What is ETX101 studying for Dravet syndrome?

ETX101 is being studied as a gene therapy for children with SCN1A-positive Dravet syndrome. This trial aims to evaluate how ETX101 affects seizure frequency and cognitive function. Encoded Therapeutics is leading this research to understand its potential impact on this condition.

Who may be eligible for this Dravet syndrome clinical trial?

Children aged between 6 months and 17 years with a diagnosis or strong suspicion of Dravet syndrome may be eligible. They must have had their first seizure between 3 and 15 months of age and be taking at least one antiseizure medication. Please review eligibility criteria for more details.

Is there any cost to participate in this Dravet syndrome clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel and related expenses may vary and are not guaranteed to be covered. Participants should plan accordingly.

Actively Recruiting

Phase 1

Phase 2

Age: 6Months - 17Years

All Genders

ID05419492

ENDEAVOR Study of ETX101 Gene Therapy for Infants and Children With SCN1A-Positive Dravet Syndrome from 6 months to 17 years

Led by Encoded Therapeutics · Updated on 2026-07-29

Participants Needed

Research Sites

261 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are investigating ETX101, a gene therapy delivered via an adeno-associated viral vector, in infants and children diagnosed with SCN1A-positive Dravet syndrome. The study includes multiple age groups ranging from 6 months to under 18 years and aims to assess the safety and effects of ETX101. It is designed as a Phase 12, multicenter trial with different parts evaluating dose levels and treatment impact using a randomized, controlled approach in later stages. The study is divided into parts Part 1A uses an open-label, dose-escalation design to test up to four doses of ETX101 in participants aged 6 to under 36 months. Part 1B is open-label and tests a single dose in participants aged 48 months to under 18 years. Part 2 is a double-blind, randomized, sham delayed-treatment control study involving participants aged 6 to under 48 months, where participants receive either ETX101 or a sham procedure with delayed treatment. This part includes two cohorts and lasts up to 52 weeks. Participants will be closely monitored through the study with assessments of seizure frequency, cognitive and adaptive behavior changes using tools like the Bayley-4 and Vineland-3 scales, and clinical global impressions over 52 weeks. Safety and effectiveness are evaluated by comparing seizure counts before and after dosing. The study includes various cognitive and behavioral outcome measures and follows participants from dosing up to one year for treatment effects and safety observations.

CONDITIONS

Brief Title

A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome

Who Can Participate

Age: 6Months - 17Years

All Genders

Eligibility Criteria

You may qualify if you...

Participant must be aged between 6 months and under 36 months in Part 1A.
Participant must be aged between 48 months and under 18 years in Part 1B.
Participant must be aged between 6 months and under 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.

You will not qualify if you...

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.

Research Team

Encoded Patient Advocacy

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