RECRUITING

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

Conditions

Dravet Syndrome Dravet SCN1A DEE developmental and epileptic encephalopathy SCN1A-positive SCN1A+

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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 months (Part 2). Part 1 follows an open-label, dose-escalation design, and Part 2 is a randomized, double-blind, sham delayed-treatment control, dose-selection study.

Official Title

ENDEAVOR: a Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Infants and Children with SCN1A-Positive Dravet Syndrome

Quick Facts

Study Start:2024-05-14

Study Completion:2031-04

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05419492

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:6 Months to 47 Months

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion Criteria
* 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.	* 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.

Inclusion Criteria

Exclusion Criteria

* 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.

* 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.

Contacts and Locations

Study Contact

Encoded Patient Advocacy

CONTACT

+1 (650) 398-4301

patientadvocacy@encoded.com

Principal Investigator

Salvador Rico, M.D., Ph.D

STUDY_DIRECTOR

Encoded Therapeutics

Study Locations (Sites)

UCSF Benioff Children's Hospitals

San Francisco, California, 94158

United States

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

Chicago, Illinois, 60611

United States

Cook Children's Medical Center

Fort Worth, Texas, 76104

United States

Collaborators and Investigators

Sponsor: Encoded Therapeutics

Salvador Rico, M.D., Ph.D, STUDY_DIRECTOR, Encoded Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-05-14

Study Completion Date2031-04

Study Record Updates

Study Start Date2024-05-14

Study Completion Date2031-04

Terms related to this study

Keywords Provided by Researchers

Dravet
SCN1A
DEE
developmental and epileptic encephalopathy
Dravet Syndrome
SCN1A-positive
SCN1A+

Additional Relevant MeSH Terms

Dravet Syndrome