Pan African Clinical Trials Registry
South African Medical Research Council, South African Cochrane Centre
PO Box 19070, Tygerberg, 7505, South Africa
Telephone: +27 21 938 0506 / +27 21 938 0834 Fax: +27 21 938 0836
Email: pactradmin@mrc.ac.za Website: pactr.samrc.ac.za
Trial no.: PACTR202311877246623 Date of Approval: 23/11/2023
Trial Status: Retrospective registration - This trial was registered after enrolment of the first participant
TRIAL DESCRIPTION
Public title Post -transplantation Cyclophosphamide as Graft Versus Host Disease prophylaxis in Children with β-Thalassemia Major undergoing Hematopoietic Stem Cell Transplantation
Official scientific title Post -transplantation Cyclophosphamide as Graft Versus Host Disease prophylaxis in Children with β-Thalassemia Major undergoing Hematopoietic Stem Cell Transplantation
Brief summary describing the background and objectives of the trial Strategies to reduce GVHD with its long term post-SCT morbidity and mortality without affecting the precious effect of graft-versus-tumor are highly needed .It is generally accepted that preventing GVHD is more effective than treating it once it has been established. PT-Cy associated with low rates of graft-versus- host- disease (GVHD). The aim of this work is to study the impact of using post transplantation cyclophosphamide (PTCy) in combination with other immunosuppressive drugs based conditioning regimen as GVHD prophylaxis in children with beta thalassemia major undergoing stem cell transplantation.
Type of trial CCT
Acronym (If the trial has an acronym then please provide)
Disease(s) or condition(s) being studied Haematological Disorders,Paediatrics
Sub-Disease(s) or condition(s) being studied
Purpose of the trial Treatment: Drugs
Anticipated trial start date 01/03/2021
Actual trial start date 01/03/2021
Anticipated date of last follow up 30/11/2023
Actual Last follow-up date 30/11/2023
Anticipated target sample size (number of participants) 30
Actual target sample size (number of participants) 30
Recruitment status Completed
Publication URL
Secondary Ids Issuing authority/Trial register
STUDY DESIGN
Intervention assignment Allocation to intervention If randomised, describe how the allocation sequence was generated Describe how the allocation sequence/code was concealed from the person allocating the participants to the intervention arms Masking If masking / blinding was used
Parallel: different groups receive different interventions at same time during study Randomised Simple randomization using a randomization table created by a computer software program Sealed opaque envelopes Masking/blinding used Care giver/Provider
INTERVENTIONS
Intervention type Intervention name Dose Duration Intervention description Group size Nature of control
Experimental Group Bone marrow transplantation . Post transplant Cyclophosphamide 40 mg/kg /day once daily 2 days :on day +3 , +4. 2. Group (2): Include 15 children with β-thalassemia major (Pesaro class I, II) undergoing allogeneic peripheral blood stem cell transplantation from matched related donor, myeloablative regimen:  Oral busulfan: (16 mg/kg total dose in 4 divided doses on days -11 to -8 if >8years). (20 mg/kg/total dose in 4 divided on days -11 to -8 if <8years).  Pre transplant Cyclophosphamide: 120 mg/kg total dose ; (30 mg/kg/day once daily on days -5 to -2).  Post transplant Cyclophosphamide : 40 mg/kg /day once daily : on day +3 , +4. 15
Control Group ATG .Methotrexate Low dose ATG. Methotrexate Low dose ATG (30 mg/kg total dose; 10 mg/kg on days -3, -2, -1).  Methotrexate (5mg/m2 /day) on day +1 ,+3 ,+6 1. Group (1): Include 15 children with β-thalassemia major (Pesaro class I, II) undergoing allogeneic hematopoietic stem cell transplantation from matched related donor, myeloablative regimen:  Oral busulfan: (16 mg/kg total dose in 4 divided doses on days -11 to -8 if >8years) . (20 mg/kg/total dose in 4 divided on days -11 to -8 if <8years).  Cyclophosphamide: 200 mg/kg total dose; 50 mg/kg/day once daily on days -5 to -2).  Low dose ATG (30 mg/kg total dose; 10 mg/kg on days -3, -2, -1).  Methotrexate (5mg/m2 /day) on day +1 ,+3 ,+6 . 15 Active-Treatment of Control Group
ELIGIBILITY CRITERIA
List inclusion criteria List exclusion criteria Age Category Minimum age Maximum age Gender
Children with β-thalassemia major (Pesaro class I, II) undergoing allogeneic peripheral blood stem cell transplantation from matched related donor aged 2- 18 years. i. Children with β-thalassemia major (Pesaro class III). ii. Conditions with contraindication to cyclophosphamide use as ( systemic infections , uric acid kidney stones , inflammation of bladder with hemorrhage , obstruction of any part of the urinary tract , bloody urine , kidney disease with likely reduction in kidney function , children with a previous hypersensitivity to it . Adolescent: 13 Year-18 Year,Child: 6 Year-12 Year,Preschool Child: 2 Year-5 Year 2 Year(s) 18 Year(s) Both
ETHICS APPROVAL
Has the study received appropriate ethics committee approval Date the study will be submitted for approval Date of approval Name of the ethics committee
Yes 20/02/2021 local ethical committee of faculty of medicine tanta university
Ethics Committee Address
Street address City Postal code Country
elgeish street tanta 31511 Egypt
OUTCOMES
Type of outcome Outcome Timepoint(s) at which outcome measured
Primary Outcome Acute Graft versus host disease during 3 months after transplantation
Secondary Outcome chronic Graft versus host disease one year after transplantation
RECRUITMENT CENTRES
Name of recruitment centre Street address City Postal code Country
tanta university hospital . Nasser Institute. Elgeish street tanta . cairo 31511 Egypt
FUNDING SOURCES
Name of source Street address City Postal code Country
self fund elgeish tanta 31511 Egypt
SPONSORS
Sponsor level Name Street address City Postal code Country Nature of sponsor
Primary Sponsor faculty of medicine tanta university elgeish tanta 31511 Egypt University
COLLABORATORS
Name Street address City Postal code Country
Mohamed Attia elgeish tanta 31511 Egypt
Nahed Hablas elgeish tanta 31511 Egypt
Mohamed elshanshory elgeish tanta 31511 Egypt
Gamal Alden Fathy elgeish tanta 31511 Egypt
CONTACT PEOPLE
Role Name Email Phone Street address
Principal Investigator Eman Elnaggar emanelnaggarmam@gmail.com 01064096092 elgeish
City Postal code Country Position/Affiliation
tanta 31511 Egypt assistant lecture of pediatric hematology
Role Name Email Phone Street address
Scientific Enquiries Nahed Hablas nahedhablas79@gmail.com 01010560350 elgeish
City Postal code Country Position/Affiliation
tanta 31511 Egypt assistant professor of pediatrics
Role Name Email Phone Street address
Public Enquiries mohammed elshanshoury elshanshory@gmail.com 01005680834 elgeish
City Postal code Country Position/Affiliation
tanta 31511 Egypt profossor of pediatrics
REPORTING
Share IPD Description Additional Document Types Sharing Time Frame Key Access Criteria
Yes we intended to share de-identified individual trial participant data collected during the trial and after deidentification will be available for anyone who wishes to access the data for any purpose. Study Protocol no first gournal publication date
URL Results Available Results Summary Result Posting Date First Journal Publication Date
url hyperlink No
Result Upload 1: Result Upload 2: Result Upload 3: Result Upload 4: Result Upload 5:
Result URL Hyperlinks Link To Protocol
Result URL Hyperlinks
Changes to trial information