Status:
RECRUITING
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
Lead Sponsor:
Encoded Therapeutics
Conditions:
Dravet Syndrome
Eligibility:
All Genders
6-47 years
Phase:
PHASE1
PHASE2
Brief Summary
ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to \<36 months (Part 1) and aged ≥6 to \<48...
Eligibility Criteria
Inclusion
- Participant must be aged between ≥6 months and \<36 months in Part 1 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 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.
Exclusion
- 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 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, including under an expanded access and/or compassionate use program.
- Participant has clinically significant underlying liver disease.
Key Trial Info
Start Date :
May 14 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
April 1 2031
Estimated Enrollment :
22 Patients enrolled
Trial Details
Trial ID
NCT05419492
Start Date
May 14 2024
End Date
April 1 2031
Last Update
June 12 2025
Active Locations (3)
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1
UCSF Benioff Children's Hospitals
San Francisco, California, United States, 94158
2
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
3
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104