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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05462548
Recruitment Status : Recruiting
First Posted : July 18, 2022
Last Update Posted : July 18, 2022
Sponsor:
Information provided by (Responsible Party):
Jianpei FANG, 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.

Condition or disease Intervention/treatment Phase
Thalassemia Major Transfusion-dependent Anemia Drug: Luspatercept Injectable Product Phase 4

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: 18 years old or above
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study
Estimated Study Start Date : July 2022
Estimated Primary Completion Date : February 28, 2023
Estimated Study Completion Date : April 30, 2023


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




Primary Outcome Measures :
  1. A 33% reduction in the red cell (RBC) transfusion burden for any 12 consecutive weeks within 24 weeks of Luspatercept treatment compared with baseline [ Time Frame: 24 weeks ]
    the incidence of 33% reduction in RBC transfusion

  2. The incidence of adverse events within 24 consecutive weeks of Luspatercept treatment [ Time Frame: 24weeks ]
    incidence of reported adverse events


Secondary Outcome Measures :
  1. the incidence of 50% reduction in transfusion in any 12 consecutive weeks [ Time Frame: 24 weeks ]
    the incidence of 50% reduction in transfusion

  2. Changes in mean cumulative transfusion volume from baseline at weeks 1-9, 1-12, and 1-24 [ Time Frame: 24 weeks ]
    volume of transfusion

  3. The rate of transfusion free at any 8 and 12 consecutive weeks in the entire study population [ Time Frame: 24 weeks ]
    volume of transfusion

  4. Changes in mean serum ferritin (SF) levels in the population from baseline [ Time Frame: 24 weeks ]
    serum ferritin (SF) levels

  5. Changes of cardiac and liver iron concentrations at 24 weeks from baseline (MRI T2*) [ Time Frame: 24 weeks ]
    MR T2*

  6. changes in reticulocyte levels from baseline during treatment [ Time Frame: 24 weeks ]
    blood routine

  7. changes in fetal hemoglobin levels from baseline during treatment [ Time Frame: 24weeks ]
    Hemoglobin electrophoresis



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05462548


Contacts
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Contact: Jianpei Fang, MD 008613682290830 jpfang2005@163.com

Locations
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China, Guangdong
The second affiliated hospital of Sun Yat-sen University Recruiting
Guangzhou, Guangdong, China, 510000
Contact: Jianpei Fang, M.D.    +8620-81332003    jpfang2005@163.com   
Principal Investigator: Jianpei Fang, M.D.         
Sponsors and Collaborators
Sun Yat-sen University
Investigators
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Study Director: Jianpei Fang, Dr. SunYat-senU2H
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Responsible Party: Jianpei FANG, Professor, Sun Yat-sen University
ClinicalTrials.gov Identifier: NCT05462548    
Other Study ID Numbers: SYSKY-2022-088-02
First Posted: July 18, 2022    Key Record Dates
Last Update Posted: July 18, 2022
Last Verified: July 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: privacy information of individual participant is not available
Supporting Materials: Study Protocol
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Time Frame: end of the study to 2 years after the end of study

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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
Additional relevant MeSH terms:
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Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Luspatercept
Immunoglobulin Fc Fragments
Hematinics
Immunologic Factors
Physiological Effects of Drugs