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

Beta-Thalassemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT06239389
• Other study ID #: NIBD/IRB-236/21-2021.
• Status: Completed
• Phase: Phase 2
• Start date: September 1, 2021
• Est. completion date: March 3, 2022

Study information

• Verified date: March 2024
• Source: National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 24
• Est. completion date: March 3, 2022
• Est. primary completion date: December 1, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 10 Years to 30 Years

Eligibility

Inclusion Criteria:

• Know case of beta thalassemia age between 10-30 years

Exclusion Criteria:

• Patients on any other haemoglobin F inducer or erythropoietin.

• Patients with co-morbidities like cardiopulmonary and neurological disease

• Pregnant, lactating women.

• Patient did not willing to take contraceptive measures.

• Participants with history of thrombosis.

Related Conditions & MeSH terms

• Beta-Thalassemia
• Thalassemia

Intervention

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.
• 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.

Locations

Country	Name	City	State
Pakistan	National Institute of Blood Diseases and Bone Marrow Transplantation	Karachi	Sindh

Sponsors (1)

Lead Sponsor	Collaborator
National Institute of Blood and Marrow Transplant (NIBMT), Pakistan

Country where clinical trial is conducted

Pakistan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	comparison of hemoglobin	Hemoglobin	06 months
Primary	comparison of red blood cells	Red blood cells	06 months
Primary	comparison of Mean corpuscular volume	Mean corpuscular volume	06 months
Primary	comparison of Mean Corpuscular Hemoglobin	Mean Corpuscular Hemoglobin	06 months
Primary	comparison of Mean Corpuscular Hemoglobin concentration	Mean Corpuscular Hemoglobin concentration	06 months
Primary	comparison of Indirect bilirubin	Indirect bilirubin	06 months
Primary	comparison of Total Bilirubin	Total Bilirubin	06 months
Primary	comparison of Serum glutamic pyruvic transaminase	Serum glutamic pyruvic transaminase	06 months
Primary	comparison of glutamic-oxaloacetic transaminase	glutamic-oxaloacetic transaminase	06 months
Primary	comparison of white blood cells	white blood cells	06 months
Primary	comparison of platelets	Platelets	06 months
Primary	comparison of urea	Urea	06 months
Primary	comparison of creatinine	Creatinine	06 months
Primary	comparison of Lactate dehydrogenase baseline and after the completion of study.	Lactate dehydrogenase	06 months
Primary	comparison of tranfusion frequency baseline and after the completion of study.	Transfusion interval	06 months