Evaluating overall survival and the rate of GVHD in children and adolescents receiving antithymocyte globulin as an immunosuppressant during hematopoietic stem cell transplantation
Evaluating the overall survival and incidence of GVHD in children receiving T globulin ۲۵ during hematopoietic stem cell transplant
Design
Open-label clinical trial with retrospective control group, on patients undergoing HSCT during a one year period
Settings and conduct
All patients undergoing HSCT during 1 year, at the Children's Medical Center will be enrolled in the study if they meet the eligibility criteria and will be prescribed anti-thymocyte globulin based on their protocol. The patients will be visited and assessed for acute and chronic GVHD and infections, daily during hospitalization, and after discharge: weekly until 3 months, biweekly until 6 months, then monthly until 12 months.
Participants/Inclusion and exclusion criteria
Inclusion criteria:
1 to 18 year-old HSCT recipients
Providing written informed consent
Lansky score above 70%
Absence of severe active infection
Exclusion criteria:
History of previous hematopoietic stem cell transplant
previous treatment with anti-thymocyte globulin in the last three months
abnormal liver, renal, and heart function
Intervention groups
Test group: anti-thymocyte globulin (manufactured by Kawsar Biotech Company) the dose and duration of administration will be based on the treatment protocol.
Control group: anti-thymocyte globulin (manufactured by Sanofi Genzyme) the dose and duration of administration was based on the treatment protocol, and this group has received the medication during the previous year.
Main outcome variables
Overall survival and GVHD
General information
Reason for update
Acronym
IRCT registration information
IRCT registration number:IRCT20210523051368N1
Registration date:2022-02-28, 1400/12/09
Registration timing:registered_while_recruiting
Last update:2022-02-28, 1400/12/09
Update count:0
Registration date
2022-02-28, 1400/12/09
Registrant information
Name
maryam behfar
Name of organization / entity
Country
Iran (Islamic Republic of)
Phone
+98 21 6147 2931
Email address
behfarm@sina.tums.ac.ir
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2022-02-24, 1400/12/05
Expected recruitment end date
2023-02-24, 1401/12/05
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Evaluating overall survival and the rate of GVHD in children and adolescents receiving antithymocyte globulin as an immunosuppressant during hematopoietic stem cell transplantation
Public title
Survival and incidence of GVHD in children receiving T globulin 25 as an immunosuppressant during hematopoietic stem cell transplantation
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
1 to 18 year-old hematopoietic stem cell transplant recipients with anti-thymocyte globulin in their conditioning regimen
parents or legally acceptable representatives must have provided a written informed consent
Lansky score above 70%
Absence of severe active infection
use of bone marrow or G-CSF-stimulated peripheral blood stem cells
Exclusion criteria:
History of previous stem cell transplantation (re-transplantation)
use of umbilical cord blood stem cells
history of previous treatment with anti-thymocyte globulin in the last three months
Known allergic reaction to rabbit proteins
abnormal liver function (total bilirubin greater than three times the upper limit of normal (ULN), conjugated bilirubin> 2 mg/dl, transaminases greater than three times the ULN)
abnormal renal function adjusted for the patient's age
abnormal heart function (left ventricular ejection fraction < 50% after ECHO, congestive heart failure, arrhythmia)
Age
From 1 year old to 18 years old
Gender
Both
Phase
3
Groups that have been masked
No information
Sample size
Target sample size:
45
Randomization (investigator's opinion)
N/A
Randomization description
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Other
Other design features
Secondary Ids
empty
Ethics committees
1
Ethics committee
Name of ethics committee
Ethics Committee in Research of Children's Medical Center-Tehran University of Medical Sciences
Street address
Childrens Medical Center, Gharib Ave, Keshavarz Blvd, Tehran
City
Tehran
Province
Tehran
Postal code
1419733151
Approval date
2021-10-19, 1400/07/27
Ethics committee reference number
IR.TUMS.CHMC.REC.1400.161
Health conditions studied
1
Description of health condition studied
hematopoietic stem cell transplantation
ICD-10 code
Z94.84
ICD-10 code description
Stem cells transplant status
2
Description of health condition studied
Graft Versus Host Disease
ICD-10 code
D89.810
ICD-10 code description
Acute graft-versus-host disease
3
Description of health condition studied
Graft Versus Host Disease
ICD-10 code
D89.811
ICD-10 code description
Chronic graft-versus-host disease
Primary outcomes
1
Description
Rate of acute GVHD
Timepoint
First 100 days after transplantation
Method of measurement
Physical examination and laboratory findings
2
Description
Overall survival
Timepoint
until 1 year after transplantation
Method of measurement
Mortality assessment
Secondary outcomes
1
Description
Rate of chronic GVHD
Timepoint
From 100 days until 1 year after transplantation
Method of measurement
Physical examination and laboratory findings
2
Description
Rate of bacterial infections
Timepoint
until 1 year after transplantation
Method of measurement
Blood culture
3
Description
Rate of fungal infections
Timepoint
until 1 year after transplantation
Method of measurement
Laboratory test (Aspergillus galactomannan antigen in blood by ELISA)
4
Description
Rate of viral infections
Timepoint
until 1 year after transplantation
Method of measurement
Laboratory test (RT-PCR for CMV infection)
5
Description
rejection
Timepoint
Until 1 month after transplantation
Method of measurement
evaluation of chimerism by Short tandem repeat or FISH
Intervention groups
1
Description
Intervention group: TGlobulin 25 (manufactured by Kawsar Biotech Company) with standard doses: haploidentical stem cell transplant, primary immunodeficiency patients: 2.5 mg/kg from day -4 to -1, Aplastic anemia: 2.5 mg/kg from day -4 to -2, other related transplant: 2.5 mg/kg from day -2 to -1, one locus mismatched transplant: 2.5 mg/kg from day -3 to day -1, and unrelated donor transplant 2.5 mg/kg from day -3 to -1
Category
Treatment - Drugs
2
Description
Control group: antithymocyte globulin vials (manufactured by Sanofi Genzyme) with the same standard doses according to the type of transplantation, that had been administered through the previous year.
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
Children's Medical Center Hospital
Full name of responsible person
Maryam Behfar
Street address
Children's Medical Center Hospital, Gharib Ave, Keshavarz Blvd, Tehran
City
Tehran
Province
Tehran
Postal code
1419733151
Phone
+98 21 6694 8444
Email
behfarm@sina.tums.ac.ir
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Kowsar Biotechnology company
Full name of responsible person
Siroos Zeinali
Street address
N0 41, Majlesi st., Valiasr Street
City
Tehran
Province
Tehran
Postal code
1595645513
Phone
+98 21 8893 0143
Email
Kawsar_biotech@yahoo.com
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Kowsar Biotechnology company
Proportion provided by this source
100
Public or private sector
Private
Domestic or foreign origin
Domestic
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
Industry
Person responsible for general inquiries
Contact
Name of organization / entity
Tehran University of Medical Sciences
Full name of responsible person
Maryam Behfar
Position
Associate professor
Latest degree
Subspecialist
Other areas of specialty/work
pediatric hematology and oncology
Street address
Children's Medical Center Hospital, Gharib Ave, Keshavarz Blvd, Tehran
City
Tehran
Province
Tehran
Postal code
1419733151
Phone
+98 21 6694 8444
Email
behfarm@sina.tums.ac.ir
Person responsible for scientific inquiries
Contact
Name of organization / entity
Tehran University of Medical Sciences
Full name of responsible person
Amir Ali Hamidieh
Position
Professor
Latest degree
Subspecialist
Other areas of specialty/work
pediatric hematology and oncology
Street address
Children's Medical Center, Gharib Ave, Keshavarz Blvd, Tehran
City
Tehran
Province
Tehran
Postal code
1419733151
Phone
+98 21 6694 8444
Email
aahamidieh@tums.ac.ir
Person responsible for updating data
Contact
Name of organization / entity
Tehran University of Medical Sciences
Full name of responsible person
Maryam Behfar
Position
Associate professor
Latest degree
Subspecialist
Other areas of specialty/work
pediatric hematology and oncology
Street address
Children's Medical Center Hospital, Gharib Ave, Keshavarz Blvd, Tehran
City
Tehran
Province
Tehran
Postal code
1419733151
Phone
+98 21 6694 8444
Email
behfarm@sina.tums.ac.ir
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