Epilepsy in ChildrenNCT04308252
Comparison of Microbiome Composition and Biomarkers in Pharmaco Resistant and Pharmaco-Sensitive Epilepsy
An observational study of Epilepsy in Children and Pharmaco-Resistant Epilepsy, run by Senda Biosciences at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Epilepsy in Children, Pharmaco-Resistant Epilepsy
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Mar 2020to 15 Mar 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
- 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).
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Boston Children's Hospital
Boston, Massachusetts, 02116
About this study
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).
Read the 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
- Keywords
- Epilepsy
- Medical subject headings
- Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Mar 2020
- Study start15 Mar 2020 (estimated)
- Primary completion15 Sep 2021 (estimated)
- Study completion15 Mar 2022 (estimated)
Why it stopped: Sponsor no longer pursuing Epilepsy development.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epilepsy in ChildrenStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Senda Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Boston Children's Hospital
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04308252
- Sponsor's protocol ID
- E-NEU-EPY-02
- Status last verified
- Feb 2021
- First submitted
- 11 Mar 2020
- Last updated
- 26 Feb 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 26 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparison of Microbiome Composition and Biomarkers in Pharmaco Resistant and Pharmaco-Sensitive Epilepsy
- ClinicalTrials.gov ID
- NCT04308252
- Acronym / sponsor ID
- E-NEU-EPY-02
- Conditions
- Epilepsy in Children, Pharmaco-Resistant Epilepsy
- Keywords
- Epilepsy
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: Sponsor no longer pursuing Epilepsy development.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Senda BiosciencesIndustry (pharma, biotech, devices)
- Collaborators
- Boston Children's Hospital
- Study start
- 15 Mar 2020 (estimated)
- Primary completion
- 15 Sep 2021 (estimated)
- Study completion
- 15 Mar 2022 (estimated)
- First posted
- 16 Mar 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (6 listed)
- 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).
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
Time frame: 24 months
Bacterial species and metabolomic biomarkers that are associated with PRE/PSE are determined.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Feb 2021.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Epilepsy in Children and Pharmaco-Resistant Epilepsy, run by Senda Biosciences at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 26 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Feb 2021.