Comparison of Microbiome Composition and Biomarkers in Pharmaco Resistant and Pharmaco-Sensitive Epilepsy

NCT ID: NCT04308252

Last Updated: 2021-02-26

Basic Information

• Recruitment Status: WITHDRAWN
• Study Classification: OBSERVATIONAL
• Study Start Date: 2020-03-15
• Study Completion Date: 2022-03-15

Brief Summary

The purpose of this study is to determine stool microbiome composition and biomarkers that are differentially abundant and those that are associated with response to treatment (eg, anticonvulsant drugs).

Detailed Description

Recent research suggests that the intestinal microbiota plays a central role in human health, and may play a role in a variety of central nervous system disorders, including seizure susceptibility. The microbiota has been shown to be associated with changes in factors relevant to neurotransmission, including neurotransmitter signaling, synaptic protein expression, long-term potentiation, and myelination. In addition to the microbiota's potential role in general neurotransmission, research suggests that the gut microbiome may differ between patients with pharmaco resistant and pharmaco-sensitive epilepsies. In support of this idea, ketogenic diet has been used as an alternative treatment for those with pharmaco-resistant epilepsy and there are indications of possible direct interactions of anti-epileptic drugs (AEDs) with the intestinal microbiota.

Conditions

Epilepsy in Children; Pharmaco-Resistant Epilepsy

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Pharmaco-resistant epilepsy (PRE)

Pharmaco resistant patients are defined by seizures despite adequate dosing of ≥2 anticonvulsant drugs.

No interventions assigned to this group

Pharmaco-sensitive epilepsy (PSE)

Pharmaco-sensitive patients are defined by no seizures for 6 months.

No interventions assigned to this group

Sibling control

Sibling of the of the PRE study participants.

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

• Study participant and/or legal representative is willing and able to give informed consent/assent for participation in the study.
• Study participant and/or legal representative is willing and able to comply with all study requirements, in the opinion of the Investigator.
• Study participant is between the ages of 12 and 21, inclusive, at the Screening Visit.
• Study participant resides at home (alone or with family).
• Study participant has a confirmed diagnosis of epilepsy (epilepsy groups only; not applicable to sibling controls).
• Study participant has continued unprovoked seizures despite trials of ≥2 appropriate antiepileptic drugs at therapeutic doses (pharmaco resistant epilepsy group only; not applicable to pharmaco sensitive epilepsy group or sibling controls).

Exclusion Criteria

• Study participant has monogenic epilepsy.
• Study participant has a neurodegenerative disease, mitochondrial disease, metabolic disease, active central nervous system disease, active autoimmune disease, or active inflammatory bowel disease.
• Study participant is on, or has been on within 30 days prior to the Screening Visit, a ketogenic diet, low glycemic index diet, or modified Atkins diet therapy.
• Study participant has had surgery within 3 months of the Screening Visit.
• Study participant is a resident in a group or hospital setting.
• Study participant requires the use of a g-tube or formula feeding.
• Study participant has had antibiotic therapy within 60 days prior to the Screening Visit.
• Study participant has participated in the study of an investigational product within 3 months prior to the Screening Visit (or within 5 half lives of the investigational product, whichever is longer) or is currently participating in a study of an investigational product.

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Boston Children's Hospital

OTHER

Sponsor Role: collaborator

Senda Biosciences

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Boston Children's Hospital

Boston, Massachusetts, United States

Countries

United States

Other Identifiers

E-NEU-EPY-02

Identifier Type: -

Identifier Source: org_study_id