Tuberous Sclerosis ComplexNCT04198181
The Effectiveness and Safety of Resective Epilepsy Surgery for TRE
An observational study of Tuberous Sclerosis Complex and Epilepsy, run by Beijing Children's Hospital at 1 site in China.
Trial summary
A prospective cohort studies to identify clinical seizure control, cognitive changes, and safety in resective epilepsy surgery in patients with TSC-related drug-resistant epilepsy. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Tuberous Sclerosis Complex, Epilepsy
- What is being tested?
- Surgery
- Type of treatment
- Surgery or procedure
- How many people
- 200 participants
- Where
- 1 siteChina
- Study dates
- 12 Dec 2019to 15 Mar 2027
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients from participating medical institutions
Who may be able to join
- 2 years old and above, no gender restriction, TSC gene monitoring with or without abnormality
- Diagnosis of tuberous sclerosis- related drug-resistant epilepsy
- Epilepsy course for more than 1 year
- Patients who have taken 3 or more reasonable choices with appropriate and tolerable antiepileptic drugs (excluding mTOR inhibitors and traditional Chinese medicine and prescriptions) had seizures more than 12 times in the 3 months before enrollment
- The family members agreed to enroll and signed the informed consent.
Who cannot join
- Obvious renal angiomyolipoma, pulmonary lymphoma leiomyomatosis, and subventricular giant cell astrocytoma
- Abnormal heart, lung, liver, and kidney functions and coagulation function
- Preoperative evaluation, it is considered that no surgical treatment is needed
- The patient received other craniocerebral surgical treatment within 1 year during the follow-up period
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Clinical Evaluation of the Effectiveness and Safety of Resective Epilepsy Surgery for Tuberous Sclerosis Complex Related Epilepsy
A prospective cohort studies to identify clinical seizure control, cognitive changes, and safety in resective epilepsy surgery in patients with TSC-related drug-resistant epilepsy.
Read the detailed description
Two hundred candidates are recruited and assigned to the control and surgery groups with 100 cases in each group, respectively. All patients will accept first-stage pre-enrollment evaluations and patients in surgery group will accept second-stage invasive evaluation.
All patients will be advised to visit the hospitals for examination each year after enrollment. Seizure outcomes will be assessed with reference to patients' seizure diaries and caregivers' oral statements by both neurosurgeons and neurologists.
All adverse effects will be classified according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0 (2017).
Statistical analyses were conducted using SPSS (version 26.0, IBM, USA), Stata (version 16.0, Stata Corp LLC, USA), and R (version 4.3.3, R Foundation for Statistical Computing, Austria). Outcomes were presented according to data type as percentages, mean ± standard deviation, or median (interquartile range, IQR).
Multiple imputation was employed to address missing data for IQ and QOL at enrollment and the 2-year follow-up. A sensitivity analysis was conducted to assess the robustness of the multiple imputation results. The "mi impute regress" statement in Stata facilitated the multiple imputations. 20 T-tests were utilized for comparing continuous variables, with results reported as mean ± standard deviation. The Mann-Whitney U test was applied for non-normally distributed continuous variables, presenting results as median values and IQRs. Chi-square and Fisher's exact tests were used for univariate analyses. A significance level of p < 0.05 was established.
To account for differences in covariates between the surgery and medicine groups, propensity score matching (PSM) was performed. Based on PSM, Kaplan-Meier curves and the log-rank test were utilized to analyze time-to-seizure recurrence differences between the surgery and medicine groups. Additionally, multivariable Cox proportional hazards regression was applied to predict risk factors for postoperative seizure recurrence within a 51-month follow-up period after PSM.
- Conditions
- Tuberous Sclerosis Complex, Epilepsy
- Being tested
- Surgery
- Keywords
- epilepsy, tuberous sclerosis complex, resective epilepsy surgery
- Medical subject headings
- Tuberous Sclerosis, Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SurgeryProcedure or surgery
Resective surgery included lobectomy (partial or total brain lobe resection), tuberectomy (epileptogenic tuber resection), and tuberectomy plus (resection of epileptogenic tuber and perituberal gyri). Tuberectomy was typically performed on the epileptogenic tuber within or near an eloquent area. Lobectomy was performed in patients with large epileptogenic tubers in the brain lobes. Multiple lobectomies, tuberectomies, tuberectomies plus, and lobectomy combined with tuberectomy/tuberectomies plus were further performed in patients with multiple epileptogenic tubers.
| Study group | Role | What they receive |
|---|---|---|
| Control Group | Patients in the surgery group will accept exclusive rational medicine therapy. | |
| Surgery Group | Patients in the surgery group will undergo resective surgery combined with rational medicine therapy. Receives: Procedure: Surgery |
What is being measured
The primary outcome is the main question the study is designed to answer.
% of patients with ILAE grading
The grading made by ILAE for the classification of outcome with respect to epileptic seizures following epilepsy surgery. It contains a total of 6 levels. The higher the level, the worse the result. We will count the percentage of patients at each level.
Measured: 3 years
Secondary outcomes (2)
IQ
Wechsler Preschool and Primary Scale of Intelligence（WPPSI-III），Wechsler Intelligence Scale for Children (WISC-IV) and Wechsler Adult Intelligence Scale (WAIS-IV)，3 scales will be used for different ages to measure IQ. Higher scores mean better outcomes.
Measured: 3 years
Quality of Life: QOLIE-31
QOLIE-31 is suitable for patients 14 years and older. QOLCE-76 is applied to patients aged 2-13 by parents.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2019
- Study start12 Dec 2019
- Primary completion15 Mar 2027 (estimated)
- Study completion15 Mar 2027 (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 Tuberous Sclerosis ComplexStudies 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
- 200 participants (actual)
Sponsor and investigators
- Lead sponsor
- Beijing Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Shenzhen Children's Hospital, Tiantan Hospital, Capital Medical University, Peking University People's Hospital, Guangdong 999 Brain Hospital, Second Affiliated Hospital of Army Medical University, Shandong Provincial Hospital, West China Hospital, Henan Provincial People's Hospital, Shanghai Children's Hospital, The First Medical Center of Chinese PLA General Hospital, Fourth Medical Center of PLA General Hospital, The First Affiliated Hospital of University of Science and Technology of China, Ruijin Hospital, First Affiliated Hospital Xi'an Jiaotong University, The Second Hospital of Hebei Medical University, The First Affiliated Hospital of Shanxi Medical University, Xuanwu Hospital, Beijing, The First Hospital of Jilin University, Xiangya Hospital of Central South University, Beijing Sanbo Brain Hospital, Nanjing Medical University
- Responsible party
- Principal investigator, Shuli Liang, Beijing Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04198181
- Sponsor's protocol ID
- TRE-RES
- Status last verified
- May 2024
- First submitted
- 9 Dec 2019
- Last updated
- 17 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Wei Z, Liu T, Cao D, Zhang K, Yang Z, Guo Q, Xu J, Liu S, Liu X, Guan Y, Peng Y, Yuan L, Chen L, Peng J, Han X, Chen C, Chen F, Lin W, Yu T, Zhao X, Wang J, Zhao R, Kuang S, Shi X, Zhai F, Zhang S, Feng W, Shan Y, Ding P, Qian R, Fang F, Chen S, Li H, Wang Y, Liu Q, Zhang H, Li W, Sun M, Zhang R, Liang S. Resective Surgery for Drug-Resistant Epilepsy in Patients With Tuberous Sclerosis Complex: A Prospective Nationwide Multicenter Cohort Study. Neurology. 2025 Nov 11;105(9):e214260. doi: 10.1212/WNL.0000000000214260. Epub 2025 Oct 16.
PubMed 41100778
Study at a glance
From the registry record, last updated by the sponsor on 17 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Evaluation of the Effectiveness and Safety of Resective Epilepsy Surgery for Tuberous Sclerosis Complex Related Epilepsy
- ClinicalTrials.gov ID
- NCT04198181
- Acronym / sponsor ID
- TRE-RES
- Conditions
- Tuberous Sclerosis Complex, Epilepsy
- Keywords
- epilepsy, tuberous sclerosis complex, resective epilepsy surgery
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 200 participants (actual)
- Lead sponsor
- Beijing Children's HospitalUniversity, hospital or other
- Collaborators
- Shenzhen Children's Hospital, Tiantan Hospital, Capital Medical University, Peking University People's Hospital, Guangdong 999 Brain Hospital, Second Affiliated Hospital of Army Medical University, Shandong Provincial Hospital, West China Hospital, Henan Provincial People's Hospital, Shanghai Children's Hospital, The First Medical Center of Chinese PLA General Hospital, Fourth Medical Center of PLA General Hospital, The First Affiliated Hospital of University of Science and Technology of China, Ruijin Hospital, First Affiliated Hospital Xi'an Jiaotong University, The Second Hospital of Hebei Medical University, The First Affiliated Hospital of Shanxi Medical University, Xuanwu Hospital, Beijing, The First Hospital of Jilin University, Xiangya Hospital of Central South University, Beijing Sanbo Brain Hospital, Nanjing Medical University
- Study start
- 12 Dec 2019 (actual)
- Primary completion
- 15 Mar 2027 (estimated)
- Study completion
- 15 Mar 2027 (estimated)
- First posted
- 13 Dec 2019 (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.
A prospective cohort studies to identify clinical seizure control, cognitive changes, and safety in resective epilepsy surgery in patients with TSC-related drug-resistant epilepsy.
Detailed description
Two hundred candidates are recruited and assigned to the control and surgery groups with 100 cases in each group, respectively. All patients will accept first-stage pre-enrollment evaluations and patients in surgery group will accept second-stage invasive evaluation.
All patients will be advised to visit the hospitals for examination each year after enrollment. Seizure outcomes will be assessed with reference to patients' seizure diaries and caregivers' oral statements by both neurosurgeons and neurologists.
All adverse effects will be classified according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0 (2017).
Statistical analyses were conducted using SPSS (version 26.0, IBM, USA), Stata (version 16.0, Stata Corp LLC, USA), and R (version 4.3.3, R Foundation for Statistical Computing, Austria). Outcomes were presented according to data type as percentages, mean ± standard deviation, or median (interquartile range, IQR).
Multiple imputation was employed to address missing data for IQ and QOL at enrollment and the 2-year follow-up. A sensitivity analysis was conducted to assess the robustness of the multiple imputation results. The "mi impute regress" statement in Stata facilitated the multiple imputations. 20 T-tests were utilized for comparing continuous variables, with results reported as mean ± standard deviation. The Mann-Whitney U test was applied for non-normally distributed continuous variables, presenting results as median values and IQRs. Chi-square and Fisher's exact tests were used for univariate analyses. A significance level of p < 0.05 was established.
To account for differences in covariates between the surgery and medicine groups, propensity score matching (PSM) was performed. Based on PSM, Kaplan-Meier curves and the log-rank test were utilized to analyze time-to-seizure recurrence differences between the surgery and medicine groups. Additionally, multivariable Cox proportional hazards regression was applied to predict risk factors for postoperative seizure recurrence within a 51-month follow-up period after PSM.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Control Group | – | –Patients in the surgery group will accept exclusive rational medicine therapy. |
| Surgery Group | – | Procedure: SurgeryPatients in the surgery group will undergo resective surgery combined with rational medicine therapy. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients from participating medical institutions
Key inclusion criteria (5 listed)
- 2 years old and above, no gender restriction, TSC gene monitoring with or without abnormality
- Diagnosis of tuberous sclerosis- related drug-resistant epilepsy
- Epilepsy course for more than 1 year
- Patients who have taken 3 or more reasonable choices with appropriate and tolerable antiepileptic drugs (excluding mTOR inhibitors and traditional Chinese medicine and prescriptions) had seizures more than 12 times in the 3 months before enrollment
- The family members agreed to enroll and signed the informed consent.
Key exclusion criteria (4 listed)
- Obvious renal angiomyolipoma, pulmonary lymphoma leiomyomatosis, and subventricular giant cell astrocytoma
- Abnormal heart, lung, liver, and kidney functions and coagulation function
- Preoperative evaluation, it is considered that no surgical treatment is needed
- The patient received other craniocerebral surgical treatment within 1 year during the follow-up period
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SurgeryResective surgery included lobectomy (partial or total brain lobe resection), tuberectomy (epileptogenic tuber resection), and tuberectomy plus (resection of epileptogenic tuber and perituberal gyri). Tuberectomy was typically performed on the epileptogenic tuber within or near an eloquent area. Lobectomy was performed in patients with large epileptogenic tubers in the brain lobes. Multiple lobectomies, tuberectomies, tuberectomies plus, and lobectomy combined with tuberectomy/tuberectomies plus were further performed in patients with multiple epileptogenic tubers. | Procedure or surgery | Surgery Group |
Primary outcome
% of patients with ILAE grading
Time frame: 3 years
The grading made by ILAE for the classification of outcome with respect to epileptic seizures following epilepsy surgery. It contains a total of 6 levels. The higher the level, the worse the result. We will count the percentage of patients at each level.
2 secondary outcomes
IQ
Time frame: 3 years
Quality of Life: QOLIE-31
Time frame: 3 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 17 Jan 2025.
- Derived from this study: PubMed 41100778Wei Z, Liu T, Cao D, Zhang K, Yang Z, Guo Q, Xu J, Liu S, Liu X, Guan Y, Peng Y, Yuan L, Chen L, Peng J, Han X, Chen C, Chen F, Lin W, Yu T, Zhao X, Wang J, Zhao R, Kuang S, Shi X, Zhai F, Zhang S, Feng W, Shan Y, Ding P, Qian R, Fang F, Chen S, Li H, Wang Y, Liu Q, Zhang H, Li W, Sun M, Zhang R, Liang S. Resective Surgery for Drug-Resistant Epilepsy in Patients With Tuberous Sclerosis Complex: A Prospective Nationwide Multicenter Cohort Study. Neurology. 2025 Nov 11;105(9):e214260. doi: 10.1212/WNL.0000000000214260. Epub 2025 Oct 16.
- 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 Tuberous Sclerosis Complex and Epilepsy, run by Beijing Children's Hospital at 1 site in China.
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 5 inclusion and 4 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: under way, not looking for participants, as of the sponsor's last update on 17 Jan 2025. 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 China. 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?
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?
% of patients with ILAE grading (time frame: 3 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 NCT04198181, 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 17 Jan 2025.