Transfusion-Dependent Beta-Thalassemia Hb E - Thalidomide - Beta-thalassemia/Hb E
Conditions
Interventions
Thalidomide 50 mg orally once daily plus best supportive care (BSC), Weeks 0-12
may continue thalidomide in open-label extension Weeks 12-24.,Matching placebo orally once daily plus BSC, Weeks 0-12
may change to receive thalidomide 50 mg orally once daily in open-label extension Weeks 12-24.
Experimental Drug,Placebo Comparator Drug
Thalidomide,Placebo
Sponsors
Thai Society of Hematology
Faculty of Medicine, Khon Kaen University
Eligibility
Sex/Gender
All
Age
13 Years to 75 Years
Inclusion criteria
Inclusion criteria: 1. Patients with Beta-thalassemia Hemoglobin E (DNA confirmed), blood-dependent, requiring 6-20 units of blood in the past 24 weeks. 2. Age more than 13 years or older and weight more than 25 kg. 3. Poor response to blood transfusion, including presence of red blood cell alloantibodies or autoantibodies. 4. Inability to receive regular blood due to travel or insufficient supply. 5. Severe iron overload (serum ferritin more than 2,500 ng/mL) despite adequate chelation therapy. 6. Willing to use contraception during the study and for 6 months after stopping Thalidomide. 7. Partner must consent to pregnancy prevention if applicable.
Exclusion criteria
Exclusion criteria: 1. Pregnant or planning to become pregnant. 2. Received erythropoiesis-stimulating agents within less than 3 months prior to study enrollment. 3. Abnormal liver function (ALT and/or AST more than 2.5 times normal) or abnormal kidney function (CrCl <30 mL/min). 4. History of blood clotting risk, including venous thromboembolism, stroke, coronary artery disease, hyperviscosity, genetic clotting risk, or arrhythmia. 5. History of neurological issues, heart disease, lung dysfunction, bone marrow failure, and/or ongoing infection.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hematologic response rate 6 months Proportion of participants achieving the predefined response criterion | — |
Secondary
| Measure | Time frame |
|---|---|
| Adverse events 0-6 months Number and proportion of participants with any adverse events / serious adverse events, over (0-6 months) | — |
Countries
Thailand
Contacts
Public ContactNattiya Teawtrakul
Khon Kaen University
Outcome results
None listed