1- Objectives: to evaluate compliance, efficacy and safety of JADENU (new formulation of EXJADE) consumption among the transfusion dependent thalassemia major patients by designed questionnaire. 2- Design: the study is designed as a phase 3, open label, multicenter study. One hundred patients who eligible for inclusion criteria will be enrolled in the study. Fifty patients for JADENU and fifty patients for EXJADE consumption. 3- Setting and conduct: all stages of the project explain for each patient, then if they want to enter the project inform consent would be obtained. The patients will be treated by a 24 week Open label treatment phase. Serum Ferritin Levels will be measured monthly. 4- Participants including major eligibility criteria, transfusion dependent beta-thalassemia major patients. 5- Intervention, 20-40 mg/kg/day dose regimen of EXJADE and 14-28 mg/kg/day dose regimen of JADENU considering serum ferritin level. 6- Main outcome measures (variables), Include assessing the compliance, efficacy and safety of JADENU consumption. Serum Ferritin Levels will be measured monthly to evaluate the response to JADENU. The rate of satisfactions and compliance of patients in two groups of therapy will be evaluated at the end of study by designed questionnaire.
General information
Acronym
IRCT registration information
IRCT registration number:IRCT2015101218603N2
Registration date:2015-12-21, 1394/09/30
Registration timing:prospective
Last update:
Update count:0
Registration date
2015-12-21, 1394/09/30
Registrant information
Name
Fatemeh Amirmoezi
Name of organization / entity
Hematology research center/SUMS
Country
Iran (Islamic Republic of)
Phone
+98 71 3628 1563
Email address
amirmoezif@sums.ac.ir
Recruitment status
Recruitment complete
Funding source
Shiraz University of Medical Sciences
Expected recruitment start date
2015-12-22, 1394/10/01
Expected recruitment end date
2016-01-21, 1394/11/01
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
To assess compliance, efficacy and satisfaction with two different formulation of deferasirox in patients with transfusion-dependent beta-thalassemia
Public title
To assess compliance, efficacy and satisfaction with two different formulation of deferasirox in patients with transfusion-dependent beta-thalassemia
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria: signing informed consent; male or female aged ≥ 2 years at screening; patients with transfusion dependent thalassemia major; regular transfusion indicated by a blood requirement ≥ 8 blood transfusions per year at screening. Exclusion criteria: patients with mean levels of ALT above 5 fold the upper limit of normal (ULN); patients with serum creatinine above ULN; significant proteinuria as indicated by a urinary protein/creatinine ratio> 0.6 (mg/mg); creatinine clearance ≤ 60 ml/min; chronic hepatitis B infection; active hepatitis C infection; pregnancy or breastfeeding; non-transfusion dependent thalassemia (NTDT)
Age
From 10 years old
Gender
Both
Phase
3
Groups that have been masked
No information
Sample size
Target sample size:
100
Randomization (investigator's opinion)
Randomized
Randomization description
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Parallel
Other design features
Secondary Ids
empty
Ethics committees
1
Ethics committee
Name of ethics committee
Ethics committee of Shiraz University of Medical Sciences
Street address
7th floor, Vice chancellery for research affairs, Shiraz University of Medical Sciences, Zand avenue, Shiaz, Iran
City
Shiraz
Postal code
Approval date
2015-11-21, 1394/08/30
Ethics committee reference number
IR.SUMS.REC.1394.152
Health conditions studied
1
Description of health condition studied
Major Thalassaemia
ICD-10 code
D56.1
ICD-10 code description
Beta thalassaemia
Primary outcomes
1
Description
Patients compliance and satisfaction
Timepoint
three months after drug consumption
Method of measurement
designed questionnaire for patient compliance and satisfaction assessing
Secondary outcomes
1
Description
Ferritin serum amount
Timepoint
at the beginning of the study and monthly for 3 months
Method of measurement
Using starfax device
2
Description
Safety
Timepoint
Base line, End of evey month for 3 months
Method of measurement
Follow up, clinical examination by expert hematologist
3
Description
Possible gastrointestinal side effects, including diarrhea, and dermatologic symptoms
Timepoint
Base line, End of every month for three months
Method of measurement
Follow up,clinical examination by expert hematologist
Intervention groups
1
Description
Control group: 20-40 mg/kg/day dose regimen of EXJADE considering serum ferritin level. Frequency and route of administration is once per day orally.
20 mg/kg EXJADE (serum ferritin level: 1000- 1500), 30 mg/kg EXJADE (serum ferritin level: 1500- 2000) and 40 mg/kg EXJADE (serum ferritin level ˃2000)
Category
Treatment - Drugs
2
Description
Intervention group: 14-28 mg/kg/day dose regimen of JADENU considering serum ferritin level. Frequency and route of administration is once per day orally.
14 mg/kg JADENU (serum ferritin level: 1000- 1500), 21 mg/kg JADENU (serum ferritin level: 1500- 2000) and 28 mg/kg JADENU (serum ferritin level ˃2000).
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
Nemazee hospital, Shiaz University of Medical Sciences, Shiraz, Iran
Full name of responsible person
Dr. Mehran Karimi
Street address
City
Shiraz
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Vice chancellor of research, Shiaz Univeisity of Medical Sciences
Full name of responsible person
Dr. Seyed Basir Hashemi
Street address
7th floor, Shiraz University of Medical Sciences, Zand avenue, Shiraz, Iran
City
Shiraz
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Vice chancellor of research, Shiaz Univeisity of Medical Sciences
Proportion provided by this source
100
Public or private sector
empty
Domestic or foreign origin
empty
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
empty
Person responsible for general inquiries
Contact
Name of organization / entity
Hematology Research Center, Nemazee Hospital, Shiraz, Iran
Full name of responsible person
Dr. Sezaneh Haghpanah
Position
Assistance professor of community medicine
Other areas of specialty/work
Street address
Hematology Research Center, Nemazee Hospital, Zand Street, Shiraz, Iran
City
Shiraz
Postal code
Phone
+98 71 3628 1563
Fax
Email
haghpanah@sums.ac.ir
Web page address
Person responsible for scientific inquiries
Contact
Name of organization / entity
Shiraz University of Medical Sciences
Full name of responsible person
Dr. Mehran Karimi
Position
Professor of Pediatric Hematology- Oncology
Other areas of specialty/work
Street address
Hematology Research center, Nemaee Hospital, Zand street, Shiraz, Iran
City
Shiraz
Postal code
Phone
+98 71 3628 1563
Fax
Email
karimim@sums.ac.ir
Web page address
Person responsible for updating data
Contact
Name of organization / entity
Hematology research center, Shiraz University of Medical Sciences
Full name of responsible person
Dr. Fatemeh Amirmoezi
Position
General physicion
Other areas of specialty/work
Street address
City
Shiraz
Postal code
Phone
+98 71 3628 1563
Fax
Email
amirmoezif@sums.ac.ir
Web page address
Sharing plan
Deidentified Individual Participant Data Set (IPD)