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Ion Channel Modulator

Part 1 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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up between the 8-week baseline period (prior to day 1) and the 48-week post dosing period (defined as week 5 through week 52)

Awards & highlights

ENDEAVOR Trial 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.Check my eligibility

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).See study design

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.

ENDEAVOR Trial 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.

ENDEAVOR Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ between the 8-week baseline period (prior to day 1) and the 48-week post dosing period (defined as week 5 through week 52)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~between the 8-week baseline period (prior to day 1) and the 48-week post dosing period (defined as week 5 through week 52)

This trial's timeline: 3 weeks for screening, Varies for treatment, and between the 8-week baseline period (prior to day 1) and the 48-week post dosing period (defined as week 5 through week 52) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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.

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.

Secondary outcome measures

Change from baseline in the Vineland-3 Expressive Communication raw score at Week 52

Proportion of participants with ≥ 90% reduction in monthly countable seizure frequency (MCSF).

ENDEAVOR 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 initially evaluate 2 dose levels of ETX101 in approximately 4 participants.

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 in approximately 18 participants. There will be up to 3 cohorts in Part 2. Participants will be randomized 1:1:1 to study treatment (ie, Dose Level 1 or Dose Level 2) or sham procedure with delayed treatment. At the conclusion of Part 1, if the recommendation is made to proceed with a single dose level of ETX101 in Part 2, participants will be randomized 1:1 to study treatment or sham procedure with delayed treatment.

0 / 3Eligibility criteria met

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Who is running the clinical trial?

Encoded TherapeuticsLead Sponsor

5 Previous Clinical Trials

108 Total Patients Enrolled

Salvador Rico, M.D., Ph.DStudy DirectorEncoded Therapeutics

2 Previous Clinical Trials

62 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are potential candidates able to apply for participation in this ongoing trial?

"The information provided by clinicaltrials.gov indicates that patient recruitment for this particular trial is currently closed. Despite being posted on May 1st, 2024 and last updated on February 6th, 2024, the study is not actively enrolling participants. Nonetheless, there are a total of 1449 ongoing studies actively seeking eligible candidates at present."

Answered by AI

What specific criteria must individuals meet to be eligible for enrollment in this clinical study?

"Enrollment is open for 22 individuals aged between 6 and 35 months diagnosed with Dravet syndrome. Eligible participants must possess a predicted disabling pathogenic or likely pathogenic SCN1A variant, have encountered their initial convulsive seizure between ages 3 and 15 months, carry either an official diagnosis of Dravet syndrome or be under the care of a clinician suspecting such a diagnosis, and currently use at least one preventive antiseizure medication."

Answered by AI

Are individuals older than 18 years of age being sought for enrollment in this study?

"Eligible candidates for this research study must be aged between 6 months and 35 months. Notably, there are a total of 371 trials targeting individuals under the age of 18, and 1026 studies focusing on participants above the age of 65."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2024. "A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05419492.

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