A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome
NCT ID: NCT06283212
Last Updated: 2025-12-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
5 participants
INTERVENTIONAL
2024-05-09
2030-10-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Cohort A
Cohort A will evaluate ETX101 dose level 1.
ETX101
ETX101 is composed of a non-replicating, recombinant adeno-associated viral serotype 9 (rAAV9) vector used to deliver a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A)
Cohort B
Cohort B will evaluate ETX101 dose level 2.
ETX101
ETX101 is composed of a non-replicating, recombinant adeno-associated viral serotype 9 (rAAV9) vector used to deliver a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A)
Cohort C
Cohort C will evaluate ETX101 dose level 3.
ETX101
ETX101 is composed of a non-replicating, recombinant adeno-associated viral serotype 9 (rAAV9) vector used to deliver a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A)
Cohort D
Cohort D will evaluate ETX101 dose level 4.
ETX101
ETX101 is composed of a non-replicating, recombinant adeno-associated viral serotype 9 (rAAV9) vector used to deliver a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A)
Interventions
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ETX101
ETX101 is composed of a non-replicating, recombinant adeno-associated viral serotype 9 (rAAV9) vector used to deliver a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A)
Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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 is currently taking or has taken antiseizure medications (ASMs) at a therapeutic dose that are contraindicated in Dravet syndrome, including sodium channel blockers.
* 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.
6 Months
47 Months
ALL
No
Sponsors
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Encoded Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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Salvador Rico, M.D., Ph. D
Role: STUDY_DIRECTOR
Encoded Therapeutics
Locations
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Queen Elizabeth Hospital
Glasgow, , United Kingdom
Great Ormond Street Hospital
London, , United Kingdom
Sheffield Children's Hospital
Sheffield, , United Kingdom
Countries
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Other Identifiers
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ETX-DS-005
Identifier Type: -
Identifier Source: org_study_id
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