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Natural History Study of Infants and Children With Developmental and Epileptic Encephalopathies | Clinical Trials | Clareo Health

TERMINATED

Natural History Study of Infants and Children With Developmental and Epileptic Encephalopathies

ENVISION: Natural History Study of Infants and Children With Developmental and Epileptic Encephalopathies

NCT04537832•Encoded Therapeutics

View on ClinicalTrials.gov

Summary

This is a multicenter, prospective, 2-year observational study in infants and children with developmental and epileptic encephalopathies (DEEs). The DEE currently being investigated is SCN1A-positive Dravet Syndrome.

Detailed Description

This prospective, longitudinal, natural history master protocol has been designed to define the seizure, neurodevelopmental, and behavioral characteristics of SCN1A-positive Dravet Syndrome in infants and children between 6 and 60 months. It will also explore the impact of the disease on the participant's parent/caregiver quality of life (QoL) and healthcare resource utilization (HCRU).

Eligibility

Age

0 - 5 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•Aged between 6 months and 60 months.
•Confirmed SCN1A mutation.
•Normal development prior to onset of first seizure as defined by the Centers for Disease -Control and Prevention (CDC 2019).
•Onset of seizures between age 3 and 15 months, inclusive.

Exclusion Criteria

•Copy number variant of SCN1A, including SCN1A microdeletion, if affecting other genes.
•SCN1A mutation present on both alleles.
•Known pathogenic or clinically suspected mutation in a seizure-associated gene besides SCN1A.
•Confirmed mutation in a gene besides SCN1A that is known to increase the severity of the seizure phenotype.
•Known gain-of-function genetic mutation, as defined by functional studies, including p.Thr226Met.
•History of notable developmental deficit that was evident prior to seizure onset.
•Known central nervous system structural abnormality as found on magnetic resonance imaging or computed tomography scan of brain.
•Currently taking or has taken for 6 or more consecutive weeks anti-seizure medications (ASMs) at a therapeutic dose that are contraindicated in SCN1A-positive Dravet Syndrome, including sodium channel blockers.
•Known concomitant genetic mutation or clinical comorbidity that potentially confounds typical Dravet phenotype.

Locations (16)

Children's Hospital Los Angeles

Los Angeles, California, United States

UCSF Benioff Children's Hospital

San Francisco, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Nicklaus Children's Hospital

Miami, Florida, United States

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Northeast Regional Epilepsy Group

Hackensack, New Jersey, United States

Abigail Wexner Research Institute at Nationwide Children's Hospital

Columbus, Ohio, United States

Oregon Health and Science University

Portland, Oregon, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Le Bonheur Children's Hospital

Memphis, Tennessee, United States

Key Dates

Start Date

January 18, 2021

Primary Completion Date

March 31, 2023

Completion Date

March 31, 2023

Last Updated

June 12, 2023

Enrollment

ACTUAL participants

Conditions

Dravet Syndrome

Interventions

No Intervention

OTHER

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

NCT05419492

Dravet Syndrome

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RECRUITINGPHASE3

A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome

NCT04462770

Dravet Syndrome

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RECRUITINGPHASE4

Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under 24 Months of Age

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: June 12, 2023Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Natural History Study of Infants and Children With Developmental and Epileptic Encephalopathies

Inclusion Criteria

Exclusion Criteria

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

A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome

Assessment of Safety of the Use of Fenfluramine in Children With Dravet Syndrome Under 24 Months of Age

A Study to Investigate the Transition of Children From 'Artisanal" Cannabidiol (CBD) to Epidiolex

A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome

Bioavailability of Stiripentol After Single Oral Dose of Capsule vs Suspension in Healthy Subjects (STILIQ)