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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT05419492
Other study ID # ETX-DS-002
Secondary ID
Status Recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date April 2024
Est. completion date April 2031

Study information

Verified date April 2024
Source Encoded Therapeutics
Contact Encoded Patient Advocacy
Phone +1 (650) 398-4301
Email patientadvocacy@encoded.com
Is FDA regulated No
Health authority
Study type Interventional

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


Recruitment information / eligibility

Status Recruiting
Enrollment 22
Est. completion date April 2031
Est. primary completion date April 2027
Accepts healthy volunteers No
Gender All
Age group 6 Months to 35 Months
Eligibility Inclusion Criteria: - Participant must have a predicted loss of function pathogenic or likely pathogenic SCN1A variant. - Participant must have experienced their first convulsive 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 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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
ETX101
ETX101 is a non-replicating, recombinant adeno-associated viral vector serotype 9 (rAAV9) comprising a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A). ETX101 is intended as a one-time intracerebroventricular (ICV) administration.

Locations

Country Name City State
United States Cook Children's Medical Center Fort Worth Texas
United States UCSF Benioff Children's Hospitals San Francisco California

Sponsors (1)

Lead Sponsor Collaborator
Encoded Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Proportions of participants experiencing any treatment-emergent adverse events (AEs), serious adverse events (SAEs), related AEs, AEs with severity Grade = 3, AEs resulting in study discontinuation, and AEs with a fatal outcome. Day 1 through Study Completion
Primary Percent change in monthly countable seizure frequency (MCSF) period, with countable seizures defined as generalized tonic-clonic/clonic, focal motor with clearly observable clinical signs, tonic, or atonic seizures. Between the 8-week baseline period (prior to Day 1) and the 48-week post dosing period (defined as Week 5 through Week 52)
Secondary Proportion of participants with = 90% reduction in monthly countable seizure frequency (MCSF). Between the 8-week baseline period (prior to Day 1) and the 48-week post dosing period (defined as Week 5 through Week 52).
Secondary Change from baseline in the Vineland-3 Expressive Communication raw score at Week 52 Baseline through Week 52
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