Clinical trial NCT06239389: Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study.
Official title: Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Beta-Thalassemia
Keywords: thalidomide,
hydroxyurea
hemoglobin F inducers
beta thalassemia
Lead sponsor: National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Sponsor class: OTHER_GOV
First posted: 2024-02-02
Enrollment: 24
Sex: ALL
Minimum age (years): 10
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Patients were divided into 2 groups, one received hydroxyurea second group received thalidomide.
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: comparison of hemoglobin
timeFrame: 06 months
measure: comparison of red blood cells
timeFrame: 06 months
measure: comparison of Mean corpuscular volume
timeFrame: 06 months
measure: comparison of Mean Corpuscular Hemoglobin
timeFrame: 06 months
measure: comparison of Mean Corpuscular Hemoglobin concentration
timeFrame: 06 months
measure: comparison of Indirect bilirubin
timeFrame: 06 months
measure: comparison of Total Bilirubin
timeFrame: 06 months
measure: comparison of Serum glutamic pyruvic transaminase
timeFrame: 06 months
measure: comparison of glutamic-oxaloacetic transaminase
timeFrame: 06 months
measure: comparison of white blood cells
timeFrame: 06 months
measure: comparison of platelets
timeFrame: 06 months
measure: comparison of urea
timeFrame: 06 months
measure: comparison of creatinine
timeFrame: 06 months
measure: comparison of Lactate dehydrogenase baseline and after the completion of study.
timeFrame: 06 months
measure: comparison of tranfusion frequency baseline and after the completion of study.
timeFrame: 06 months
Secondary outcomes:
Other outcomes:
Locations:
city: Karachi
country: Pakistan
facility: National Institute of Blood Diseases and Bone Marrow Transplantation
state: Sindh
Study officials:
affiliation: National institute if blood diseases and bone marrow transplantation
name: Safia Mehmood khan, MPHIL
role: PRINCIPAL_INVESTIGATOR
References: