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

6-17 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 1A), aged ≥48 months t...

Eligibility Criteria

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 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 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.

Key Trial Info

Start Date :

May 14 2024

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

January 1 2033

Estimated Enrollment :

47 Patients enrolled

Trial Details

Trial ID

NCT05419492

Start Date

May 14 2024

End Date

January 1 2033

Last Update

April 16 2026

Active Locations (10)

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Page 1 of 3 (10 locations)

1

UCSF Benioff Children's Hospitals

San Francisco, California, United States, 94158

2

Colorado Children's Hospital

Aurora, Colorado, United States, 80045

3

Nicklaus Children's Hospital

Miami, Florida, United States, 33155

4

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States, 60611

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