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

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

Status and phase: Completed; Phase 2

Conditions: Beta-Thalassemia

Treatments: Drug: Hydroxyurea; Drug: Thalidomide 100mg

Study type: Interventional

Funder types: Other

Identifiers

NCT06239389

NIBD/IRB-236/21-2021.

Details and patient eligibility

About

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.

Enrollment: 24 patients

Sex: All

Ages: 10 to 30 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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24 participants in 2 patient groups

hydroxyurea

Active Comparator group

Description:

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

Treatment:

Drug: Thalidomide 100mg

Thalidomide

Experimental group

Description:

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)

Treatment:

Drug: Hydroxyurea