RecruitingPHASE1, PHASE2INTERVENTIONAL

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

This research study, called ENDEAVOR, is testing a new medicine, ETX101, for young children and teenagers with Dravet Syndrome. Dravet Syndrome is a severe form of epilepsy. The main goals are to check if ETX101 is safe and how well it works to control seizures. The study is divided into different parts, involving children from 6 months to just under 18 years old. Some parts of the study will involve all participants receiving the study medicine, while another part will compare the new medicine to a ‘dummy’ treatment (placebo) to see its true effects. This is a common way to test new medicines and helps us understand if the new treatment is genuinely effective.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Encoded Therapeutics

Enrolment target

Start

14 May 2024

Estimated completion

01 Jan 2033

Dravet Syndrome

What is this study about?

This study, called ENDEAVOR, is investigating a new medicine named ETX101 for children diagnosed with Dravet Syndrome. Dravet Syndrome is a rare and severe type of epilepsy that usually starts in the first year of life. It causes frequent and prolonged seizures, and often leads to developmental challenges.

The main aim of this research is to see if ETX101 is safe for children with Dravet Syndrome and if it can help reduce the number or severity of their seizures. The study will look at children across different age groups, specifically: very young children aged 6 to 35 months; older children and teenagers aged 4 to 17 years; and another group of children from 6 to 47 months old. By studying different age ranges, the researchers hope to understand best how the medicine works in various stages of childhood.

The study has different parts. In some parts, all children will receive the new medicine and their health will be closely monitored. In another part, children will be randomly chosen to receive either ETX101 or a 'dummy' treatment (called a placebo). This helps researchers fairly compare how well ETX101 works compared to no active treatment, without anyone knowing who received what until the study is over. This is a standard and important step in testing new medicines to ensure accurate results.

Key takeaways

Tests a new medicine (ETX101) for Dravet Syndrome.
Aims to check safety and seizure control.
Includes babies and children aged 6 months to 17 years.
Some participants will receive the new medicine, others a dummy treatment.
Close medical monitoring and care provided.
You can withdraw your child at any time.

Who may be eligible?

To join this study, children need to meet specific criteria. They must be between 6 months and just under 18 years old, depending on the study part they might join. A key requirement is that they must have a confirmed genetic change in a gene called SCN1A, which is known to cause Dravet Syndrome. They should also have experienced their first seizure between 3 and 15 months of age, and have a clear diagnosis of Dravet Syndrome, or their doctor must have a strong suspicion they have it. Also, they should already be taking at least one medicine to prevent seizures.

There are also reasons why a child might not be able to join. For example, if they have other genetic conditions or health problems that might make it difficult to tell if ETX101 is working properly for their Dravet Syndrome. Children with certain brain abnormalities, or who have specific medical devices like a shunt in their brain, or who have recently had a seizure-free period of a month, would not be able to participate. Also, children who have received certain other types of experimental treatments or are already in another clinical trial cannot join this one. Finally, significant liver disease would prevent participation.

Quick self-check

Is my child between 6 months and 17 years old?
Does my child have a confirmed SCN1A gene change?
Did my child have their first seizure between 3 and 15 months of age?
Is my child currently taking at least one seizure prevention medicine?
Does my child NOT have any other major genetic conditions or brain abnormalities?
Has my child NOT had any experimental gene or cell therapy before, or been seizure-free for a month recently?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

The details of what taking part involves would be fully explained by the study team. Generally, joining a clinical study means you and your child will have regular visits to the clinic. These visits will involve health checks, assessments of your child's seizures, and potentially blood tests or other procedures to monitor their health and how the study medicine is affecting them. The study medicine, ETX101, would be given under careful medical supervision. The total length of your child's participation in the study, including follow-up, would vary depending on which part of the study they are enrolled in, but this would be made clear to you before you agree to take part. You would also be asked to keep a record of your child's seizures.

Potential risks and benefits

Participating in a study like this could potentially offer benefits, such as access to a new medicine that isn't yet widely available, which might help to control your child's seizures. Your child would also receive close medical attention and monitoring. However, there are also potential risks; ETX101 is an experimental medicine, and like all medicines, it might have side effects that are not yet fully known. There could also be discomfort from tests or procedures. The medical team will explain all known and potential risks in detail. It's very important to remember that you have the right to withdraw your child from the study at any time, for any reason, without it affecting their regular medical care.

Locations (13)

UCSF Benioff Children's Hospitals
San Francisco, United States· Recruiting
Colorado Children's Hospital
Aurora, United States· Not yet recruiting
Nicklaus Children's Hospital
Miami, United States· Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, United States· Recruiting
Boston Children's Hospital
Boston, United States· Not yet recruiting
Mott Children's Hospital
Ann Arbor, United States· Not yet recruiting
Mayo Clinic
Rochester, United States· Not yet recruiting
Nationwide Children's Hospital
Columbus, United States· Not yet recruiting
Oregon Health and Science University (OSHU)
Portland, United States· Recruiting
Cook Children's Medical Center
Fort Worth, United States· Recruiting
The Royal Children's Hospital
Melbourne, Australia· Recruiting
Queen Elizabeth Hospital
Glasgow, United Kingdom· Recruiting

+1 more sites — see the official record for the full list.

Common questions

What is Dravet Syndrome?

Dravet Syndrome is a severe form of epilepsy that usually starts in infancy, causing frequent and prolonged seizures and often affecting development.

What is ETX101?

ETX101 is a new, experimental medicine being tested to see if it can help children with Dravet Syndrome control their seizures.

Will my child definitely get the new medicine?

It depends on which part of the study your child joins. In some parts, they will; in others, they might be randomly assigned to receive either ETX101 or a 'dummy' treatment (placebo).

How long does the study last?

The exact duration depends on the specific part of the study your child is in. The research team will provide full details before you decide to participate.

Can I take my child out of the study if we change our minds?

Yes, you are free to withdraw your child from the study at any time, for any reason, and this will not affect their future medical care.

How to find out more

Encoded Patient Advocacy

patientadvocacy@encoded.com +1 (650) 398-4301 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.