ETX101: ETX101 is a non-replicating, recombinant adeno-associated viral vector serotype 9 (rAAV9) comprising a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A). ETX101 is intended as a one-time intracerebroventricular (ICV) administration.
Study summary
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.
Eligibility
Sex
ALL
Min age
6 Months
Max age
17 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
Percent change in monthly countable seizure frequency (MCSF) between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period. — Between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period (defined as Week 5 to Week 52).
Trial sites (14)
Facility
City
Region
Status
UCSF Benioff Children's Hospitals
San Francisco
California
Recruiting
Colorado Children's Hospital
Aurora
Colorado
Recruiting
Nicklaus Children's Hospital
Miami
Florida
Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago
Illinois
Recruiting
Boston Children's Hospital
Boston
Massachusetts
Not Yet Recruiting
Mott Children's Hospital
Ann Arbor
Michigan
Recruiting
Mayo Clinic
Rochester
Minnesota
Recruiting
Duke Children's Hospital & Health Center
Durham
North Carolina
Recruiting
Nationwide Children's Hospital
Columbus
Ohio
Not Yet Recruiting
Oregon Health and Science University (OSHU)
Portland
Oregon
Recruiting
Cook Children's Medical Center
Fort Worth
Texas
Recruiting
The Royal Children's Hospital
Melbourne
Australia
Recruiting
Queen Elizabeth Hospital
Glasgow
United Kingdom
Recruiting
Great Ormond Street Hospital
London
United Kingdom
Not Yet Recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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