Adding danazol and prednisolone to thalidomide for children with transfusion-dependent beta-thalassemia
Compares thalidomide alone with thalidomide plus two drugs in children with beta-thalassemia.
Plain-language title generated from the registry record
Registry title: Combination Therapy With Danazol, Prednisolone, and Thalidomide for Thalidomide-Refractory Transfusion-Dependent β-Thalassemia
Key facts
- Where
- Peshawar, PakistanSee the site
- Phase
- Phase 3
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 5 and 15 years old?
Source: NCT07884773, who can take part
Where in Pakistan
- Pak International Medical CollegePeshawar, PakistanTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Khyber Medical University Peshawar
- Trial code
- NCT07884773
- Record updated
- 21 October 2026
- Original description
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.Read full original descriptionShow less
Safety and Efficacy of Combination Therapy (Danazol + Prednisolone + Thalidomide) in Thalidomide-Refractory Transfusion-Dependent β-Thalassemia Patients: A Prospective Randomized Controlled Clinical Trial
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.
Inclusion: 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: 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.