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: 0
• Participant gender: All

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).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Dr.Saqib Hussain Ansari
• Treatments: Hydroxyurea

Criteria

Inclusion Criteria:

• Patients with homozygous beta thalassemia major diagnosed on the basis of genetic mutation

• Beta thalassemia intermedia (patients carrying homozygous, heterozygous or compound heterozygous beta thalassemia genes) (HbS, HbE)

• Those patients who do not have baseline HbE suggestive of beta thalassemia, genetic mutations will be performed to confirm the diagnosis.

• Age: 6 months and onwards

• Gender: Either

• Able to understand study procedures and to comply with them for the entire length of the study.

• Provide written informed consent if aged 18 years and above and if minor that is below 18 years (Parental consent will be taken)

Exclusion Criteria:

• Chronic liver disease, renal failure, history of stroke

• Participants who have developed immune hemolytic anemia

• Spleenomegaly (liver and spleen >5 cm below coastal margin

• If allergic or sensitive to Hydroxyurea or its ingredients

• Patients on immunosuppressants including Azathiopine or any other drug causing bone marrow suppression, Hepatitis C treatment that may cause red cell suppression and NSAIDS

• Inability or unwillingness of individual or legal guardian/representative to give written informed consent.