ß-Thalassaemia
Conditions
Interventions
Luspatercept + Thalidomide Group:Luspatercept: 1.0 mg/kg, subcutaneous (SC) administration, every 21 days
Thalidomide: 1.0 mg/kg, oral (PO) administration, once nightly
Sponsors
The 923rd Hospital of the Joint Logistics Support Force of the People's Liberation Army
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Subjects must be aged 18 years or older at the time of signing the informed consent form. 2. Subjects must understand and voluntarily participate in this study, signing the informed consent form. 3. The subject must have a documented diagnosis of ß-thalassaemia or HbE/ß-thalassaemia, having received 12 weeks of single-agent treatment with Rotexip prior to enrolment without achieving the expected therapeutic effect. 4. The patient's disease status must be stable, with an ECOG performance status score of 0-2. 5. Adequate organ function levels: (1) Echocardiography demonstrating left ventricular ejection fraction (LVEF) >=40%; (2) Serum total bilirubin 60 mL/min.
Exclusion criteria
Exclusion criteria: 1. History of uncontrolled epilepsy, psychiatric disorders, or other mental illnesses; 2. Pregnant or breastfeeding women, and reproductive-age participants unwilling to use contraception; 3. Concurrent severe cardiopulmonary diseases, including history of serious cardiovascular conditions such as heart function classification >= Class III or myocardial infarction; Severe respiratory disease requiring high-flow non-invasive ventilation or ventilator-assisted ventilation; 4. Concurrent severe hepatic or renal impairment, such as total bilirubin >5× upper limit of normal, alanine transaminase (ALT) or aspartate transaminase (AST) >3× upper limit of normal, or estimated glomerular filtration rate (eGFR) <30 mL/min; 5. Concurrent malignant tumours (excluding cervical carcinoma in situ, etc.); 6. History of autoimmune diseases or immunodeficiency disorders; 7. Anaemia due to other causes; 8. Uncorrected bleeding disorders; 9. Patients currently receiving any other investigational medicinal products; 10. Previous allogeneic haematopoietic stem cell transplantation or gene therapy; 11. Presence of other diseases or conditions likely to interfere with the study protocol; 12. Participants deemed unsuitable for this study by the investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of non-transfusion-dependent beta thalassemia patients with mean hemoglobin (Hb) increase >=1.5 g/dL from baseline at Week 12;Proportion of transfusion-dependent beta thalassemia patients with reduction in red blood cell (RBC) transfusion burden >=50% from baseline at Week 12; | — |
Secondary
| Measure | Time frame |
|---|---|
| Proportion of non-transfusion-dependent beta thalassemia patients with mean hemoglobin (Hb) increase >=1.0 g/dL from baseline at Week 12;Proportion of transfusion-dependent beta thalassemia patients with reduction in red blood cell (RBC) transfusion burden >=33% from baseline at Week 12;Change in serum ferritin at Week 12; | — |
Countries
China
Contacts
Public ContactYin Xiaolin
The 923rd Hospital of the People's Liberation Army
Outcome results
None listed