Lennox Gastaut SyndromeSOM-LNCT05374824
Comparative Effectiveness of Palliative Surgery Versus Additional Anti-Seizure Medications for Lennox-Gastaut Syndrome
An observational study of Lennox Gastaut Syndrome, run by Ann & Robert H Lurie Children's Hospital of Chicago at 4 sites in United States.
Trial summary
Lennox-Gastaut syndrome is a serious and rare form of epilepsy that begins in infancy and early childhood. Seizures and their consequences need medical attention, emergency encounters, and hospitalizations. Seizures disrupt home life for the patient and for family. Read more about this study
- Who is it for?
- Ages Up to 26 years
- Conditions the study is for
- Lennox Gastaut Syndrome
- How many people
- 4,680 participants
- Where
- 4 sitesUnited States
- Study dates
- 1 Apr 2022to 31 Dec 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 Up to 26 years
- All sexes
- Child, Adult
Study population: For Aim 1a, 928 patients up to age 30 years with a chart-review validated diagnosis of Lennox-Gastaut Syndrome between 1/1/2016 and 12/31/2021 will be identified from 7 principal pediatric hospitals. For Aim 1b, a subset of half of those 928 patients (N=464) will be recruited. Analyses for Aim 2 will be based on ≥4000 patients with LGS ICD-10 codes from 18 pediatric hospitals (representing 5 PCORnet CRNs).
Who may be able to join
- Patients with the diagnosis of Lennox Gastaut syndrome with medical records at the seven plus eleven pediatric centers
Who cannot join
- Patients without Lennox Gastaut Syndrome
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
C.S. Mott Children's Hospital
Ann Arbor, Michigan, 48109
Madelyn Eckel, 734-998-0278, eckelma@med.umich.edu
Laurel Reed, MD
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Megan Votoupal, 312-227-0684, mvotoupal@luriechildrens.org
Sandi Lam, MD
- Recruiting
Nationwide Children's
Columbus, Ohio, 43205
Evelynne Wentzel, evelynne.wentzel@nationwidechildrens.org
Anup Patel, MD
- Recruiting
St. Louis Children's Hospital
St Louis, Missouri, 63110
Janette Coble, 314-454-6120, janette.coble@wustl.edu
About this study
Official title: Comparative Effectiveness of Palliative Surgery Versus Additional Anti-Seizure Medications for Lennox-Gastaut Syndrome - PCORI
Lennox-Gastaut syndrome is a serious and rare form of epilepsy that begins in infancy and early childhood. Seizures and their consequences need medical attention, emergency encounters, and hospitalizations. Seizures disrupt home life for the patient and for family. Lennox-Gastaut syndrome is typically accompanied by disabilities in motor, communication, eating, and other skills needed for daily function.
Lennox-Gastaut syndrome (LGS) has no cure. Although current treatments may help reduce the number of seizures, none are expected to eliminate them entirely; these treatments are palliative. The main treatments include anti-seizure medications and some surgical approaches, including the implantation of a vagus nerve stimulator (a pacemaker-like generator implanted in the chest wall and programmed by a physician to stimulate the vagus nerve in the neck) and corpus callosotomy (cutting through the band of fibers that connect the two sides of the brain). While both types of treatment (medications and surgeries) produce some benefit by reducing how often the seizures occur, both also have some risks. All medications can, in some patients, produce moderate to severe side effects. This is true of anti-seizure medications. Most patients with LGS take several anti-seizure medications at a time. Surgeries can also have associated risks and is additionally stressful for parents and family members. Currently, there is no strong evidence to support parents and physicians in deciding which type of treatment (more medicines or surgery) will be most successful for a child with LGS, and whether one or the other approach may lessen the toll that seizures take on a child's development and ability to function.
Read more
This study has two components. It will engage a network of seven pediatric hospitals in the United States where children with Lennox-Gastaut syndrome are cared for and determine whether seizure-related emergency department visits and hospitalizations are more likely to be reduced following the use of additional medications or adding palliative surgery to existing medications. The investigators will determine whether medical versus surgical treatment is more likely to lessen some of the developmental and functional difficulties that affect patients with LGS. The study will also determine whether starting therapies at a younger versus older age makes a difference.
The second component of the study will provide a description of the use of surgical versus medical treatment approaches across 18 pediatric hospitals in the United States (seven plus 11 centers). The investigators will describe how treatments differ across hospitals and over time.
The results from this study will help parents and providers make more informed choices about treatment for children with Lennox-Gastaut syndrome and will highlight areas for improvement in providing the best possible health care for this severe, lifelong disorder.
Read the detailed description
Background and Significance: Lennox-Gastaut Syndrome (LGS) is a rare developmental-epileptic encephalopathy characterized by life-long refractory seizures which result in frequent utilization of emergency healthcare resources and also contribute to the severe developmental and functional impairment ubiquitously seen in affected patients. Current treatment pathways are the addition of anti-seizure medication treatment or palliative surgical procedures (including neurostimulation), both of which have strong evidence supporting their effectiveness in reducing seizure frequency, but without cures. No evidence exists about the comparative effectiveness of these two pathways. Until such evidence is developed, parents and physicians will continue to struggle with difficult and daunting decisions about whether to add anti-seizure medications or to pursue palliative surgery, and when to do so.
Study Aims: Determine the comparative effectiveness of adding palliative surgery versus adding an LGS-approved medication (Aim 1a) for decreasing utilization of seizure-related emergency health care and (Aim 1b) for limiting the impairment of functional abilities. (Aim 2) Describe the frequency and patterns of utilization of the two therapy pathways at 18 PCORnet sites for variation over time and across sites as well as by patient features - age, medical fragility, race, ethnicity, and insurance type. In addressing these aims, the study will assess and improve PCORnet infrastructure and will enhance methods for future studies of LGS and other rare epilepsies.
Study Description: (Aim 1a) Observational retrospective cohort study of emergency health-care utilization and (Aim 1b) cross sectional study of the impact of treatment pathways on functional outcomes. (Aim 2) Retrospective open cohort study of frequency and patterns (by race, ethnicity, insurance, age) of utilization of the two treatment pathways.
- Conditions
- Lennox Gastaut Syndrome
- Keywords
- epilepsy, seizure
- Medical subject headings
- Lennox Gastaut Syndrome, Epilepsy, Seizures
What is being measured
The primary outcome is the main question the study is designed to answer.
Seizure-related emergency health care utilization
Emergency health-care utilization for Aim 1a will be defined as the frequency per year of seizure-related emergency department visits and separately seizure-related inpatient admissions in the two years postintervention (surgery or medical), with adjustment for the rate during the year prior to intervention.
Measured: 2 years
Secondary outcomes (1)
Parent-reported Clinical Outcome Assessments
Parent-reported qualitative input through descriptive assessment. The measures will be assessed once.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 May 2022
- Study start1 Apr 2022
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Lennox Gastaut SyndromeStudies 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
- Retrospective
- Enrolment
- 4,680 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
- Sponsor type
- University, hospital or other
- Collaborators
- Patient-Centered Outcomes Research Institute, Nationwide Children's Hospital, University of Colorado, Denver, University of Michigan, University of Pittsburgh, Seattle Children's Hospital, St. Louis Children's Hospital, University of Cincinnati, Nemours Children's Health System, Stanford University, Monroe Carell Jr. Children's Hospital at Vanderbilt, University of North Carolina, Duke University, Indiana University, Johns Hopkins University, Nicklaus Children's Hospital f/k/a Miami Children's Hospital
- Responsible party
- Principal investigator, Sandi Lam, Ann & Robert H Lurie Children's Hospital of Chicago
- Investigators
- Sandi Lam, MD MBA, Principal investigator, Ann & Robert H Lurie Children's Hospital of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05374824
- Sponsor's protocol ID
- 2021-4532
- Other IDs
- RD-2020C2-20356
- Acronym
- SOM-L
- Status last verified
- Aug 2026
- First submitted
- 6 May 2022
- Last updated
- 6 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Lam S, Rosenman M, Dixon-Salazar T, Knupp KG, Thio LL, Abel TJ, Welch WP, Reed L, Randle SC, Garcia-Sosa R, Hauptman JS, Foster CC, Alpern ER, Zhang L, Villalba N, Carroll M, Patel AD. Comparative effectiveness of epilepsy surgery versus additional anti-seizure medications for Lennox-Gastaut syndrome: study protocol for a multicenter, mixed-methods study. Front Neurol. 2025 Jun 18;16:1569551. doi: 10.3389/fneur.2025.1569551. eCollection 2025.
PubMed 40606139
Study at a glance
From the registry record, last updated by the sponsor on 6 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparative Effectiveness of Palliative Surgery Versus Additional Anti-Seizure Medications for Lennox-Gastaut Syndrome - PCORI
- ClinicalTrials.gov ID
- NCT05374824
- Acronym / sponsor ID
- SOM-L / 2021-4532
- Conditions
- Lennox Gastaut Syndrome
- Keywords
- epilepsy, seizure
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 4,680 participants (estimated)
- Lead sponsor
- Ann & Robert H Lurie Children's Hospital of ChicagoUniversity, hospital or other
- Collaborators
- Patient-Centered Outcomes Research Institute, Nationwide Children's Hospital, University of Colorado, Denver, University of Michigan, University of Pittsburgh, Seattle Children's Hospital, St. Louis Children's Hospital, University of Cincinnati, Nemours Children's Health System, Stanford University, Monroe Carell Jr. Children's Hospital at Vanderbilt, University of North Carolina, Duke University, Indiana University, Johns Hopkins University, Nicklaus Children's Hospital f/k/a Miami Children's Hospital
- Study start
- 1 Apr 2022 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 16 May 2022 (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.
Lennox-Gastaut syndrome is a serious and rare form of epilepsy that begins in infancy and early childhood. Seizures and their consequences need medical attention, emergency encounters, and hospitalizations. Seizures disrupt home life for the patient and for family. Lennox-Gastaut syndrome is typically accompanied by disabilities in motor, communication, eating, and other skills needed for daily function.
Lennox-Gastaut syndrome (LGS) has no cure. Although current treatments may help reduce the number of seizures, none are expected to eliminate them entirely; these treatments are palliative. The main treatments include anti-seizure medications and some surgical approaches, including the implantation of a vagus nerve stimulator (a pacemaker-like generator implanted in the chest wall and programmed by a physician to stimulate the vagus nerve in the neck) and corpus callosotomy (cutting through the band of fibers that connect the two sides of the brain). While both types of treatment (medications and surgeries) produce some benefit by reducing how often the seizures occur, both also have some risks. All medications can, in some patients, produce moderate to severe side effects. This is true of anti-seizure medications. Most patients with LGS take several anti-seizure medications at a time. Surgeries can also have associated risks and is additionally stressful for parents and family members. Currently, there is no strong evidence to support parents and physicians in deciding which type of treatment (more medicines or surgery) will be most successful for a child with LGS, and whether one or the other approach may lessen the toll that seizures take on a child's development and ability to function.
This study has two components. It will engage a network of seven pediatric hospitals in the United States where children with Lennox-Gastaut syndrome are cared for and determine whether seizure-related emergency department visits and hospitalizations are more likely to be reduced following the use of additional medications or adding palliative surgery to existing medications. The investigators will determine whether medical versus surgical treatment is more likely to lessen some of the developmental and functional difficulties that affect patients with LGS. The study will also determine whether starting therapies at a younger versus older age makes a difference.
The second component of the study will provide a description of the use of surgical versus medical treatment approaches across 18 pediatric hospitals in the United States (seven plus 11 centers). The investigators will describe how treatments differ across hospitals and over time.
The results from this study will help parents and providers make more informed choices about treatment for children with Lennox-Gastaut syndrome and will highlight areas for improvement in providing the best possible health care for this severe, lifelong disorder.
Detailed description
Background and Significance: Lennox-Gastaut Syndrome (LGS) is a rare developmental-epileptic encephalopathy characterized by life-long refractory seizures which result in frequent utilization of emergency healthcare resources and also contribute to the severe developmental and functional impairment ubiquitously seen in affected patients. Current treatment pathways are the addition of anti-seizure medication treatment or palliative surgical procedures (including neurostimulation), both of which have strong evidence supporting their effectiveness in reducing seizure frequency, but without cures. No evidence exists about the comparative effectiveness of these two pathways. Until such evidence is developed, parents and physicians will continue to struggle with difficult and daunting decisions about whether to add anti-seizure medications or to pursue palliative surgery, and when to do so.
Study Aims: Determine the comparative effectiveness of adding palliative surgery versus adding an LGS-approved medication (Aim 1a) for decreasing utilization of seizure-related emergency health care and (Aim 1b) for limiting the impairment of functional abilities. (Aim 2) Describe the frequency and patterns of utilization of the two therapy pathways at 18 PCORnet sites for variation over time and across sites as well as by patient features - age, medical fragility, race, ethnicity, and insurance type. In addressing these aims, the study will assess and improve PCORnet infrastructure and will enhance methods for future studies of LGS and other rare epilepsies.
Study Description: (Aim 1a) Observational retrospective cohort study of emergency health-care utilization and (Aim 1b) cross sectional study of the impact of treatment pathways on functional outcomes. (Aim 2) Retrospective open cohort study of frequency and patterns (by race, ethnicity, insurance, age) of utilization of the two treatment pathways.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
Who may qualify
- Ages
- Up to 26 years
- Sex
- All
- Age groups
- Child, Adult
- Study population
- For Aim 1a, 928 patients up to age 30 years with a chart-review validated diagnosis of Lennox-Gastaut Syndrome between 1/1/2016 and 12/31/2021 will be identified from 7 principal pediatric hospitals. For Aim 1b, a subset of half of those 928 patients (N=464) will be recruited. Analyses for Aim 2 will be based on ≥4000 patients with LGS ICD-10 codes from 18 pediatric hospitals (representing 5 PCORnet CRNs).
Key inclusion criteria (1 listed)
- Patients with the diagnosis of Lennox Gastaut syndrome with medical records at the seven plus eleven pediatric centers
Key exclusion criteria (1 listed)
- Patients without Lennox Gastaut Syndrome
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
Seizure-related emergency health care utilization
Time frame: 2 years
Emergency health-care utilization for Aim 1a will be defined as the frequency per year of seizure-related emergency department visits and separately seizure-related inpatient admissions in the two years postintervention (surgery or medical), with adjustment for the rate during the year prior to intervention.
1 secondary outcome
Parent-reported Clinical Outcome Assessments
Time frame: 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Aug 2026.
- Derived from this study: PubMed 40606139Lam S, Rosenman M, Dixon-Salazar T, Knupp KG, Thio LL, Abel TJ, Welch WP, Reed L, Randle SC, Garcia-Sosa R, Hauptman JS, Foster CC, Alpern ER, Zhang L, Villalba N, Carroll M, Patel AD. Comparative effectiveness of epilepsy surgery versus additional anti-seizure medications for Lennox-Gastaut syndrome: study protocol for a multicenter, mixed-methods study. Front Neurol. 2025 Jun 18;16:1569551. doi: 10.3389/fneur.2025.1569551. eCollection 2025.
- PCORI websiteLink provided by the sponsor.
- Project PageLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Not yet recruitingPhase 4
A Study to Investigate the Effect of Gradual Titration to Optimize Cannabidiol Treatment in Adults With LGS
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A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)
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An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome
Lennox Gastaut Syndrome
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Clinical Utility of Reduced EEG Home Monitoring in Fenfluramine Titration for Dravet and LGS
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Not yet recruiting
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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.
Sandi Lam, MD MBA principal investigator
Ann & Robert H Lurie Children's Hospital of Chicago
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Lennox Gastaut Syndrome, run by Ann & Robert H Lurie Children's Hospital of Chicago at 4 sites 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 1 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 6 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sandi Lam, MD MBA (312 227 4220, slam@luriechildrens.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Seizure-related emergency health care utilization (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05374824, which also lists 1 linked publication.
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 6 Aug 2026.