| CTRI Number |
CTRI/2024/08/071925 [Registered on: 05/08/2024] Trial Registered Prospectively |
| Last Modified On: |
23/01/2026 |
| Post Graduate Thesis |
Yes |
| Type of Trial |
Interventional |
|
Type of Study
|
Drug |
| Study Design |
Single Arm Study |
|
Public Title of Study
|
To Study the Potential role of drug Hyrdoxyurea in Thalassemia in reducing the burden of frequent blood transfusion in children of Southern Rajasthan |
|
Scientific Title of Study
|
To Study the role of Hydroxyurea in Transfusion Dependent Beta Thalassemia Major in children aged Less than 18 years in Southern Rajasthan - A Pre Post Interventional Study |
| Trial Acronym |
NIL |
|
Secondary IDs if Any
|
| Secondary ID |
Identifier |
| NIL |
NIL |
|
|
Details of Principal Investigator or overall Trial Coordinator (multi-center study)
|
| Name |
Dr Dhruv Malik |
| Designation |
Postgraduate Resident |
| Affiliation |
R.N.T Medical college |
| Address |
Department Of Paediatrics
R.N.T Medical college
Near Court Choraha
Udaipur
Rajasthan
Udaipur
RAJASTHAN
313001
India |
| Phone |
7349058358 |
| Fax |
|
| Email |
dhruv10.dm@gmail.com |
|
Details of Contact Person
Scientific Query
|
| Name |
Dr Mohammad Asif |
| Designation |
Head and Professor |
| Affiliation |
R.N.T Medical college |
| Address |
Department Of Paediatrics
R.N.T Medical college
Near Court Choraha
Udaipur
Rajasthan
Udaipur
RAJASTHAN
313001
India |
| Phone |
9785056044 |
| Fax |
|
| Email |
rntianasif@gmail.com |
|
Details of Contact Person
Public Query
|
| Name |
Dr Mohammad Asif |
| Designation |
Head and Professor |
| Affiliation |
R.N.T Medical college |
| Address |
Department Of Paediatrics
R.N.T Medical college
near Court Choraha
Udaipur
Rajasthan
Udaipur
RAJASTHAN
313001
India |
| Phone |
9785056044 |
| Fax |
|
| Email |
rntianasif@gmail.com |
|
|
Source of Monetary or Material Support
|
| R.N.T Medical College Udaipur 313001,Rajasthan |
|
|
Primary Sponsor
|
| Name |
RNT Medical College |
| Address |
R.N.T Medical college
near Court Choraha
Udaipur 313001
Rajasthan |
| Type of Sponsor |
Government medical college |
|
|
Details of Secondary Sponsor
|
|
|
Countries of Recruitment
|
India |
|
Sites of Study
|
| No of Sites = 1 |
| Name of Principal
Investigator |
Name of Site |
Site Address |
Phone/Fax/Email |
| Dr Dhruv Malik |
R.N.T Medical College |
Room Number 509,Thalassemia Ward
Bal Chikitsalaya
Department Of Paediatrics
Udaipur
RAJASTHAN |
7349058358
dhruv10.dm@gmail.com |
|
|
Details of Ethics Committee
|
| No of Ethics Committees= 1 |
| Name of Committee |
Approval Status |
| R.N.T Medical College and Controller and attached Hospital, Udaipur |
Approved |
|
|
Regulatory Clearance Status from DCGI
|
|
|
Health Condition / Problems Studied
|
| Health Type |
Condition |
| Patients |
(1) ICD-10 Condition: D561||Beta thalassemia, |
|
|
Intervention / Comparator Agent
|
| Type |
Name |
Details |
| Intervention |
Hydroxyurea |
Capsule Hydroxyurea 20Mg/kg/Day once daily for a period of 6 Months with concurrent Folic acid, Calcium supplements.Patient will be advised to take Capsule Hydroxyurea Empty stomach preferably in the morning.
|
| Comparator Agent |
NOT APPLICABLE |
NOT APPLICABLE |
|
|
Inclusion Criteria
|
| Age From |
1.00 Year(s) |
| Age To |
18.00 Year(s) |
| Gender |
Both |
| Details |
All Known case of Beta Thallasemia Major less than 18 years of age visiting Thalassemia Day Care Centre for regular Blood Transfusion/Followup |
|
| ExclusionCriteria |
| Details |
Children having other causes of Anemia,Pre- exisitng Renal disease ,Pre- existing hepatic disease, Thromboctyopenia, Neutropenia, children having Chronic hepatitis B,C HIV ,HBsAg seropositivity,Not giving Informed consent were excluded from this study |
|
|
Method of Generating Random Sequence
|
Not Applicable |
|
Method of Concealment
|
Not Applicable |
|
Blinding/Masking
|
Not Applicable |
|
Primary Outcome
|
| Outcome |
TimePoints |
| To study the Efficacy of Hydroxyurea on Reduction of Need for frequent Blood Transfusion Requirements in children of Beta thalassemia Major |
6 MONTHS |
|
|
Secondary Outcome
|
| Outcome |
TimePoints |
| To study the effect of Hydroxyurea in reduction of serum Ferritin Levels and overall burden of need for frequent Blood transfusion |
6 MONTHS |
|
|
Target Sample Size
|
Total Sample Size="70"
Sample Size from India="70"
Final Enrollment numbers achieved (Total)= "35"
Final Enrollment numbers achieved (India)="35" |
|
Phase of Trial
|
Phase 3/ Phase 4 |
|
Date of First Enrollment (India)
|
07/08/2024 |
| Date of Study Completion (India) |
04/08/2025 |
| Date of First Enrollment (Global) |
Date Missing |
| Date of Study Completion (Global) |
04/08/2025 |
|
Estimated Duration of Trial
|
Years="0"
Months="6"
Days="0" |
Recruitment Status of Trial (Global)
Modification(s)
|
Not Applicable |
| Recruitment Status of Trial (India) |
Completed |
|
Publication Details
|
N/A |
|
Individual Participant Data (IPD) Sharing Statement
|
Will individual participant data (IPD) be shared publicly (including data dictionaries)?
Response - NO
|
|
Brief Summary
|
This study Aims to ascertain the role of Hydroxyurea as a Potential Pharmacological Agent that can be used in Transfusion Dependat Beta Thalassemia Major in children aged Less than 18 years in Southern Rajasthan. Hydroxyurea is a DNA antimetabolite is being used extensively for many years in Sickle cell disease and Beta Thalassemia Intermedia successfully. Hydroxyurea is known to induce Gamma globin chain synthesis in Human erythroid cells. In Beta Hemoglobinopthaies there is deficiency of beta globin chains and excess of alpha chains. By induction of gamma chain synthesis this excess of gamma chains can combine with alpha chains can form fetal hemoglobin(HBF). This mechanism to induce Gamma chain synthesis and increase HbF levels is the Mechanism for its use Possible in Beta Hemoglobinopathies . This study is a type of Pre- Post intervention Study to be conducted in Thalassemia Day Care Centre in RNT medical college Udaipur . After satisfying inclusion exclusion criteria enrollment of children will be done by collecting Baseline Data like Mean Pre transfusion Hemoglobin levels , Number of blood transfusion needed in last 6 months , Serum ferritin levels . After intervention of this drug Hydroxyurea at dose 20/mg/day Once Daily with concurrent administration of folic acid and calcium supplements effects on Mean pretransfusion hemoglobin levels , serum ferritin levels, reduction in need for transfusion and increase in duration between each transfusion will be measured . For now the cornerstone of cure for Beta thalassemia is Allogenic bone marrow(BMT) transplantation and factors such as high cost , inability to find suitable donor , possible chances of rejection leads to many difficulties in management .Hence this study can help to ascertain the role of hydroxyurea as a potential pharmacological agent which can be used as a adjunct to management of beta thalassemia and help in reduction of morbidity and reduce overall burden of disease and improve the Quality of life of children suffering from this disease. |