Clinical Observation of Luspatercept in Treatment of Chinese Adult β-thalassaemia Patients With TD β-thalassemia

• Status: Recruiting
• Trial end date: 2024-11-30
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hematology department of the 920th hospital
• Treatments: Luspatercept

Criteria

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.