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

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.

Study Overview

• Status: Recruiting
• Conditions: Thalassemia Major; Transfusion-dependent Anemia
• Intervention / Treatment: Drug: Luspatercept Injectable Product

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 Type: Interventional
• Enrollment (Anticipated): 20
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Jianpei Fang, MD; Phone Number: 008613682290830; Email: jpfang2005@163.com

Study Locations

• China
  • Guangdong
    • Guangzhou, Guangdong, China, 510000
      • Recruiting
      • The Second Affiliated Hospital of Sun Yat-sen University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: treatment group; accept Luspatercept treatment	Drug: Luspatercept Injectable Product; 1-1.25mg/kg every 3 weeks subcutaneous injection; Other Names: luspatercept

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
The incidence of adverse events within 24 consecutive weeks of Luspatercept treatment	incidence of reported adverse events	24weeks

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Sun Yat-sen University
• Investigators: Study Director: Jianpei Fang, Dr., SunYat-senU2H

Study record dates

Study Major Dates

• Study Start (Anticipated): July 1, 2022
• Primary Completion (Anticipated): February 28, 2023
• Study Completion (Anticipated): April 30, 2023

Study Registration Dates

• First Submitted: July 7, 2022
• First Submitted That Met QC Criteria: July 15, 2022
• First Posted (Actual): July 18, 2022

Study Record Updates

• Last Update Posted (Actual): July 18, 2022
• Last Update Submitted That Met QC Criteria: July 15, 2022
• Last Verified: July 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Genetic Diseases, Inborn; Anemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Hemoglobinopathies; Thalassemia; beta-Thalassemia; Physiological Effects of Drugs; Immunologic Factors; Hematinics; Immunoglobulin Fc Fragments; Luspatercept
• Other Study ID Numbers: SYSKY-2022-088-02

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD Plan Description: privacy information of individual participant is not available
• IPD Sharing Time Frame: end of the study to 2 years after the end of study
• IPD Sharing Supporting Information Type: Study Protocol; Informed Consent Form (ICF); Clinical Study Report (CSR)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes