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Ion Channel Modulator

ETX101 for Dravet Syndrome (ENDEAVOR Trial)

Phase 1 & 2

Recruiting

Research Sponsored by Encoded Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Must not have

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 previously received gene or cell therapy

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Summary

"This trial is testing a new drug called ETX101 in young children with Dravet syndrome caused by a specific gene mutation. The study has two parts: the first part will test different doses of

Who is the study for?

This trial is for infants and children aged 6 to less than 36 months with a confirmed genetic variant linked to Dravet Syndrome. They must have had their first seizure between 3-15 months old, be diagnosed or suspected of having Dravet Syndrome by a clinician, and be on at least one anti-seizure medication.

What is being tested?

The study tests ETX101's safety and effectiveness in young patients with SCN1A-positive Dravet syndrome. It has two parts: the first part increases doses to find safe levels (open-label), and the second part compares ETX101 against a sham treatment without participants knowing which they're getting (double-blind).

What are the potential side effects?

As this is an early phase trial for ETX101, specific side effects are being studied but may include typical drug reactions such as irritation at the injection site, fever, changes in appetite or sleep patterns, and potential interactions with existing medications.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a genetic mutation in SCN1A linked to my condition.

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My first seizure happened when I was between 3 and 15 months old.

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I am taking medication to prevent seizures.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am taking or have taken seizure medications not recommended for Dravet syndrome.

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I have had gene or cell therapy before.

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I have a brain abnormality confirmed by MRI or CT scan.

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I have a serious liver condition.

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I haven't had a seizure in the last 4 weeks.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Part 1Experimental Treatment1 Intervention

Part 1 will follow an open-label, rule-based, dose-escalation design and will evaluate 3 dose levels of ETX101.

Group II: Part 2Placebo Group1 Intervention

Part 2 is a dose-selection study, which will follow a double-blind (up through Week 52), randomized, sham delayed-treatment control design. There will be up to 3 cohorts in Part 2. If it is determined two dose levels of ETX101 will be evaluated in Part 2, participants will be randomized 1:1:1 to study treatment or sham procedure with delayed treatment. If Part 2 proceeds with a single dose level of ETX101, participants will be randomized 2:1 to study treatment or sham procedure with delayed treatment.

0 / 8Eligibility criteria met