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NCT Number: NCT06239389

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

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.

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Key information

Conditions

Beta-Thalassemia Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Thalassemia

Age range

10 year–30 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

National Institute of Blood Diseases and Bone Marrow Transplantation

Karachi, Sindh, 75300, Pakistan

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Thalidomide 100mg

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.

Other names: thalido

Hydroxyurea

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.

Other names: Hydrea

Primary outcomes

  1. comparison of hemoglobin

    Time frame: 06 months

    Hemoglobin

  2. comparison of red blood cells

    Time frame: 06 months

    Red blood cells

  3. comparison of Mean corpuscular volume

    Time frame: 06 months

    Mean corpuscular volume

  4. comparison of Mean Corpuscular Hemoglobin

    Time frame: 06 months

    Mean Corpuscular Hemoglobin

  5. comparison of Mean Corpuscular Hemoglobin concentration

    Time frame: 06 months

    Mean Corpuscular Hemoglobin concentration

  6. comparison of Indirect bilirubin

    Time frame: 06 months

    Indirect bilirubin

  7. comparison of Total Bilirubin

    Time frame: 06 months

    Total Bilirubin

  8. comparison of Serum glutamic pyruvic transaminase

    Time frame: 06 months

    Serum glutamic pyruvic transaminase

  9. comparison of glutamic-oxaloacetic transaminase

    Time frame: 06 months

    glutamic-oxaloacetic transaminase

  10. comparison of white blood cells

    Time frame: 06 months

    white blood cells

  11. comparison of platelets

    Time frame: 06 months

    Platelets

  12. comparison of urea

    Time frame: 06 months

    Urea

  13. comparison of creatinine

    Time frame: 06 months

    Creatinine

  14. comparison of Lactate dehydrogenase baseline and after the completion of study.

    Time frame: 06 months

    Lactate dehydrogenase

  15. comparison of tranfusion frequency baseline and after the completion of study.

    Time frame: 06 months

    Transfusion interval

Sponsors and collaborators

Lead sponsor

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

Other Gov

Registry information

Important dates

Study start
2021
Primary completion
2021
Study completion
2022
First posted
Feb 2, 2024
Registry last updated
Mar 12, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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