Thalassaemia
Conditions
Interventions
Experimental Arm:25mg/d, participant takes the medicine after dinner and the dose can be further adjusted if any adverse reactions are observed
Experimental Arm:50mg/d, participant takes the medicine after dinner and the dose can be further adjusted if any adverse reactions are observed
Experimental Arm:75mg/d, participant takes the medicine after dinner and the dose can be further adjusted if any adverse reactions are observed
Placebo Arm:Placebo, participant takes the placebo after dinner and the dose can be further adjusted if any adverse reactions are observed.
Sponsors
Wuzhou Gongren Hospital, Guangxi Zhuang Autonomous Region
Eligibility
Sex/Gender
All
Age
10 Years to 18 Years
Inclusion criteria
Inclusion criteria: The inclusion criteria are: 1. The patient is clinically and genetically diagnosed with transfusion-dependent beta-thalassemia (TDT). 2. Transfusion dependence is defined as receiving at least eight transfusions or 100 ml per kilogram of body weight of leucoreduced packed RBCs per year. 3. Patient age is 10-18 years old. 4. ECOG performance score is 0-2 points. 5. The written informed consent is signed before enrollment. 6. The hemoglobin concentration and content of reticulocytes are stable 4 weeks after the gastrointestinal bleeding stops. 7.TDT patients with G-6PD deficiency can be considered for enrollment if no hemolysis-inducing factor is present within 3 months before enrollment.
Exclusion criteria
Exclusion criteria: Patients are excluded from this study if they meet any of the following criteria: 1. Those who have received EPO, Yisui Shengxue Granules, hydroxyurea, thalidomide, decitabine, azacitidine, or butyrate treatment 3 months before enrollment; 2. Patients with vitamin B12 and folic acid deficiency; 3. Patients with severe cardiopulmonary or cerebrovascular diseases; 4. Patients with abnormal liver or kidney function; 5. Patients with a history of arteriovenous thrombosis 3 months before enrollment; 6. Patients with uncorrected anemia caused by other reasons; 7. Mental patients; 8. Pregnant or lactating women and those who are unwilling to consider using contraceptive methods; 9. Allergic to the drug; 10. Patients who are participating in other clinical trials at the same time; 11. The participants are not qualified for this study assessed by the investigators.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hemoglobin;Volume of blood transfusion; | — |
Secondary
| Measure | Time frame |
|---|---|
| Height and weight;Hemoglobin electrophoresis;Indirect bilirubin;Lactate dehydrogenase;Total bilirubin;Reticulocyte count;Peripheral nucleated red blood cell count;Aspartate aminotransferase;Alanine aminotransferase;Creatinine;Urea nitrogen;Erythrocyte life span; | — |
Countries
China
Contacts
Public ContactJiangming Chen
Wuzhou Gongren Hospital, Guangxi Zhuang Autonomous Region
Outcome results
None listed