX101

Phase

PHASE1

PHASE2

Study Type : INTERVENTIONAL
Estimated Enrollment : 4 participants
Masking : NONE
Primary Purpose : TREATMENT
Official Title : WAYFINDER: a Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children with SCN1A-positive Dravet Syndrome
Actual Study Start Date : 2024-02-28
Estimated Primary Completion Date : 2030-06
Estimated Study Completion Date : 2030-06

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 6 Months to 83 Months
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
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.
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)

Location Details

NCT06112275

Please Choose a site

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

RECRUITING

Australia,

The Royal Children's Hospital

Melbourne, Australia,