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

Thalassemia Major Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05462548
• Other study ID #: SYSKY-2022-088-02
• Status: Recruiting
• Phase: Phase 4
• Start date: July 2022
• Est. completion date: April 30, 2023

Study information

• Verified date: July 2022
• Source: Sun Yat-sen University
• Contact: Jianpei Fang, MD
• Phone: 008613682290830
• Email: jpfang2005[@]163.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: April 30, 2023
• Est. primary completion date: February 28, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

-=18 years old

• The patient was clearly diagnosed as transfusion-dependent ß -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.

Related Conditions & MeSH terms

• beta-Thalassemia
• Thalassemia
• Thalassemia Major
• Transfusion-dependent Anemia

Intervention

Drug:
• Luspatercept Injectable Product
1-1.25mg/kg every 3 weeks subcutaneous injection

Locations

Country	Name	City	State
China	The second affiliated hospital of Sun Yat-sen University	Guangzhou	Guangdong

Sponsors (1)

Lead Sponsor	Collaborator
Sun Yat-sen University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	A 33% reduction in the red cell (RBC) transfusion burden for any 12 consecutive weeks within 24 weeks of Luspatercept treatment compared with baseline	the incidence of 33% reduction in RBC transfusion	24 weeks
Primary	The incidence of adverse events within 24 consecutive weeks of Luspatercept treatment	incidence of reported adverse events	24weeks
Secondary	the incidence of 50% reduction in transfusion in any 12 consecutive weeks	the incidence of 50% reduction in transfusion	24 weeks
Secondary	Changes in mean cumulative transfusion volume from baseline at weeks 1-9, 1-12, and 1-24	volume of transfusion	24 weeks
Secondary	The rate of transfusion free at any 8 and 12 consecutive weeks in the entire study population	volume of transfusion	24 weeks
Secondary	Changes in mean serum ferritin (SF) levels in the population from baseline	serum ferritin (SF) levels	24 weeks
Secondary	Changes of cardiac and liver iron concentrations at 24 weeks from baseline (MRI T2*)	MR T2*	24 weeks
Secondary	changes in reticulocyte levels from baseline during treatment	blood routine	24 weeks
Secondary	changes in fetal hemoglobin levels from baseline during treatment	Hemoglobin electrophoresis	24weeks