Evaluating ETX101 for treating Dravet syndrome in young children

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical study aims to assess the safety and efficacy of ETX101 in infants and children diagnosed with SCN1A-positive Dravet syndrome. It consists of two parts: Part 1 is an open-label, dose-escalation study for participants aged 6 to 36 months, while Part 2 is a randomized, double-blind, sham-controlled study for those aged 6 to 48 months. The study will evaluate how well ETX101 works in reducing seizures in this specific patient population.

Why it matters

Who can join: the full rules

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 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.

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's running

• UCSF Benioff Children's Hospitals
  San Francisco, California, United States Recruiting
• Colorado Children's Hospital
  Aurora, Colorado, United States Recruiting
• Nicklaus Children's Hospital
  Miami, Florida, United States Recruiting
• Ann & Robert H. Lurie Children's Hospital of Chicago
  Chicago, Illinois, United States Recruiting
• Mott Children's Hospital
  Ann Arbor, Michigan, United States Recruiting
• Mayo Clinic
  Rochester, Minnesota, United States Recruiting

Study contacts

• Study coordinator: Encoded Patient Advocacy
• Email: patientadvocacy@encoded.com
• Phone: +1 (650) 398-4301

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• ION337 safety and tolerability in children with Dravet syndrome

  This will test whether ION337 is safe and well tolerated in children ages 2 to 12 with Dravet syndrome.

  Recruiting at 7 sites Phase 1/2 Ionis Pharmaceuticals, Inc. · Ann Arbor, Michigan and 6 other locations · NCT07531745

• Tracking development, seizures, and biomarkers over four years in children and young adults with SCN1A-related Dravet syndrome

  This project will follow children and young people with SCN1A-related Dravet syndrome for four years to see how their development, seizure patterns, and blood metabolite markers change over time.

  Recruiting Assistance Publique - Hôpitaux de Paris · Paris, Ap-hp / DRCI · NCT07251673

• Fintepla for Dravet Syndrome: testing brain blood flow and connections

  This study tests how Fintepla affects brain blood flow and network connections in Dravet syndrome patients who have convulsive seizures.

  Recruiting Phase 4 The University of Texas Health Science Center, Houston · Houston, Texas · NCT07112365

• PET‑MRI mapping of brainstem serotonin pathways in adults with Dravet syndrome

  This uses a PET‑MRI scan with the tracer [18F]MPPF to see if serotonin pathways in the brainstem differ in adults with Dravet syndrome compared with healthy controls.

  Recruiting Hospices Civils de Lyon · Bron, Rhone · NCT07013331

• Testing BMB-101 for Absence Epilepsy and Related Conditions

  This study is testing a new treatment called BMB-101 to see if it can help reduce seizures in people with Absence Epilepsy and related conditions.

  Recruiting at 5 sites Phase 2 Bright Minds Biosciences Pty Ltd · Randwick, New South Wales and 4 other locations · NCT06401538

• Measuring GABA Levels in Children with Dravet Syndrome

  This study is trying to measure GABA levels in the brains of children with Dravet Syndrome to see how they compare to kids without the condition, hoping to find new ways to understand and treat the syndrome.

  Recruiting Cook Children's Health Care System · Fort Worth, Texas · NCT05651204

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.