The Efficacy and Safety of HYDROXYUREA in Management of Beta Thalassemia Patients in Karachi Pakistan
Status:
Unknown status
Trial end date:
2018-06-30
Target enrollment:
Participant gender:
Summary
Objectives
Primary objectives:
- To determine the efficacy of Hydroxyurea in the study participants.
- Hypothesis: The study will result in either maintenance or rise in hemoglobin as
compared to the control treatment.
Secondary objectives:
- To determine the compliance of Hydroxyurea in study participants.
- To determine the safety of Hydroxyurea in the study participants. Design and Outcomes
An open label randomized controlled trial to test the efficacy and safety of Hydroxyurea on
beta thalassemia major patients. It is a six months study. Findings of physical examination,
vital sign variables, laboratory variables and ultrasound at baseline, during and end of the
study will be listed. Schedule of intervention is mentioned in section 6.1. later in the
protocol.
Interventions and Duration Hydroxyurea will be given to the participants in intervention arm
along with the standard treatment if thalassemia (blood transfusion and iron chelation
therapy) and the control arm will receive the standard treatment (blood transfusion and iron
chelation therapy) only. Each participant will be followed up for 6 months after initiating
the intervention. Intervention will be given for 6 months or until the participant withdraws
from the study or due to any reason, the investigator stops the intervention.
Sample Size and Population This pilot study will be done on 100 patients initially.
Stratified randomization will be done on the basis of presence of Xmn polymorphism. And the
study population will be assigned to intervention or control arm randomly through a computer
software (randomizer.org).