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

NCT04308252 | Withdrawn

• 1 other identifier: E-NEU-EPY-02
• Study Type: observational
• Target: N/A
• Locations: 1 country
• Sites: 1

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).

Conditions

Epilepsy in Children; Pharmaco-Resistant Epilepsy

Keywords

Epilepsy

Outcome Measures

Primary Outcomes (1)

• Pharmaco-sensitive (PSE) vs. pharmaco-resistant epilepsy (PRE) defined by no seizures for 6 months, and seizures despite adequate dosing of ≥2 anticonvulsant drugs, respectively

  Bacterial species and metabolomic biomarkers that are associated with PRE/PSE are determined.

  24 months

Study Arms (3)

Pharmaco-resistant epilepsy (PRE)

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

Pharmaco-sensitive epilepsy (PSE)

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

Sibling control

Sibling of the of the PRE study participants.

Eligibility Criteria

• Age: 1 Year - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

150 study participants are planned, including approximately 50 pharmaco-resistant study participants, approximately 50 pharmaco sensitive study participants, and approximately 50 sibling controls of the pharmaco-resistant study participants.

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Senda Biosciences: lead
• Boston Children's Hospital: collaborator

Study Sites (1)

Boston Children's Hospital

Boston, Massachusetts, 02116, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

Collecting blood and stool

MeSH Terms

Conditions

Epilepsy

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 11, 2020
• First Posted: March 16, 2020
• Study Start: March 15, 2020
• Primary Completion: September 15, 2021
• Study Completion: March 15, 2022
• Last Updated: February 26, 2021

Record last verified: 2021-02

Locations

US (1)