NCT06239389

Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study.

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (actual)
• Sponsor: National Institute of Blood and Marrow Transplant (NIBMT), Pakistan · Other Government
• Sex: All
• Age: 10 Years – 30 Years
• Healthy volunteers: Not accepted

Summary

The goal of this study is to compare the effectiveness and safety of two hemoglobin F inducer. This is single centered interventional pilot study is to compare the efficacy and safety parameters in beta thalassemia patients. As this is a pilot study, the investigator took a small number of patients. The Sample size was calculated by the World health organization sample size calculator. After screening 39 patients and 24 patients were eligible for enrollment in this study. The main objective was to evaluate safety of both drugs in genetic disorder like thalassemia. for safety evaluation, hematological parameters were evaluated that includes total bilirubin , indirect bilirubin, Serum Glutamate Pyruvate Transaminase, serum glutamic-oxaloacetic transaminase, urea, creatinine and lactate dehydrogenase were monitored . Moreover to evaluate the efficacy of drug, hematological parameters that includes hemoglobin, red blood cells , nucleated red blood cells , reticulocytes count, Red blood cells indices ( mean corpuscular hemoglobin, mean corpuscular volume and mean corpuscular hemoglobin concentration) , white blood cells and platelets were done. Another important parameters to evaluate the efficacy of hemoglobin F inducer is transfusion frequency. Test were done at baseline and after completion of study means after 06 months.

Conditions

• Beta-Thalassemia

Interventions

Type	Name	Description
DRUG	Thalidomide 100mg	Thalidomide is available in capsulated form in the strength of 100 mg and 50 mg. It was given according to the weight of the patient. Due to somnolence, it was advised to give at night only.
DRUG	Hydroxyurea	Hydroxyurea is available in capsulated form under the brand of hydrea. Its available strength is 500 mg . Dosing depends upon weight of patient, that is 15mg/kg/day. so patients unable to take full capsule due to low weight, was advised to take hydroxyurea syrup.

Timeline

• Start date: 2021-09-01
• Primary completion: 2021-12-01
• Completion: 2022-03-03
• First posted: 2024-02-02
• Last updated: 2024-03-12

Locations

1 site across 1 country: Pakistan