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

NCT05462548 | Unknown Phase 4 DMC

• 1 other identifier: SYSKY-2022-088-02
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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.

Conditions

Thalassemia Major; Transfusion-dependent Anemia

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (7)

• the incidence of 50% reduction in transfusion in any 12 consecutive weeks

  24 weeks

• Changes in mean cumulative transfusion volume from baseline at weeks 1-9, 1-12, and 1-24

  24 weeks

• The rate of transfusion free at any 8 and 12 consecutive weeks in the entire study population

  24 weeks

• Changes in mean serum ferritin (SF) levels in the population from baseline

  24 weeks

• Changes of cardiac and liver iron concentrations at 24 weeks from baseline (MRI T2*)

  24 weeks

Study Arms (1)

treatment group

EXPERIMENTAL

accept Luspatercept treatment

Drug: Luspatercept Injectable Product

Interventions

Luspatercept Injectable Product DRUG

1-1.25mg/kg every 3 weeks subcutaneous injection

Also known as: luspatercept

treatment group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• ≥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;

You may not qualify if:

• (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

Sponsors & Collaborators

• Sun Yat-sen University: lead

Study Sites (1)

The second affiliated hospital of Sun Yat-sen University

Guangzhou, Guangdong, 510000, China

RECRUITING

MeSH Terms

Conditions

beta-Thalassemia

Interventions

luspatercept

Condition Hierarchy (Ancestors)

Thalassemia; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Jianpei Fang, Dr.

  SunYat-senU2H

  STUDY DIRECTOR

Central Study Contacts

Jianpei Fang, MD

CONTACT

008613682290830; jpfang2005@163.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: 18 years old or above

Study Record Dates

• First Submitted: July 7, 2022
• First Posted: July 18, 2022
• Study Start: July 1, 2022
• Primary Completion: February 28, 2023
• Study Completion: April 30, 2023
• Last Updated: July 18, 2022

Record last verified: 2022-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, ICF, CSR
• Time Frame: end of the study to 2 years after the end of study

Locations

CN (1)