To determine the efficacy of thalidomide in the treatment of transfusion dependent thalassemia.
Design
Randomized Controlled Trial, single group study of 55 patients with non blinded outcome assessment. Non probability convenience sampling.
Settings and conduct
This study will be conducted at Department of Pediatrics, Sheikh Zayed Medical College/Hospital, Rahim Yar Khan. This study will be conducted after approval from Ethical Review Board (ERB) of Sheikh Zayed Hospital and University of Health Sciences, Lahore.
Participants/Inclusion and exclusion criteria
inclusion criteria: thalassemia patients aged 2- 15 years
Exclusion criteria: Patient with liver, renal, cardiac, pulmonary, neurological deficits or thrombotic episodes.
Intervention groups
Patients diagnosed with beta thalassemia diagnosed according to clinical characteristics and hemoglobin electrophoresis were included in this study according to the selection criteria. At the time of treatment the patients will interviewed and tested for pre-treatment variables which include hemoglobin levels, transfusion rate. All the patients will then be given 2-5mg/kg thalidomide for 6 months as prescribed by the consultant and these variables will be tested again. Response rate will be tested on the basis of operational definition. The principal investigator will follow up with all the patients and see post-treatment results.
Main outcome variables
This study will help us determine if we can use thalidomide in the treatment of beta thalassemia patients for reduction of transfusion, increase Hemoglobin level and response rate.
General information
Reason for update
Acronym
IRCT registration information
IRCT registration number:IRCT20220215054023N1
Registration date:2022-04-27, 1401/02/07
Registration timing:prospective
Last update:2022-04-27, 1401/02/07
Update count:0
Registration date
2022-04-27, 1401/02/07
Registrant information
Name
Muhammad Saleem
Name of organization / entity
Sheikh Zayed Medical college/Hospital, Rahim Yar khan, Punjab, Pakistan
Country
Pakistan
Phone
+92 68 9230109
Email address
profdrmsaleem@szmc.edu.pk
Recruitment status
Recruitment complete
Funding source
Expected recruitment start date
2022-06-01, 1401/03/11
Expected recruitment end date
2022-09-01, 1401/06/10
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Effectiveness of Thalidomide in Transfusion Dependent Thalassemia patients
Public title
Effectiveness of Thalidomide in Transfusion Dependent Thalassemia patients
Purpose
Health service research
Inclusion/Exclusion criteria
Inclusion criteria:
Beta thalassemia patients are diagnosed by clinical characteristics and by hemoglobin electrophoresis
Age 2 years to 15 years
Both Gender
Exclusion criteria:
Patients already undergoing treatment for induction of HbF
Patients with liver, renal, cardiac, pulmonary, neurological deficits or history of thrombotic episodes
Patients not giving consent to treatment
Age
From 2 years old to 15 years old
Gender
Both
Phase
N/A
Groups that have been masked
No information
Sample size
Target sample size:
55
Randomization (investigator's opinion)
Not 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
Institutional Review Board Sheikh Zayed Medical College/Hospital, Rahim Yar Khan
Street address
Sheikh Zayed Medical College/Hospital, Rahim Yar Khan
City
Rahim Yar Khan
Postal code
64200
Approval date
2022-03-04, 1400/12/13
Ethics committee reference number
441-/IRB/SZMC/SZH
Health conditions studied
1
Description of health condition studied
Beta thalassemia is genetic disorder which is caused by insertions and deletions in chromosome number 11. The disease is treated by blood transfusions which leads to iron over load and demand for chelation therapy. Some patients do not respond to this treatment and suffer from complications. Although treatment with hydroxyurea has leads to improved conditions by reducing transfusion demand, but some patients do not respond to this treatment. Hence, other treatment modalities which induce fetal hemoglobin are being explored. The aim of this study is to see the effectiveness of thalidomide in treating beta thalassemia patients
ICD-10 code
D56.1
ICD-10 code description
Beta thalassemia
Primary outcomes
1
Description
Response to treatment
Timepoint
before intervention and after 6 months after intervention
Method of measurement
become transfusion free after treatment
Secondary outcomes
1
Description
number of transfusion and hemoglobin level
Timepoint
before intervention and after 6 months of intervention
Method of measurement
number of transfusion and hemoglobin level
Intervention groups
1
Description
Intervention group: Patients diagnosed with beta thalassemia diagnosed according to clinical characteristics and hemoglobin electrophoresis were included in this study according to the selection criteria. At the time of treatment the patients will interviewed and tested for pre-treatment variables which include hemoglobin levels, transfusion rate. All the patients will then be given 2-5mg/kg thalidomide for 6 months as prescribed by the consultant and these variables will be tested again. Response rate will be tested on the basis of operational definition. The principal investigator will follow up with all the patients and see post-treatment results. Drug used in the study is Capsule Thalido(Thalidomide) with strength 50mg and 100mg will ne given as 2-5mg/kg in once daily dose for 6 months manufactured by ATCO laboratories
Category
Treatment - Drugs
2
Description
Control group: This group will not receive any type of treatment as assigned to interventional group. Effectiveness will be measured in interventional group only
Category
N/A
Recruitment centers
1
Recruitment center
Name of recruitment center
Department of Pediatrics (unit 2), Sheikh Zayed Medical College/Hospital, Rahim Yar Khan
Full name of responsible person
Prof. Dr. Muhammad Saleem Laghari
Street address
Department of Pediatrics (unit 2), Sheikh Zayed Medical College/Hospital, Rahim Yar Khan
City
Rahim Yar Khan
Postal code
64200
Phone
+92 333 8808633
Email
profdrmsaleem@szmc.edu.pk
Sponsors / Funding sources
1
Sponsor
Name of organization / entity
Sheikh Zayed Medical College/Hospital, Rahim Yar Khan
Full name of responsible person
Prof. Dr. Muhammad Saleem Laghari
Street address
Department of Pediatrics (unit 2), Sheikh Zayed Medical College/Hospital, Rahim Yar Khan
City
Rahim Yar Khan
Postal code
64200
Phone
+92 333 8808633
Email
profdrmsaleem@szmc.edu.pk
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Sheikh Zayed Medical College/Hospital, Rahim Yar Khan
Proportion provided by this source
100
Public or private sector
Public
Domestic or foreign origin
Domestic
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
Academic
Person responsible for general inquiries
Contact
Name of organization / entity
Department of Pediatrics (unit 2), Sheikh Zayed Medical College/Hospital, Rahim Yar Khan
Full name of responsible person
Prof. Dr. Muhammad Saleem Laghari
Position
Professor
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
Department of Pediatrics(unit 2), Sheikh Zayed Medical College/Hospital, Rahim Yar Khan
City
Rahim Yar Khan
Province
Punjab
Postal code
64200
Phone
+92 333 8808633
Email
profdrmsaleem@szmc.edu.pk
Person responsible for scientific inquiries
Contact
Name of organization / entity
Department of Pediatrics (unit 2), Sheikh Zayed Medical College/Hospital, Rahim Yar Khan
Full name of responsible person
Prof. Dr. Muhammad Saleem Laghari
Position
Professor
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
Department of Pediatrics (unit 2), Sheikh Zayed Medical College/Hospital, Rahim Yar Khan
City
Rahim Yar Khan
Province
Punjab
Postal code
64200
Phone
+92 333 8808633
Email
profdrmsaleem@szmc.edu.pk
Person responsible for updating data
Contact
Name of organization / entity
Department of Pediatrics (unit 2), Sheikh Zayed Medical College/Hospital, Rahim Yar Khan
Full name of responsible person
Prof. Dr. Saleem Laghari
Position
Professor
Latest degree
Specialist
Other areas of specialty/work
Pediatrics
Street address
Department of Pediatrics (unit 2), Sheikh Zayed Medical College/Hospital, Rahim Yar Khan
City
Rahim Yar Khan
Province
Punjab
Postal code
64200
Phone
+92 333 8808633
Email
profdrmsaleem@szmc.edu.pk
Sharing plan
Deidentified Individual Participant Data Set (IPD)
Yes - There is a plan to make this available
Study Protocol
Yes - There is a plan to make this available
Statistical Analysis Plan
Yes - There is a plan to make this available
Informed Consent Form
Yes - There is a plan to make this available
Clinical Study Report
Yes - There is a plan to make this available
Analytic Code
Yes - There is a plan to make this available
Data Dictionary
Yes - There is a plan to make this available
Title and more details about the data/document
Effectiveness of Thalidomide in Transfusion Dependent Thalassemia Patients
This study will be conducted at Department of Pediatrics, Sheikh Zayed Medical College/Hospital, Rahim Yar Khan. This study will be conducted after approval from Ethical Review Board (ERB) of Sheikh Zayed Hospital and University of Health Sciences, Lahore. Performa, attached with the synopsis, will be filled by the principle investigator. Patients diagnosed with beta thalassemia diagnosed according to clinical characteristics and hemoglobin electrophoresis were included in this study according to the selection criteria. At the time of treatment the patients will interviewed and tested for pre-treatment variables which include hemoglobin levels and transfusion rate. All the patients will then be given 2-5mg/kg thalidomide for 6 months as prescribed by the consultant hematologist and these variables will be tested again. Response rate will be tested on the basis of operational definition. The principal investigator will follow up with all the patients and see post-treatment results.
When the data will become available and for how long
Starting 6 months after publication
To whom data/document is available
For people working in academic institutions
Under which criteria data/document could be used
This data will de used for research purposes and also improve the treatment of beta thalassemia
From where data/document is obtainable
Contact the relevant person via phone number, email addresses or postal
What processes are involved for a request to access data/document
Just contact the relevant person via phone number, email addresses or postal to request to access data