Combining Thalidomide with Danazol and Prednisolone for difficult beta-thalassemia in children

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 3, two-arm, open-label randomized controlled trial enrolling 172 children aged 5 to 15 with transfusion-dependent beta-thalassemia who had inadequate response to thalidomide. Participants are randomized 1:1 to continue thalidomide alone or receive thalidomide plus danazol and prednisolone, with the aim of reducing transfusion needs and improving hemoglobin while monitoring safety. Primary outcomes include transfusion independence (no transfusions for 8 weeks with Hb ≥9.0 g/dL), change in pre-transfusion hemoglobin at 6 months, and safety/tolerability per CTCAE v5.0. Secondary outcomes cover transfusion burden, hematologic response, quality of life, and potential genetic associations with response. Follow-up lasts 9–12 months at sites in Peshawar, Pakistan.

Why it matters

Who can join: the full rules

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.

Where it's running

• Pak International Medical College
  Peshawar, Khyber Pakhtunkhwa, Pakistan Recruiting

Study contacts

• Principal investigator: Syeda Nayab Imtiaz, MBBS — Institute of Pathology and Diagnostic Medicine, Khyber Medical University, Peshawar
• Study coordinator: Syeda Nayab Imtiaz, MBBS
• Email: dr.nayabimtiaz@hotmail.com
• Phone: +923369966014

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.