A prospective single arm study to assess the efficacy and safety of deferasirox 20 mg/kg BID in transfusion dependent beta-thalassemia patients unresponsive to current treatment with doses > 35 mg/kg QD or with intolerance to single dose therapy
The aim of this study was evaluation safety and efficacy of deferasirox used as divided dose in transfusion dependent beta-thalassemia patients unresponsive to current treatment or with intolerance to single dose therapy. Inclusion of study: Aged 2 years; Serum ferritin>2000 ng/ml; unresponsive to current treatment with doses>35 mg/kg QD(decrease in ferritin level less than 10%);patients who did not tolerate single dose therapy due to adverse effect. Exclusion of study: Patients will be excluded from this trial if they have one of the following criteria: positive serology for HBS Ag , HCV Ab, HIV Ab; Abnormal liver or kidney function tests;An alanine aminotranferase (ALT) level greater than 250 U/L;Also serum creatinine level above the upper limit of normal, and a urinary protein-creatinine ratio of greater than 0.5 mg/mg); a history of ocular toxicity related to iron chelation therapy. Target sample size was forthy. Intervention studied was deferasirox 20 mg/kg BID in transfusion dependent beta-thalassemia patients unresponsive to current treatment with doses > 35 mg/kg QD or with intolerance to single dose therapy. Variation in serum ferritin (every three months for 12 months) was evaluated.
General information
Acronym
IRCT registration information
IRCT registration number:IRCT2014122920051N1
Registration date:2014-12-29, 1393/10/08
Registration timing:prospective
Last update:
Update count:0
Registration date
2014-12-29, 1393/10/08
Registrant information
Name
Mehran Karimi
Name of organization / entity
Hematology Research Center, Shiraz University of Medical Sciences
Country
Iran (Islamic Republic of)
Phone
+98 71 3612 5617
Email address
karimim@sums.ac.ir
Recruitment status
Recruitment complete
Funding source
Shiraz University of Medical Sciences
Expected recruitment start date
2015-02-20, 1393/12/01
Expected recruitment end date
2015-03-21, 1394/01/01
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
A prospective single arm study to assess the efficacy and safety of deferasirox 20 mg/kg BID in transfusion dependent beta-thalassemia patients unresponsive to current treatment with doses > 35 mg/kg QD or with intolerance to single dose therapy
Public title
A prospective single arm study to assess the efficacy and safety of deferasirox in transfusion dependent beta-thalassemia patients unresponsive to current treatment or with intolerance to single dose therapy
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion: Aged 2 years; Serum ferritin >2000 ng/ml and unresponsive to current treatment with doses > 35 mg/kg QD (decrease in ferritin level less than 10%); patients who did not tolerate single dose therapy due to adverse effects
Exclusion: Patients will be excluded from this trial if they have one of the following criteria: positive serology for HBS Ag , HCV Ab, HIV Ab. Abnormal liver or kidney function tests;An alanine aminotranferase (ALT) level greater than 250 U/L;serum creatinine level above the upper limit of normal, and a urinary protein-creatinine ratio of greater than 0.5 mg/mg); a history of ocular toxicity related to iron chelation therapy;uncontrolled systemic hypertention, a prolonged corrected QT interval; systemic infection within 10days prior to enrollment; gastrointestinal absorption problem ;Patients who found complication of chelation therapy during trial as well as breast feeding and pregnant women.
Age
No age limit
Gender
Both
Phase
3
Groups that have been masked
No information
Sample size
Target sample size:
40
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
Shiraz University of Medical Sciences
Street address
Zand Avenue,Shiraz University of Medical Sciences, Shiraz – Iran
City
Shiraz
Postal code
Approval date
2014-10-19, 1393/07/27
Ethics committee reference number
CT-P-9375-7645
Health conditions studied
1
Description of health condition studied
Thalassemia
ICD-10 code
D56.1
ICD-10 code description
Beta thalassaemia
Primary outcomes
1
Description
efficacy as reduction in serum ferritin levels as well as change in T2MRI.
Timepoint
Base line,three month,six month,nine month,end of study
Method of measurement
For better evaluation of serum ferritin changes during one year, we define an index: Mean of Relative Change of Serum Ferritin (MRC-SF) from the baseline levels that will be calculated as the percentage of difference between mean serum ferritin levels of each occasion from mean baseline serum ferritin levels.
Secondary outcomes
1
Description
gastrointestinal symptoms and skin rash fatal, acute, irreversible renal failure
Timepoint
Baseline,three months,six months,nine months,End of study
Method of measurement
Exact follow up of the patients examination,laboratory data
2
Description
safety
Timepoint
12 years
Method of measurement
exact follow up of the patients examination and laboratory data
Intervention groups
1
Description
Deferasirox 20 mg/kg BID ,During on year,all patients took defreasirox during the study
Category
Treatment - Drugs
Recruitment centers
1
Recruitment center
Name of recruitment center
Hematology Research Center
Full name of responsible person
Mozhgan Rezaei
Street address
Hematology Research Center, Nemazee Hospital, Zand Avenue, Shiraz
City
Shiraz
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Novartis
Full name of responsible person
Pooneh Heidari
Street address
# 2551, 10th Floor, KIAN Bldg, Valiasr Ave
City
Tehran
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Novartis
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
Full name of responsible person
Seazaneh Haghpanah
Position
Assistance professor of community medicine
Other areas of specialty/work
Street address
Hematology Research Center, Nemazee Hospital, Zand Avenue, Shiraz
City
Shiraz
Postal code
7193711351
Phone
+98 71 3612 5617
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
Mehran Karimi
Position
Professor of Pediatric Hematology-Oncology Hematology Research Center
Other areas of specialty/work
Street address
Hematology Research Center, Nemazee Hospital, Zand Avenue, Shiraz
City
Shiraz
Postal code
7193711351
Phone
+98 71 3612 5617
Fax
+98 71 3647 4298
Email
karimim@sums.ac.ir
Web page address
mkarimi820@gmail.com
Person responsible for updating data
Contact
Name of organization / entity
Hematology Research Center, Nemazee Hospital, Zand Avenue, Shiraz
Full name of responsible person
Tahereh Zarei
Position
Msc Microbiologist
Other areas of specialty/work
Street address
City
Postal code
Phone
+98 71 3612 5617
Fax
Email
zarei_t@sums.ac.ir
Web page address
Sharing plan
Deidentified Individual Participant Data Set (IPD)