Dravet Syndrome study: ETX101
Official title A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
ClinicalTrials.gov ID: NCT05419492
What this study is testing
What is ETX101?
an investigational one-time intracerebroventricular gene‑therapy being studied as a potential treatment for Dravet syndrome is designed to deliver a viral vector that carries a regulatory element and engineered transcription factor to increase SCN1A gene expression in GABAergic neurons
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is testing whether ETX101 is safe and how well it works for kids with Dravet syndrome.
- Who it's for
- You may be able to join if you are a child with Dravet syndrome and are between 6 months and 18 years old.
- What's involved
- You would take ETX101, a new medicine, and have visits to check your health. In part 2 you might get a pretend treatment first.
- Time commitment
- The study lasts up to 12 months with several visits over that time.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 to 17
You may be able to join if
- 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 1 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...
- Participant is receiving at least one prophylactic antiseizure medication.
You likely can't join if
- 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.
See the full eligibility 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 1 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.
- 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.
The study team makes the final eligibility decision.
Where it is enrolling
- San Francisco, California, United States
- Aurora, Colorado, United States
- Miami, Florida, United States
- Chicago, Illinois, United States
- Ann Arbor, Michigan, United States
- Rochester, Minnesota, United States
- Durham, North Carolina, United States
- Columbus, Ohio, United States
- Portland, Oregon, United States
- Fort Worth, Texas, United States
- Melbourne, Australia
- Glasgow, United Kingdom
- London, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 months to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at San Francisco, California, United States; Aurora, Colorado, United States; Miami, Florida, United States; Chicago, Illinois, United States; Ann Arbor, Michigan, United States; Rochester, Minnesota, United States and 7 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.