At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- ETX101 (drug)
- How long the study runs
- Study runs about 104 months (dates as stated)
- About the drug or intervention
- ETX101 — drug: 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).
- Patient visit burden
- Not specified by the sponsor
In plain English
This study is looking at how safe and effective a treatment called ETX101 is for babies and children with Dravet syndrome caused by a change (variant) in a gene called SCN1A. Dravet syndrome is a rare condition that causes seizures that are hard to control. The study is being run by a company called Encoded Therapeutics.
Who can take part
- Aged between 6 months and under 36 months for Part 1A, between 48 months and under 18 years for Part 1B, and between 6 months and under 48 months for Part 2
- Has a predicted loss of function SCN1A gene variant that is known or likely to cause disease
- Had their first seizure between 3 and 15 months of age
- Has a diagnosis of Dravet syndrome, or their doctor strongly suspects Dravet syndrome
- Is already taking at least one medicine to prevent seizures
Who may not be able to
- Has another genetic change or health condition that could affect the typical pattern of Dravet syndrome
- Has a known structural or blood vessel problem in the brain (shown by an MRI or CT scan)
- Has a problem that may affect fluid around the brain and spinal cord, or has a shunt draining fluid from the brain to the tummy
- Has taken medicines called sodium channel blockers during the pre-dosing seizure period
- Has been seizure-free for 4 weeks in a row within the 90 days before agreeing to take part
- Has had gene therapy or cell therapy before
- Is currently in another clinical trial or having another experimental treatment
- Has significant liver disease
What taking part involves
- • Taking part in a study of ETX101, a treatment being tested for SCN1A-positive Dravet syndrome. Further details about how it is given are not stated — ask the trial team.
Time commitment: How many visits, how long the study lasts, and exactly what taking part involves are not stated — ask the trial team.
Plain-English summary (AI-generated) from registry data. Not eligibility advice — only the trial team can confirm whether you can take part. Use the eligibility checker to see how your health profile matches this trial.
- Type of study
- Testing a treatment
- Ages
- 6 Months to 17 Years
- Who
- All
- Number of participants
- 47
- Started
- 2024-05-14
- Last checked
- 2026-02
Plain English Summary
What is this study?
- • Testing a new treatment for dravet syndrome
- • Phase1/Phase2 - 47 participants
- • 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)
Who can take part?
- • Ages 6 Months to 17 Years
- • Diagnosed with dravet syndrome
Where?
- • Glasgow - Queen Elizabeth Hospital
- • London - Great Ormond Street Hospital
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 6 Months - 17 Years
- Who can join: All genders
Treatment history
Treatments you must have had:
- ✓ a predicted loss of function pathogenic or likely pathogenic SCN1A variant
- ✓ experienced their first seizure between the ages of 3 and 15 months
What the study is looking for
- ✓Participant must be aged between ≥6 months and \<36 months in Part 1A, ≥48 months and \<18 years in Part 1B, ≥6...
- ✓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...
- ✓Participant is receiving at least one prophylactic antiseizure medication.
Who cannot take part
- ✗Participant has another genetic mutation or clinical other health condition which could potentially confound the typical Dravet...
- ✗Participant has a known central nervous system structural and/or vascular abnormality (indicated by an MRI or CT...
- ✗Participant has an abnormality that may interfere with CSF distribution and/or has an existing ventriculoperitoneal...
- ✗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...
See the full criteria
Where Is This Study? (2 UK sites)
Queen Elizabeth Hospital
Glasgow G51 4TF, United Kingdom
Great Ormond Street Hospital
London WC1N3JH, United Kingdom
How to Get in Touch
Encoded Patient Advocacy
Sponsor contactCONTACT