The evaluation and comparison of the brain, renal, skin lesions, and seizure severity in the TSC patients before and after 6 months of everolimus treatment.
Design
20 eligible children, age under 15 years with TSC will include in this study after obtaining consent. A code was allocated to each one of them.
Settings and conduct
TSC patients referring to the Neurology Clinic of Tabriz Children Hospital after including in the study, brain, renal, skin lesions, and seizure severity will be evaluated in the patients by standard methods. Based on the references, 3 mg/m2/day of everolimus will be administered to the patients for 6 months. The patients will be evaluated for any probable side effect of the drug during the administration period. After six months of the treatment, the severity of the lesions will be evaluated again by the standard methods and will be statistically compared with the basal severity of the lesions.
Participants/Inclusion and exclusion criteria
20 Children with TSC, age under 15 years that referred to Neurology Clinic of Tabriz Children Hospital will purposefully be chosen and included in the study after the diagnisis and confirmation of the TSC disease by a medical specialist and getting informed consent. The patients with underlying diseases and or with drug prohibition will be excluded from the study.
Intervention groups
Based on the references, 3 mg/m2/day of everolimus will be administered to the patients for 6 months. The patients will be evaluated for any probable side effect of the drug during the administration period.
Main outcome variables
The results of this study can be considered as an important step in controlling and treating TSC. If the positive therapeutic effect of everolimus on TSC-associated lesions is proven; this drug could also be used in the treatment process of the TSC.
General information
Reason for update
Acronym
IRCT registration information
IRCT registration number:IRCT20131012014988N2
Registration date:2017-12-05, 1396/09/14
Registration timing:registered_while_recruiting
Last update:2017-12-05, 1396/09/14
Update count:0
Registration date
2017-12-05, 1396/09/14
Registrant information
Name
Mohammad Barzegar
Name of organization / entity
Pediatric Health Research Center,Tabriz University of Medical Sciences
Country
Iran (Islamic Republic of)
Phone
+98 41 3526 2265
Email address
phrc_mb@tbzmed.ac.ir
Recruitment status
Recruitment complete
Funding source
Pediatric Health Research Center, Tabriz University of Medical Sciences.
Expected recruitment start date
2017-07-04, 1396/04/13
Expected recruitment end date
2018-01-03, 1396/10/13
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
The Effect of Everolimus on Renal, Brain, Skin Lesions and Seizure of Tuberous Sclerosis Complex
Public title
The Effect of Everolimus in the Treatment of Tuberous Sclerosis Complex
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
20 children, age under 15 years, with TSC proven by an expert physician through clinical and paraclinical criteria will include in this study
Parental consent of the patients
Exclusion criteria:
Having any underlying disease
Drug prohibition
Age
To 15 years old
Gender
Both
Phase
N/A
Groups that have been masked
No information
Sample size
Target sample size:
20
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
Regional Ethics Committee, Office of the Vice Chancellor for Research, Tabriz University of Medical
Street address
International Relations Office, No 2 Central Building, Tabriz University of Medical Sciences, University Street, Tabriz, PO Box: 5165665931, Iran
City
Tabriz
Province
East Azarbaijan
Postal code
5165665931
Approval date
2017-07-03, 1396/04/12
Ethics committee reference number
IR.TBZMED.REC.1396.287
Health conditions studied
1
Description of health condition studied
Tuberous sclerosis
ICD-10 code
Q85.1
ICD-10 code description
Other congenital malformations
Primary outcomes
1
Description
Renal lesions severity
Timepoint
before treatment-six months after the treatment
Method of measurement
MRI, Medical ultrasound (sonography), questionnaire
2
Description
Brain lesions severity
Timepoint
before treatment-six months after the treatment
Method of measurement
MRI, Medical ultrasound (sonography), questionnaire
3
Description
Skin lesions severity
Timepoint
before treatment-six months after the treatment
Method of measurement
Medical examination, questionnaire
4
Description
Seizure
Timepoint
before treatment-six months after the treatment
Method of measurement
Questionnaire
Secondary outcomes
1
Description
constipation
Timepoint
During the six months of the administration period
Method of measurement
Clinical Examination, Questionnaire
2
Description
nausea
Timepoint
During the six months of the administration period
Method of measurement
Clinical Examination, Questionnaire
3
Description
diarrhea
Timepoint
During the six months of the administration period
Method of measurement
Clinical Examination, Questionnaire
4
Description
vomiting
Timepoint
During the six months of the administration period
Method of measurement
Clinical Examination, Questionnaire
5
Description
Body weight
Timepoint
During the six months of the administration period
Method of measurement
Scale, Questionnaire
Intervention groups
1
Description
In the case (intervention) group, based on the scientific references, 3 mg/m2/day "Everolimus" (C53H83NO14), in 5 mg tablet form (Novartis Co, Swiss), will administer for 6 months.
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
Tabriz Children Hospital
Full name of responsible person
Dr Mohammad Barzegar
Street address
Tabriz Children Hospital, Sheshgelan Street, Tabriz, Iran.
City
Tabriz
Province
East Azarbaijan
Postal code
5136735886
Phone
+98 41 3526 2250
Fax
+98 41 3526 2265
Email
phrc_mb@tbzmed.ac.ir
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Pediatric Health Rsearch Center, Tabriz University of Medical Sciences
Full name of responsible person
Dr. Mohammad Barzegar
Street address
Pediatric Health Research Center, Tabriz Children Hospital, Sheshgelan Street, Tabriz, Iran.
City
Tabriz
Province
East Azarbaijan
Postal code
5136735886
Phone
+98 41 3526 2265
Fax
+98 41 3526 2265
Email
phrc_mb@tbzmed.ac.ir
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Pediatric Health Rsearch Center, Tabriz 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
Pediatric Health Research Center, Tabriz University of Medical Sciences
Full name of responsible person
Dr. Mohammad Barzegar
Position
Pediatric neurologist, Professor, Faculty Member
Latest degree
Subspecialist
Other areas of specialty/work
Neuroscience
Street address
Pediatric Health Research Center, Children Hospital, Sheshgelan street, Tabriz, Iran.
City
Tabriz
Province
East Azarbaijan
Postal code
5136735886
Phone
+98 41 3526 2265
Fax
+98 41 3526 2265
Email
phrc_mb@tbzmed.ac.ir
Web page address
Person responsible for scientific inquiries
Contact
Name of organization / entity
Pediatric Health Research Center, Tabriz University of Medical Sciences
Full name of responsible person
Dr. Moahhamad Barzegar
Position
Pediatric Neurologist
Latest degree
Subspecialist
Other areas of specialty/work
Neuroscience
Street address
Pediatric Health Research Center, Tabriz Children Hospital, Sheshgelan Street, Tabriz, Iran.
City
Tabriz
Province
East Azarbaijan
Postal code
5136735886
Phone
+41 35262265
Fax
+98 41 3526 2265
Email
phrc_mb@tbzmed.ac.ir
Web page address
Person responsible for updating data
Contact
Name of organization / entity
Tabriz Children Hospital
Full name of responsible person
Dr Hassan Bakhtiari
Position
Pediatric neurology fellowship
Latest degree
Subspecialist
Other areas of specialty/work
Neuroscience
Street address
Tabriz Children Hospital, Sheshgelan Street, Tabriz, Iran.
City
Tabriz
Province
East Azarbaijan
Postal code
5136735886
Phone
+98 41 3526 2265
Fax
+98 41 3526 2265
Email
phrc_mb@tbzmed.ac.ir
Web page address
Sharing plan
Deidentified Individual Participant Data Set (IPD)
Undecided - It is not yet known if there will be a plan to make this available
Study Protocol
Undecided - It is not yet known if there will be a plan to make this available
Statistical Analysis Plan
Undecided - It is not yet known if there will be a plan to make this available
Informed Consent Form
Undecided - It is not yet known if there will be a plan to make this available
Clinical Study Report
Undecided - It is not yet known if there will be a plan to make this available
Analytic Code
Undecided - It is not yet known if there will be a plan to make this available
Data Dictionary
Undecided - It is not yet known if there will be a plan to make this available