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.: PACTR201902478249291 Date of Approval: 07/02/2019
Trial Status: Retrospective registration - This trial was registered after enrolment of the first participant
TRIAL DESCRIPTION
Public title Effects of amlodipine on myocardial iron deposition in pediatric patients with thalassemia major
Official scientific title Effects of amlodipine on myocardial iron deposition in pediatric patients with thalassemia major
Brief summary describing the background and objectives of the trial Mortality rates increased due to iron disposition in cardiac muscles in thalassemia major (TM) patients. Iron overload cardiomyopathy could be treated with a combination therapy of iron chelator and L-type calcium channel blockers. We designed a randomized controlled study to assess the potential of amlodipine to chelation in reducing myocardial iron Concentration in TM patients compared with placebo. Objective This study aims to estimate the change in myocardial iron concentration (MIC) determined by magnetic resonance imaging after 6 months and follow up the change in Liver iron concentration (LIC) , Serum ferritin level (SF), and left ventricle ejection fraction (LVEF).
Type of trial RCT
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 09/10/2017
Actual trial start date 15/10/2017
Anticipated date of last follow up 09/10/2018
Actual Last follow-up date 04/10/2018
Anticipated target sample size (number of participants) 40
Actual target sample size (number of participants) 40
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 Allocation was determined by the holder of the sequence who is situated off site Masking/blinding used Participants
INTERVENTIONS
Intervention type Intervention name Dose Duration Intervention description Group size Nature of control
Experimental Group treatment arm oral amlodipine 2.5-5 mg/day , in addition to a Deferasirox chelation regimen for 6 months After doing the MRI scans and other laboratory tests, patients were allocated into the iron chelator (Deferasirox) plus amlodipine group (2.5 mg/day for patients weighing less than 30 kg and 5 mg/day for patients weighing more than 30 kg) for 6 months . The study medications were dispensed at each monthly visit. 20
Control Group placebo arm the iron chelator Deferasirox (Exjade; Novartis 20-40 mg/kg/day) for 6 months After doing the MRI scans and other laboratory tests, patients were allocated randomly into the iron chelator Deferasirox placebo group (Exjade; Novartis 20-40 mg/kg/day) for 6 months The study medications were dispensed at each monthly visit. 20 Placebo
ELIGIBILITY CRITERIA
List inclusion criteria List exclusion criteria Age Category Minimum age Maximum age Gender
Patients eligible for enrollment at baseline were male or female aged between 6 and 18 years old with β-Thalassemia major. Potential participants had been diagnosed with thalassemia major (TM), as confirmed by the presence of microcytic hypochromic anemia and hemoglobin electrophoresis; additionally, patients who had been receiving a regular blood transfusion during the last two years, with a serum ferritin (SF) level more than 1000 ng /ml were also considered. Participants were excluded if they were more than 20 years old, their SF was less than 1000 ng /ml, they experienced heart failure (ejection fraction (EF) less than 30%), they were contraindicated to undergo the magnetic resonance imaging (MRI) scan, or they were expected to change their chelation therapy regimen during the next 6 months. Adolescent: 13 Year-18 Year,Child: 6 Year-12 Year 6 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 09/10/2017 Faculty of Medicine Beni suef university Research Ethical committee FM BSU REC
Ethics Committee Address
Street address City Postal code Country
Al Shamlah , Beni suef Beni suef 62515 Egypt
OUTCOMES
Type of outcome Outcome Timepoint(s) at which outcome measured
Primary Outcome The primary trial endpoint was the change in the MIC and cardiac T2* after 6 months in either the placebo or treatment group. Efficacy data were analyzed as changes from core baseline to month 6 6 months
Secondary Outcome The secondary trial endpoint were potential changes in the liver iron concentration , liver T2*, serum ferritin level , and left ventricle ejection fraction after 6 months of treatment in either group. 6 months
RECRUITMENT CENTRES
Name of recruitment centre Street address City Postal code Country
Out patient Hematology clinic Beni Suef university hospital Al Shamlah , Beni suef Beni suef 62515 Egypt
FUNDING SOURCES
Name of source Street address City Postal code Country
Arwa khaled Abd el rhman 18 El Fondi st Mokbal Beni Suef 62515 Egypt
SPONSORS
Sponsor level Name Street address City Postal code Country Nature of sponsor
Primary Sponsor Beni suef university hospital Alshamlah street Beni suef 62515 Egypt Hospital
COLLABORATORS
Name Street address City Postal code Country
Hoda A.Salem 52 Gameat Al-dwal Alarabya, Almohandseen, Cairo 12655 Egypt
Dina Ezzat 6th Octobar City, Fourth touristic area Giza 12568 Egypt
Hadeel Seif 00202 Mkatam Cairo 8582 Egypt
Hoda Rabee 23 Faculty of pharmacy st Beni Suef 62514 Egypt
CONTACT PEOPLE
Role Name Email Phone Street address
Principal Investigator Arwa Khaled dr_arwakhaled@yahoo.com 00201011332315 18 el fondi st , Mokbal , Beni suef
City Postal code Country Position/Affiliation
Beni suef 62515 Egypt Clinical pharmacist faculty of medicine Beni suef university hospital.
Role Name Email Phone Street address
Public Enquiries Hoda Hagag hoda_cp@yahoo.com 00201144073236 Faculty of pharmay st
City Postal code Country Position/Affiliation
Beni Suef 62514 Egypt lecturer of clinical pharmacy faculty of pharmacy Beni suef university
Role Name Email Phone Street address
Scientific Enquiries Dina Ezzat dinaezzat72@live.com 00201001253636 6th Octobar City, Fourth touristic area
City Postal code Country Position/Affiliation
Giza 12568 Egypt Professor of Pediatrics and Pediatric Hematology Faculty of Medicine Beni Suef University.
REPORTING
Share IPD Description Additional Document Types Sharing Time Frame Key Access Criteria
No
URL Results Available Results Summary Result Posting Date First Journal Publication Date
Yes After 6 months, a significant reduction was noted in the MIC of patients receiving amlodipine (n=20), compared with the patients receiving the placebo (n=20). At baseline, the mean was 0.76±0.11 mg/g dry weight, while at 6 months, the mean was 0.51±0.07 mg/g dry weight (p< 0.001). Also, there was a significant change in the myocardial T2* after 6 months; the amlodipine increased the myocardial T2* from 40.63±5.45 ms at baseline to 43.25±5.35 ms (p< 0.001). However, amlodipine did not significantly affect the secondary outcomes by the end of the study. 31/12/2018 04/02/2019
Result Upload 1: Result Upload 2: Result Upload 3: Result Upload 4: Result Upload 5:
Result URL Hyperlinks Link To Protocol
Result URL Hyperlinks file:///C:/Users/Eng%20Mostfa/Arwa%20khaled/Amlodipine/paper%20for%20journal/thalassemia%20proposal.pdf
Changes to trial information