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

Launched by ENCODED THERAPEUTICS · Jun 10, 2022

Trial Information

Current as of May 28, 2025

Recruiting

Keywords

Dravet Scn1 A Dee Developmental And Epileptic Encephalopathy Dravet Syndrome Scn1 A Positive Scn1 A+

ClinConnect Summary

This clinical trial, called ENDEAVOR, is studying a new treatment called ETX101 for infants and children with a specific type of epilepsy known as Dravet syndrome, which is linked to a genetic change called SCN1A. The trial is open to children aged 6 to 36 months who have had their first seizure between 3 and 15 months old and are already taking at least one medication to help control their seizures. This trial is looking for participants who have a certain genetic variant that affects how their body responds to treatment.

The study has two parts: the first part will test different doses of ETX101 to see how safe it is, while the second part will compare the new treatment to a placebo (a dummy treatment that doesn’t contain the active drug) to better understand its effectiveness. Families who join the trial can expect regular check-ups and monitoring throughout the study. It's important for participants not to have other serious health issues that could affect the study results. This trial is currently recruiting participants, and it aims to help find a better treatment option for children with Dravet syndrome.

Gender

ALL

Eligibility criteria

  • Inclusion 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.
  • 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.

About Encoded Therapeutics

Encoded Therapeutics is a pioneering biotechnology company focused on developing innovative gene therapies for the treatment of severe neurological disorders. By harnessing the power of its proprietary gene delivery platform, Encoded Therapeutics aims to address unmet medical needs through targeted, durable, and safe interventions. The company’s commitment to advancing therapeutic options is underscored by its robust pipeline of candidates designed to improve the quality of life for patients and their families. Through collaboration with leading researchers and institutions, Encoded Therapeutics strives to bring transformative solutions to the forefront of neurology.

Locations

Fort Worth, Texas, United States

Chicago, Illinois, United States

San Francisco, California, United States

Patients applied

0 patients applied

Trial Officials

Salvador Rico, M.D., Ph.D

Study Director

Encoded Therapeutics

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

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