The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study

Phase 4

• Conditions: Transfusion-dependent Anemia; Thalassemia Major
• Interventions: Drug: Luspatercept Injectable Product

• Registration Number: NCT05462548
• Lead Sponsor: Sun Yat-sen University

Brief Summary

This is a prospective, single-arm, open-label study. Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, including blood transfusion and iron removal, based on the clinician's judgment and practice. The main objective of this study was to evaluate the efficacy and safety of Luspatercept in the treatment of adult patients with transfusion-dependent β -thalassaemia in Chinese clinical practice, and to provide evidence reference for subsequent clinical use.

Detailed Description

Twenty adult patients with transfusion-dependent β -thalassemia will be enrolled to receive Luspatercept with optimal supportive care, being injected subcutaneously every 3 weeks ,by the dose of 1-1.25 mg/kg. The main objective of this study was to evaluate the efficacy and safety of Luspatercept in the treatment of adult patients with transfusion-dependent β -thalassaemia in Chinese clinical practice, and to provide evidence reference for subsequent clinical use.

Study Timeline

• Status Verified: 2022-07
• Study Start: 2022-07
• Study First Submitted: 2022-07-07
• Study First Submitted QC: 2022-07-15
• Last Update Submitted: 2022-07-15
• Study First Posted: 2022-07-18
• Last Update Posted: 2022-07-18
• Primary Completion: 2023-02-28
• Study Completion: 2023-04-30

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

-≥18 years old

• The patient was clearly diagnosed as transfusion-dependent β -thalassemia, the blood transfusion period was ≤60 days, and the red blood cell infusion volume was not less than 3-12U in the 12 weeks before enrollment, which could provide the red blood cell infusion volume record in the 12 weeks before enrollment
• Patients who were treated with thalidomide before enrollment were required to stop the treatment for more than 4 weeks, and their hemoglobin decreased to below 90g/L. Blood transfusion was required, and records of blood transfusion within 12 weeks could be provided
• Voluntarily participate in the study and sign the informed consent;

Exclusion Criteria

• (1) Pregnant or lactating women
• persons known to be allergic to Luspatercept and/or Luspatercept excipients for injection
• Severe liver dysfunction (liver enzyme (ALT or AST) ≥3 TIMES ULN)
• Severe renal impairment (eGFR < 30 ml/min/1.73m3 or patients with end-stage renal disease)
• Heart disease, heart failure classified as Class 3 or higher by the New York Heart Association (NYHA), or severe arrhythmia requiring treatment, or recent myocardial infarction within 6 months of randomization.
• The patient has uncontrolled hypertension. According to NCI CTCAE version 5.0, controlled hypertension in this protocol is considered to be ≤1.
• Patients with a history of deep vein thrombosis or stroke within 24 weeks prior to enrollment
• Erythropoietin, Luspatercept or hydroxyurea were used 8 weeks before enrollment
• Any other significant medical condition, laboratory abnormality, or mental illness that the Investigator deems inappropriate for inclusion.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
treatment group	Luspatercept Injectable Product	accept Luspatercept treatment

Primary Outcome Measures

Name	Time	Method
The incidence of adverse events within 24 consecutive weeks of Luspatercept treatment	24weeks	incidence of reported adverse events
A 33% reduction in the red cell (RBC) transfusion burden for any 12 consecutive weeks within 24 weeks of Luspatercept treatment compared with baseline	24 weeks	the incidence of 33% reduction in RBC transfusion

Secondary Outcome Measures

Name	Time	Method
the incidence of 50% reduction in transfusion in any 12 consecutive weeks	24 weeks	the incidence of 50% reduction in transfusion
The rate of transfusion free at any 8 and 12 consecutive weeks in the entire study population	24 weeks	volume of transfusion
Changes in mean serum ferritin (SF) levels in the population from baseline	24 weeks	serum ferritin (SF) levels
changes in reticulocyte levels from baseline during treatment	24 weeks	blood routine
Changes in mean cumulative transfusion volume from baseline at weeks 1-9, 1-12, and 1-24	24 weeks	volume of transfusion
Changes of cardiac and liver iron concentrations at 24 weeks from baseline (MRI T2*)	24 weeks	MR T2\*
changes in fetal hemoglobin levels from baseline during treatment	24weeks	Hemoglobin electrophoresis

Trial Locations

Locations (1)

The second affiliated hospital of Sun Yat-sen University; 🇨🇳 Guangzhou, Guangdong, China