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Combination Therapy With Danazol, Prednisolone, and Thalidomide for Thalidomide-Refractory Transfusion-Dependent ฮฒ-Thalassemia

RecruitingPhase 3Ages 5 Years - 15 YearsAllKhyber Medical University Peshawar
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In plain words

What the trial is, and whether it could suit you

This study will compare two treatments for children with beta-thalassemia who need regular blood transfusions and have not responded well to thalidomide. One group will continue thalidomide alone. The other group will receive thalidomide plus danazol and prednisolone. The study will check whether the combination treatment reduces the need for blood transfusions, raises hemoglobin levels, and is safe. About 172 children aged 5 to 15 will be assigned by chance to one of the two groups. They will be followed for 9 to 12 months.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: * Age 5 to 15 years with confirmed transfusion-dependent beta-thalassemia major. * Documented non-response to adequately dosed thalidomide therapy for at least 6 months. * Pre-transfusion hemoglobin less than 9.0 g/dL with continued transfusion dependence (more than 2 units per month). * Adequate baseline clinical and laboratory assessment for safe study treatment. * Stable transfusion and iron-chelation regimen according to the protocol. * Written informed consent from parent/legal guardian and age-appropriate assent from the participant, with willingness to comply with study procedures and follow-up. Exclusion Criteria: * Previous allogeneic hematopoietic stem cell or bone marrow transplantation. * Diabetes mellitus or clinically significant uncontrolled metabolic disease. * Active severe infection or uncontrolled hepatic, renal, or cardiovascular disease. * Significant pre-existing peripheral neuropathy or history of clinically significant thromboembolic disease. * Known hypersensitivity to any study medication.
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What it tests

  • Thalidomide
  • Danazol
  • Prednisolone
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Where

1 site
Pak International Medical College
Peshawar, Pakistan
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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