A Phase 3 interventional study of Thalidomide and Danazol in Beta-Thalassemia and Thalassemia, sponsored by Khyber Medical University Peshawar. Recruiting at 1 site in Pakistan. Open to participants aged 5 Years to 15 Years. Per ClinicalTrials.gov, last updated 2026-10-21.
Sponsored by Khyber Medical University Peshawar · Phase 3, Interventional, and Treatment
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.
This is a two-arm, parallel-group, open-label, randomized controlled superiority trial. It will enroll 172 participants aged 5 to 15 years with transfusion-dependent beta-thalassemia major who have had an inadequate response to at least 6 months of adequately dosed thalidomide. Participants will be randomly assigned 1:1 to thalidomide monotherapy or thalidomide plus danazol plus prednisolone. The primary outcomes are transfusion independence, defined as no red blood cell transfusion for at least 8 consecutive weeks with hemoglobin at least 9.0 g/dL; mean change in pre-transfusion hemoglobin from baseline to 6 months; and safety and tolerability graded by CTCAE v5.0. Secondary outcomes include transfusion burden, hematological improvement, health-related quality of life using TranQOL, and exploratory associations of HBB mutation class and HbF-modifier genotypes (XmnI, BCL11A, HBS1L-MYB) with treatment response. Follow-up will be 9 to 12 months. The trial will be conducted at the Blood Care Clinic, Pak International Medical College, and Peshawar General Hospital, Hayatabad, Phase 5, Peshawar, Pakistan. The trial will be registered before the first participant is enrolled.
312 studies on the registry are indexed under beta-Thalassemia; 73 are open to participants now.
This study's planned enrollment of 172 is above the median of 38 across 215 interventional studies indexed under beta-Thalassemia.
Browse beta-Thalassemia studies →Khyber Medical University Peshawar is the lead sponsor of 54 studies on the registry; 13 are open to participants now.
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In this treatment regimen, Thalidomide and Danazol are administered at a dosage of 100 mg orally three times daily for 30 days each cycle. Prednisolone is given at a dosage of 5 mg orally twice daily for 10 days each month. Supportive care includes Famotidine, folic acid, and vitamin B12. This treatment plan is continued for a duration of 9 to 12 months.
Drug: Thalidomide · Drug: Danazol · Drug: Prednisolone
Thalidomide should be taken orally at a dose of 1 to 5 mg/kg at night, with dosage titrated upward every 2 to 4 weeks based on response and tolerance. Clopidogrel and aspirin should be taken together for thromboprophylaxis. This regimen should continue for 9 to 12 months.
Drug: Thalidomide
Initial oral capsule dose is 1 to 5 mg/kg taken once daily at night, with adjustments made every 2 to 4 weeks based on response and tolerance. The treatment is administered in both arms for a duration of 9 to 12 months.
Also known as: Thalomid, alpha-(N-phthalimido)glutarimide
Oral capsule of 100 mg taken three times daily, continuously for 30 days per cycle. This medication is administered only in the combination arm.
Also known as: Danocrine
Oral tablet of 5 mg twice daily for 10 days each month, only administered in the combination arm.
Also known as: Pediapred, Orapred
Transfusion independence
Proportion of participants who remain free from red blood cell transfusion for at least 8 consecutive weeks while maintaining hemoglobin greater than or equal to 9.0 g/dL.
Time frame: Assessed continuously from randomization through final study visit (Month 9 to 12).
Hemoglobin response
Mean change in pre-transfusion hemoglobin (g/dL) from baseline to 6 months.
Time frame: Baseline and Month 6.
Safety and tolerability
Incidence and severity of treatment-emergent adverse events, including hepatic toxicity, renal dysfunction, hyperglycemia, peripheral neuropathy, and thromboembolic events, graded according to CTCAE v5.0. Incidence and severity of treatment-emergent adverse events, including hepatic toxicity, renal dysfunction, hyperglycemia, peripheral neuropathy, and thromboembolic events, graded according to CTCAE v5.0. Incidence and severity of treatment-emergent adverse events, including hepatic toxicity, renal dysfunction, hyperglycemia, peripheral neuropathy, and thromboembolic events, graded according to CTCAE v5.0.
Time frame: Assessed at every scheduled visit (Months 1, 3, 6, 9, and final visit).
Plan to share: Yes — De-identified individual participant data (IPD), the full trial protocol, statistical analysis plan, informed consent form, and analytic code will be made available to other researchers upon reasonable request after publication of the primary results. Data sharing will comply with KMU data-sharing policy and patient confidentiality requirements.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Analytic code
Time frame: Data will become available after publication of the primary results and will remain available for at least 5 years.
Access criteria: Access will be granted upon reasonable request to the Principal Investigator, subject to approval by the Institutional Ethics Committee and a signed data-use agreement. Requests must comply with KMU data-sharing policy and patient confidentiality requirements.
Started Oct 2026 (actual). Primary completion is estimated for Apr 2027, 6 months after the start.
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From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Studies of the drugs tested here: Danazol · Prednisolone
Khyber Medical University Peshawar