Evaluating ETX101 for treating Dravet syndrome in young children
ENDEAVOR: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Infants and Children With SCN1A-Positive Dravet Syndrome
This study is testing a new treatment called ETX101 to see if it can help reduce seizures in young children with Dravet syndrome.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 47 (estimated) |
| Ages | 6 Months to 17 Years |
| Sex | All |
| Sponsor | Encoded Therapeutics Industry-sponsored |
| Locations | 10 sites (San Francisco, California and 9 other locations) |
| Trial ID | NCT05419492 on ClinicalTrials.gov |
What this trial studies
This clinical study aims to assess the safety and efficacy of ETX101 in infants and children diagnosed with SCN1A-positive Dravet syndrome. It consists of two parts: Part 1 is an open-label, dose-escalation study for participants aged 6 to 36 months, while Part 2 is a randomized, double-blind, sham-controlled study for those aged 6 to 48 months. The study will evaluate how well ETX101 works in reducing seizures in this specific patient population.
Who should consider this trial
Good fit: Ideal candidates are infants and children aged 6 months to 48 months with a confirmed SCN1A mutation and a clinical diagnosis of Dravet syndrome.
Not a fit: Patients with other genetic mutations or significant comorbidities that could affect the study outcomes may not benefit from this treatment.
Why it matters
Potential benefit: If successful, this treatment could significantly reduce seizure frequency and improve the quality of life for children with Dravet syndrome.
How similar studies have performed: Previous studies targeting SCN1A-positive Dravet syndrome have shown promise, but this specific approach with ETX101 is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Participant must be aged between ≥6 months and \<36 months in Part 1A, ≥48 months and \<18 years in Part 1B, ≥6 months and \<48 months in Part 2. * 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 has received sodium channel blockers during the Pre-Dosing Seizure Period. * 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.
Where this trial is running
San Francisco, California and 9 other locations
- UCSF Benioff Children's Hospitals — San Francisco, California, United States (Recruiting)
- Colorado Children's Hospital — Aurora, Colorado, United States (Not_yet_recruiting)
- Nicklaus Children's Hospital — Miami, Florida, United States (Recruiting)
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (Recruiting)
- Boston Children's Hospital — Boston, Massachusetts, United States (Not_yet_recruiting)
- Oregon Health and Science University (OSHU) — Portland, Oregon, United States (Recruiting)
- Cook Children's Medical Center — Fort Worth, Texas, United States (Recruiting)
- The Royal Children's Hospital — Melbourne, Australia (Recruiting)
- Queen Elizabeth Hospital — Glasgow, United Kingdom (Not_yet_recruiting)
- Great Ormond Street Hospital — London, United Kingdom (Not_yet_recruiting)
Study contacts
- Study coordinator: Encoded Patient Advocacy
- Email: patientadvocacy@encoded.com
- Phone: +1 (650) 398-4301
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.