NCT06283212Active, not recruitingPhase 1/2
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome
EXPEDITION: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome
Source: ClinicalTrials.gov · Last updated 8 Jun 2026
Brief summary (registry, in English)
EXPEDITION is a Phase 1/2 study in the UK to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet Syndrome aged 6 to \< 48 months. The study follows and open-label, dose-escalation design.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 5 (actual)
- First submitted
- 22 Feb 2024
- Start date
- 9 May 2024
- Primary completion date
- Oct 2030
- Lead sponsor
- Encoded Therapeutics
- Diseases
- Epilepsy
- NCT number
- NCT06283212
What you commit to
- Study type: Interventional · Phase 1/2
- Participants enrolled: 5
- From 9 May 2024 to the primary completion date, Oct 2030
- 3 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
Other illness
- Participant has clinically significant underlying liver disease.
Lab values
- Participant is currently taking or has taken antiseizure medications (ASMs) at a therapeutic dose that are contraindicated in Dravet syndrome, including sodium channel blockers.
Other
- 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.
Who the study is looking for
- Participant has a predicted loss of function pathogenic or likely pathogenic SCN1A variant
- Participant must have experienced their first seizure between the age of 3 and 15 months
- Participant must have a clinical diagnosis of Dravet syndrome or the treating clinician must have high clinical suspicion of a diagnosis of Dravet syndrome
- Participant is receiving at least one prophylactic antiseizure medication
Age in the registry: 6 months to 47 months · Sex: All · Does not accept healthy volunteers
Sites (3)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Queen Elizabeth Hospital Coordinator of this site? Confirm its status | Glasgow | United Kingdom | Unknown status |
| Great Ormond Street Hospital Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| Sheffield Children's Hospital Coordinator of this site? Confirm its status | Sheffield | United Kingdom | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.