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

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Brief Title

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

Official Title

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

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 Phase

Phase 4

Study Type

Interventional


Primary Outcome

A 33% reduction in the red cell (RBC) transfusion burden for any 12 consecutive weeks within 24 weeks of Luspatercept treatment compared with baseline

Secondary Outcome

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

Condition

Thalassemia Major

Intervention

Luspatercept Injectable Product

Study Arms / Comparison Groups

 treatment group
Description:  accept Luspatercept treatment

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

20

Start Date

July 2022

Completion Date

April 30, 2023

Primary Completion Date

February 28, 2023

Eligibility Criteria

        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.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Jianpei Fang, Dr., 008613682290830, [email protected]

Location Countries

China

Location Countries

China

Administrative Informations


NCT ID

NCT05462548

Organization ID

SYSKY-2022-088-02


Responsible Party

Principal Investigator

Study Sponsor

Sun Yat-sen University


Study Sponsor

Jianpei Fang, Dr., Study Director, SunYat-senU2H


Verification Date

July 2022