Ignite Creation Date:
2024-10-26 @ 3:40 PM
Last Modification Date:
2024-10-26 @ 3:40 PM
Study NCT ID:
NCT06606886
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-09-11
Brief Title:
Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This clinical trial aims to evaluate and compare the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major and correlate respondersnon-responders with the single nucleotide polymorphism mutations SNPs by DNA analysis
The main question to answer is
Is there a significant difference in the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major
Participants will be divided into two treatment groups Participants of group A will receive Tab Metformin 500mgdose twice daily orally for one year Participants of group B will receive Tab Hydroxyurea 10-20mgkg once daily orally for one year
At the end of the study Investigators will compare the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major and correlate respondersnon-responders with the single nucleotide polymorphism mutations SNPs by DNA analysis
The main question to answer is
Is there a significant difference in the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major
Participants will be divided into two treatment groups Participants of group A will receive Tab Metformin 500mgdose twice daily orally for one year Participants of group B will receive Tab Hydroxyurea 10-20mgkg once daily orally for one year
At the end of the study Investigators will compare the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major and correlate respondersnon-responders with the single nucleotide polymorphism mutations SNPs by DNA analysis
Detailed Description:
The study will be conducted after obtaining approval from the Institutional Review Board IRB at Bahria University Health Sciences Campus Karachi BUHSCK and the Faculty Research Committee FRC After obtaining written informed consent 48 participants in each group with beta-thalassemia major will be selected from the National Institute of Blood Diseases and Bone Marrow Transplantation Participants will be randomized into two treatment groups using computer-generated randomization
Group A participants will receive Metformin 500 mgdose twice daily orally for one year Group B participants will receive Hydroxyurea 10-20 mgkg once daily orally for one year
Dose escalation therapy will be implemented to improve participant compliance and tolerance The first month will not be included in the study period for each participant A general physical examination will be conducted monthly Blood pressure mm Hg will be assessed using a sphygmomanometer The pulse rate beats per minute will be determined by palpating the radial artery and the Body Mass Index kgm2 will be calculated for each participant
Dietary Counseling
People with thalassemia are advised to consume vitamin C-rich foods like vegetables and fruits to prevent depletion from iron overload They should also include dairy products in their diet to counter calcium depletion while avoiding high-iron foods such as beef liver spinach and dates
SF Short-form Questionnaire
All participants will complete the SF-36 a 36-item Short-Form Health Survey at baseline 6 months and 12 months The SF-36 is a widely used tool for evaluating overall health status consisting of 36 items across 8 domains The scores contribute to two main components the physical component summary PCS and the mental component summary MCS The SF-36 is employed to assess Health-Related Quality of Life HRQoL in both the general population and individuals with various medical conditions including beta-thalassemia major β-TM allowing for comparisons of HRQoL across different illnesses and health statuses
I Baseline InvestigationsParameters at day zero
Baseline investigations will include Complete Blood Count CBC Random Blood Sugar RBS Erythrocyte Sedimentation Rate ESR Serum Creatinine Serum Ferritin Liver Function Tests LFTs HCV RNA test Fetal Hemoglobin HbF level Abdominal Ultrasound Dual-Energy X-ray Absorptiometry DEXA Scan and DNA mutations Health-Related Quality of Life HRQoL will be assessed by the SF-36 Questionnaire at baseline 6 months and 12 months
II Monthly Follow-up for Clinical Features
Only CBC will be performed monthly and any adverse effects related to the drugs will be inquired about
III At 3 Months
CBC RBS Serum Creatinine and LFTs will be assessed for any adverse effects on blood kidneys and liver
IV At 6 12 Months
All baseline investigations except DNA mutations will be repeated
V At 9 Months
Random Blood Sugar Serum Ferritin and Serum Creatinine will be assessed
Adverse Effects of Drugs
Adverse effects will be explained to each participant as follows A Metformin nausea vomiting abdominal bloating diarrhea etc B Hydroxyurea cytopenia nausea vomiting abdominal pain rashes etc
DNA mutations will be analyzed using Amplification Refractory Mutation System-Polymerase Chain Reaction ARMS-PCR in responders and non-responders
Data will be collected using the Consent Form and SF-36 Questionnaire Data analysis will be performed using IBM Statistical Package for the Social Sciences SPSS version 27 A p-value 005 will be considered statistically significant Descriptive statistics mean SD will be used for continuous variables while categorical variables will be presented as frequencies and percentages To determine the mean difference a repeated-measures two-way ANOVA within each group will be used For correlation analysis an independent T-test will be employed
Group A participants will receive Metformin 500 mgdose twice daily orally for one year Group B participants will receive Hydroxyurea 10-20 mgkg once daily orally for one year
Dose escalation therapy will be implemented to improve participant compliance and tolerance The first month will not be included in the study period for each participant A general physical examination will be conducted monthly Blood pressure mm Hg will be assessed using a sphygmomanometer The pulse rate beats per minute will be determined by palpating the radial artery and the Body Mass Index kgm2 will be calculated for each participant
Dietary Counseling
People with thalassemia are advised to consume vitamin C-rich foods like vegetables and fruits to prevent depletion from iron overload They should also include dairy products in their diet to counter calcium depletion while avoiding high-iron foods such as beef liver spinach and dates
SF Short-form Questionnaire
All participants will complete the SF-36 a 36-item Short-Form Health Survey at baseline 6 months and 12 months The SF-36 is a widely used tool for evaluating overall health status consisting of 36 items across 8 domains The scores contribute to two main components the physical component summary PCS and the mental component summary MCS The SF-36 is employed to assess Health-Related Quality of Life HRQoL in both the general population and individuals with various medical conditions including beta-thalassemia major β-TM allowing for comparisons of HRQoL across different illnesses and health statuses
I Baseline InvestigationsParameters at day zero
Baseline investigations will include Complete Blood Count CBC Random Blood Sugar RBS Erythrocyte Sedimentation Rate ESR Serum Creatinine Serum Ferritin Liver Function Tests LFTs HCV RNA test Fetal Hemoglobin HbF level Abdominal Ultrasound Dual-Energy X-ray Absorptiometry DEXA Scan and DNA mutations Health-Related Quality of Life HRQoL will be assessed by the SF-36 Questionnaire at baseline 6 months and 12 months
II Monthly Follow-up for Clinical Features
Only CBC will be performed monthly and any adverse effects related to the drugs will be inquired about
III At 3 Months
CBC RBS Serum Creatinine and LFTs will be assessed for any adverse effects on blood kidneys and liver
IV At 6 12 Months
All baseline investigations except DNA mutations will be repeated
V At 9 Months
Random Blood Sugar Serum Ferritin and Serum Creatinine will be assessed
Adverse Effects of Drugs
Adverse effects will be explained to each participant as follows A Metformin nausea vomiting abdominal bloating diarrhea etc B Hydroxyurea cytopenia nausea vomiting abdominal pain rashes etc
DNA mutations will be analyzed using Amplification Refractory Mutation System-Polymerase Chain Reaction ARMS-PCR in responders and non-responders
Data will be collected using the Consent Form and SF-36 Questionnaire Data analysis will be performed using IBM Statistical Package for the Social Sciences SPSS version 27 A p-value 005 will be considered statistically significant Descriptive statistics mean SD will be used for continuous variables while categorical variables will be presented as frequencies and percentages To determine the mean difference a repeated-measures two-way ANOVA within each group will be used For correlation analysis an independent T-test will be employed
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None