A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
Recruiting · NCT05419492 · Interventional (participants receive a specific treatment) · Lead sponsor: Encoded Therapeutics
View the official record on ClinicalTrials.gov →Interventions studied
ETX101
What this trial is about
ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to \<36 months (Part 1A), aged ≥48 months to \<18 years (Part 1B), and aged ≥6 to \<48 months (Part 2). Part 1A follows an open-label, dose-escalation design, Part 1B follows an open-label design, and Part 2 is a randomized, double-blind, sham delayed-treatment control study.
Who can take part
Age range
6 Months to 17 Years
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 1, Phase 2
Study type
Interventional (participants receive a specific treatment)
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 it is running
14 locations listed across 11 US states.
- UCSF Benioff Children's Hospitals - San Francisco, California, United States
- Colorado Children's Hospital - Aurora, Colorado, United States
- Nicklaus Children's Hospital - Miami, Florida, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago, Illinois, United States
- Boston Children's Hospital - Boston, Massachusetts, United States
- Mott Children's Hospital - Ann Arbor, Michigan, United States
- Mayo Clinic - Rochester, Minnesota, United States
- Duke Children's Hospital & Health Center - Durham, North Carolina, United States
- Nationwide Children's Hospital - Columbus, Ohio, United States
- Oregon Health and Science University (OSHU) - Portland, Oregon, United States
- Cook Children's Medical Center - Fort Worth, Texas, United States
- Queen Elizabeth Hospital - Glasgow, United Kingdom
- Great Ormond Street Hospital - London, United Kingdom
- The Royal Children's Hospital - Melbourne, Australia