Molecular Genetic Mechanisms of Infantile Epilepsies and the Impact of Genetic Diagnosis

NCT ID: NCT06701084

Last Updated: 2024-11-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

NA

Total Enrollment

600 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-09-02

Study Completion Date

2029-11-30

Brief Summary

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The goal of this study is to discover new genetic causes of infantile epilepsies and evaluate the impact of these discoveries on infants with epilepsy and their families.

Detailed Description

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Infantile epilepsies are common, affecting 1 in 1000 infants, and are associated with significant morbidity, mortality, healthcare costs, and caregiver burden. Although most infantile epilepsies are believed to have genetic causes, most infants with epilepsy remain genetically "unsolved" and the full genetic landscape of infantile epilepsies is unknown, which limits our ability to develop precision therapies and ultimately improve outcomes for this vulnerable population. This study aims to discover new genetic causes of infantile epilepsies and evaluate the impact of these discoveries on infants with epilepsy and their families, contributing to knowledge that will inform our scientific understanding of normal and abnormal brain development and guide clinical care and implementation of precision medicine for infants with epilepsy.

Conditions

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Neonatal Epilepsy Infantile Epilepsy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

HEALTH_SERVICES_RESEARCH

Blinding Strategy

NONE

Study Groups

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Genomic Sequencing

All enrolled infants receive the intervention (genomic sequencing, including rapid genome sequencing). Comprehensive genomic analyses will be performed to identify genetic diagnoses. Genetic results will be returned to families and infants will be followed until 2.5 years old to evaluate the impact of genetic diagnosis using quantitative validated outcome measures and qualitative parent interviews.

Group Type EXPERIMENTAL

Genomic Sequencing

Intervention Type GENETIC

Genomic sequencing data will be comprehensively analyzed for pathogenic variants that explain the participants epilepsy.

Interventions

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Genomic Sequencing

Genomic sequencing data will be comprehensively analyzed for pathogenic variants that explain the participants epilepsy.

Intervention Type GENETIC

Eligibility Criteria

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Inclusion Criteria

* Seizure onset at less than 12 months of age
* Enrollment within 6 weeks of seizure-related presentation
* Patient at Boston Children's Hospital

Exclusion Criteria

* Simple febrile seizures
* Acute provoked seizures (e.g., due to sepsis, hemorrhage, electrolyte abnormality, cerebral infarction, hypoxic ischemic encephalopathy, non-accidental injury)
* Genetic or acquired cause of epilepsy already identified, including brain magnetic resonance imaging findings consistent with a specific genetic etiology (e.g., tuberous sclerosis complex)
* Deceased prior to enrollment


\- Not the legal guardian of the eligible infant
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Boston Children's Hospital

OTHER

Sponsor Role lead

Responsible Party

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Alissa D'Gama

Instructor of Pediatrics

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Alissa M D'Gama, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital

Locations

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Boston Children's Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Beth R Sheidley, MS

Role: CONTACT

8572185533

Facility Contacts

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Beth R Sheidley, MS

Role: primary

8572185533

Alissa M D'Gama, MD, PhD

Role: backup

Other Identifiers

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1K23NS140397

Identifier Type: NIH

Identifier Source: org_study_id

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