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

Launched by HEMATOLOGY DEPARTMENT OF THE 920TH HOSPITAL · Dec 1, 2023

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ClinConnect Summary

This clinical trial is examining the effectiveness and safety of a drug called luspatercept in treating adult patients in China who have transfusion-dependent β-thalassemia, a blood disorder that requires regular blood transfusions. The study aims to compare the effects of luspatercept with a placebo (a substance with no active drug) to see how well it helps patients manage their condition.

To be eligible for this trial, participants must be at least 18 years old and have a confirmed diagnosis of transfusion-dependent β-thalassemia, having received up to 15 units of blood transfusions in the past 24 weeks. However, some individuals, such as pregnant or breastfeeding women, those with severe liver or kidney issues, or certain heart conditions, cannot participate. If you join the trial, you will receive either the luspatercept treatment or a placebo, and the study team will closely monitor your health throughout the process. This trial is currently recruiting participants, and your involvement could contribute to important research in managing this condition.

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Eligibility 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.