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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT06239389
Other study ID # NIBD/IRB-236/21-2021.
Secondary ID
Status Completed
Phase Phase 2
First received
Last updated
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
Health authority
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.

Study Design


Related Conditions & MeSH terms


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
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