Observing the effects of luspatercept in treating adult β-thalassemia patients in China

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

This study is testing if a new drug called luspatercept can help adults in China with β-thalassemia who need regular blood transfusions feel better and improve their hemoglobin levels.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 10 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Hematology department of the 920th hospital (Academic / other)
• Locations: 1 site (Kunming, Yunnan)
• Trial ID: NCT06164821 on ClinicalTrials.gov

What this trial studies

This clinical observation focuses on the efficacy and safety of luspatercept in adult patients with transfusion-dependent β-thalassemia in China. The study is a prospective, multicenter, open-label design that involves administering luspatercept subcutaneously every three weeks for 24 weeks. Patients are monitored for hemoglobin levels and potential adverse reactions throughout the treatment period. The study aims to provide insights into the drug's effectiveness in a specific population, building on previous findings from similar studies in Asian patients.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with a clear diagnosis of transfusion-dependent β-thalassemia requiring regular blood transfusions.

Not a fit: Patients with severe liver or renal dysfunction, heart disease, or those who are pregnant or lactating may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce the need for blood transfusions in patients with β-thalassemia.

How similar studies have performed: Previous studies, such as the BELIEVE study, have shown promising results for luspatercept in similar populations, indicating potential for success in this study.

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.

Where this trial is running

Kunming, Yunnan

• 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China — Kunming, Yunnan, China (Recruiting)

Study contacts

• Study coordinator: Chaoen Zhen, MD

Conditions Effect of Drug; luspatercept; β-Thalassemia