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

NCT ID: NCT06239389

Last Updated: 2024-03-12

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-09-01
• Study Completion Date: 2022-03-03

Brief 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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

hydroxyurea

Hydroxyurea is available in oral dosage form which is capsule. frequency: once a day duration: at every 24 hours dose: 15/mg/kg/day.

Group Type: ACTIVE_COMPARATOR

Thalidomide 100mg

Intervention Type: DRUG

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.

Thalidomide

Thalidomide is available in oral dosage form which is capsule. frequency: once a day at night duration: at every 24 hours dose:50 mg / day (in patients \>10-13 years) while the adult dose was 100 mg /day (age \>13 Years)

Group Type: EXPERIMENTAL

Hydroxyurea

Intervention Type: DRUG

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.

Interventions

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.

Intervention Type: 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.

Intervention Type: DRUG

Other Intervention Names

thalido; Hydrea

Eligibility Criteria

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.

• Minimum Eligible Age: 10 Years
• Maximum Eligible Age: 30 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

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

OTHER_GOV

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Safia Mehmood khan, MPHIL

Role: PRINCIPAL_INVESTIGATOR

National institute if blood diseases and bone marrow transplantation

Locations

National Institute of Blood Diseases and Bone Marrow Transplantation

Karachi, Sindh, Pakistan

Countries

Pakistan

Other Identifiers

NIBD/IRB-236/21-2021.

Identifier Type: -

Identifier Source: org_study_id