specify the effect of Everolimus on SEGA volume change before and after treatment and to determine the effect of this drug on renal angiomyolipoma (AML), skin lesions, and seizures in TSC patients.
Design
Sample size was calculated based on the two-proportion comparison (before-after) [McNemar's test], considering a proportion of ۲۸.۵% response to treatment
Settings and conduct
All patients with the diagnosis of tuberculosis and all the above criteria who had referred to Imam Reza (AS) pediatric oncology clinic were included in the study, and the examination and questionnaire were completed at specific time intervals.
Participants/Inclusion and exclusion criteria
Inclusion criteria: Patients who definitely diagnosed by tuberous sclerosis complex and medically stable.
Exclusion criteria: To require any surgery for subependymal giant cell astrocytomas, critical hydrocephalus or imminent cerebral herniation
patients with severe hepatic impairment
Intervention groups
Everolimus will be administered orally at a starting dose of ۴.۵ mg/m۲ body surface area per day and adjusted to attain blood trough concentrations of ۵-۱۵ ng/mL considering toxic side effects.
Main outcome variables
Primary endpoint will be considered as the confirmed tumor response, defined as a reduction in the total volume of all target subependymal giant cell astrocytomas of ۵۰% or more relative to baseline, in the absence of worsening of non-target subependymal giant cell astrocytomas.
General information
Reason for update
Acronym
IRCT registration information
IRCT registration number:IRCT20240112060688N1
Registration date:2024-12-23, 1403/10/03
Registration timing:registered_while_recruiting
Last update:2024-12-23, 1403/10/03
Update count:0
Registration date
2024-12-23, 1403/10/03
Registrant information
Name
Hadi Mottaghipisheh
Name of organization / entity
Country
Iran (Islamic Republic of)
Phone
+98 71 3830 3110
Email address
mottaghipisheh@sums.ac.ir
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2024-12-21, 1403/10/01
Expected recruitment end date
2025-12-22, 1404/10/01
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Efficacy of Everolimus in pediatric patients with tuberous sclerosis and sub ependymal giant cell astrocytoma
Public title
Everolimus in sub ependymal giant cell astrocytoma
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
Patients who definitely diagnosed by tuberous sclerosis complex and medically stable
children 1 to 18 years old
Exclusion criteria:
To require any surgery for subependymal giant cell astrocytomas, critical hydrocephalus or imminent cerebral herniation
Age
From 1 year old to 18 years old
Gender
Both
Phase
4
Groups that have been masked
No information
Sample size
Target sample size:
29
Randomization (investigator's opinion)
N/A
Randomization description
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Single
Other design features
Secondary Ids
empty
Ethics committees
1
Ethics committee
Name of ethics committee
research Ethics committees of school of medicine - shiraz university of medical sciences
Street address
farhangshahr station 11 Amir oncology hospital
City
shiraz
Province
Fars
Postal code
۷۱۸۷۹۱۵۹۹۸
Approval date
2024-09-28, 1403/07/07
Ethics committee reference number
IR.SUMS.MED.REC.1403.433
Health conditions studied
1
Description of health condition studied
tuberous sclerosis
ICD-10 code
Q85.1
ICD-10 code description
Tuberous sclerosis
Primary outcomes
1
Description
decreased size of sub ependymal giant cell astrocytoma
Timepoint
at the beginning and every 3 months till one year
Method of measurement
MRI findings
2
Description
Absolute change from baseline to ۲۴ weeks in seizure frequency per ۲۴ hours
Timepoint
at the beginning and every 3 months till one year
Method of measurement
Electroencephalography
3
Description
skin lesion response rate in patients with at least one skin lesion at baseline;
Timepoint
at the beginning and every 3 months till one year
Method of measurement
physical examination
4
Description
angiomyolipoma response rate
Timepoint
at the beginning and every 3 months till one year
Method of measurement
ultrasound sonography
Secondary outcomes
empty
Intervention groups
1
Description
Intervention group: eligible children (aged under ۱۸ years old) with a definite diagnosis of tuberous sclerosis complex will be included by convenience sampling method based on the inclusion exclusion criteria: Everolimus will be administered orally at a starting dose of ۴.۵ mg/m۲ body surface area per day for ۱۲ months duration and adjusted to attain blood trough concentrations of ۵-۱۵ ng/mL considering toxic side effects. All patients were monitored for adverse effects during everolimus treatment, like stomatitis, aphthous ulcer, flushing, hypertension, peripheral edema, hyperglycemia, abdominal pain and others included in questionnaire. In some patients because of unavailability of test for everolimus level we decreased ۲۰% of prescription dose after severe aphthous lesions or severe toxicity occurred. if patients do not tolerate everolimus, drug stopped and excluded from the study. Our patients followed for minimum one year after start of everolimus. Brain MRI will be done every ۳ months after initiation of the treatment and abdominal and pelvic sonography for angiomyolipima lesions in and reported in single specialized center and reported by a single expert radiologist. All patients will be completed an EEG at baseline and ۲۴ weeks (or end of treatment for those who discontinued) .Skin lesions will be assessed every ۳ months by physician in clinic according to size, number, color and location. Blood will be drawn every visit starting at week ۲ for everolimus drug level, including hematology and blood chemistry, will be done every ۲ weeks for the first ۴ weeks, then monthly.
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
Shiraz- Pediatric Oncology Imam Reza clinic
Full name of responsible person
Hadi Mottaghipisheh
Street address
Namazi square
City
Shiraz
Province
Fars
Postal code
7134814734
Phone
+98 71 3212 7000
Email
hmottaghipisheh@gmail.com
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Shiraz University of Medical Sciences
Full name of responsible person
Mohammad Hashem Hashempoor
Street address
Zand St., Central Building of Shiraz University of Medical Sciences, 7th floor, Research and Technology Deputy
City
Shiraz
Province
Fars
Postal code
7134814336
Phone
+98 71 3212 2430
Email
vcrdep@sums.ac.ir
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Shiraz University of Medical Sciences
Proportion provided by this source
100
Public or private sector
Public
Domestic or foreign origin
Domestic
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
Academic
Person responsible for general inquiries
Contact
Name of organization / entity
Shiraz University of Medical Sciences
Full name of responsible person
Hadi Mottaghipisheh
Position
Assistant Professor
Latest degree
Subspecialist
Other areas of specialty/work
Oncology
Street address
Farhang Shahr - Station 11 - Amir Oncology Hospital
City
Shiraz
Province
Fars
Postal code
7187915998
Phone
+98 71 3632 3534
Email
hmottaghipisheh@gmail.com
Person responsible for scientific inquiries
Contact
Name of organization / entity
Shiraz University of Medical Sciences
Full name of responsible person
Hadi Mottaghipisheh
Position
Assistant Professor
Latest degree
Subspecialist
Other areas of specialty/work
Oncology
Street address
Farhang Shahr - Station 11 - Amir Oncology Hospital
City
Shiraz
Province
Fars
Postal code
7187915998
Phone
+98 71 3632 3534
Email
hmottaghipisheh@gmail.com
Person responsible for updating data
Contact
Name of organization / entity
Shiraz University of Medical Sciences
Full name of responsible person
Hadi Mottaghipisheh
Position
Assistant Professor
Latest degree
Subspecialist
Other areas of specialty/work
Hematology
Street address
Farhang Shahr - Station 11 - Amir Oncology Hospital
City
Shiraz
Province
Fars
Postal code
7187915998
Phone
+98 71 3632 3534
Email
hmottaghipisheh@gmail.com
Sharing plan
Deidentified Individual Participant Data Set (IPD)
Yes - There is a plan to make this available
Study Protocol
Yes - There is a plan to make this available
Statistical Analysis Plan
Yes - There is a plan to make this available
Informed Consent Form
Yes - There is a plan to make this available
Clinical Study Report
Yes - There is a plan to make this available
Analytic Code
Yes - There is a plan to make this available
Data Dictionary
Yes - There is a plan to make this available
Title and more details about the data/document
all collected deidentified IPD
When the data will become available and for how long
starting after publication
To whom data/document is available
people working in academic institutions
Under which criteria data/document could be used
no any other criteria
From where data/document is obtainable
email
What processes are involved for a request to access data/document