Efficary and Safety of Luspatercept for Treatment of Anaemia in Transfusion-dependent β-thalassaemia in Southwest China

B-thal01

To assesse the efficacy and safety of luspatercept versus placebo in China patients with transfusion-dependent β-thalassaemia.

2024-01-01

2024-01-01

2024-11-30

Recruiting

10

Inclusion Criteria: 1. ≥18 years old; 2. A clear diagnosis of transfusion-dependent β-thalassemia (including αβ mixed type) with red blood cell transfusion ≤15u within 24 weeks before enrollment (one unit of red blood cell in overseas clinical research is 200-350ml packed red blood cells, which should be converted according to Chinese clinical practice); 3. Voluntarily participate in the study and sign the informed consent; Exclusion Criteria: 1. pregnant or lactating women; 2. Allergic to luspatercept and/or luspatercept for injection excipients; 3. Severe liver dysfunction: Liver enzymes (alanine aminotransferase ALT or aspartate aminotransferase AST) ≥ 3 times normal value.; 4. Severe renal injury: eGFR\<30 ml/min/1.73m3 or end-stage renal disease; 5. heart disease, New York Heart Association (NYHA) class 3 or higher heart failure, or the need for treatment Severe arrhythmia, or recent myocardial infarction within 6 months; 6. The patient had uncontrolled hypertension; 7. Patients with a history of deep vein thrombosis or stroke within 24 weeks prior to enrollment.; 8. Treatment with ESA, luspatercept, thalidomide or hydroxyurea within 12 weeks before enrollment; 9. Any significant other medical condition, laboratory abnormality, or mental illness; 10. Investigators deemed enrollment inappropriate.

{'studyType': 'INTERVENTIONAL', 'phases': ['NA'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': '5 cases with transfusion burden\\<7.5 units/24 weeks, and 5 cases with transfusion burden 7.5-15 units/24 weeks', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 10, 'type': 'ESTIMATED'}}

2023-12-22