Back to resultsThe Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study
Primary Purpose
Thalassemia Major, Transfusion-dependent Anemia
Status
Recruiting
Phase
Phase 4
Locations
China
Study Type
Interventional
Intervention
Luspatercept Injectable Product
Sponsored by
About this trial
This is an interventional treatment trial for Thalassemia Major
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.
Sites / Locations
- The second affiliated hospital of Sun Yat-sen UniversityRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
treatment group
Arm Description
accept Luspatercept treatment
Outcomes
Primary Outcome Measures
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
The incidence of adverse events within 24 consecutive weeks of Luspatercept treatment
incidence of reported adverse events
Secondary Outcome Measures
the incidence of 50% reduction in transfusion in any 12 consecutive weeks
the incidence of 50% reduction in transfusion
Changes in mean cumulative transfusion volume from baseline at weeks 1-9, 1-12, and 1-24
volume of transfusion
The rate of transfusion free at any 8 and 12 consecutive weeks in the entire study population
volume of transfusion
Changes in mean serum ferritin (SF) levels in the population from baseline
serum ferritin (SF) levels
Changes of cardiac and liver iron concentrations at 24 weeks from baseline (MRI T2*)
MR T2*
changes in reticulocyte levels from baseline during treatment
blood routine
changes in fetal hemoglobin levels from baseline during treatment
Hemoglobin electrophoresis
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05462548
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
Study Type
Interventional
2. Study Status
Record Verification Date
July 2022
Overall Recruitment Status
Recruiting
Study Start Date
July 2022 (Anticipated)
Primary Completion Date
February 28, 2023 (Anticipated)
Study Completion Date
April 30, 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Sun Yat-sen University
4. Oversight
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
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thalassemia Major, Transfusion-dependent Anemia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Model Description
18 years old or above
Masking
None (Open Label)
Allocation
N/A
Enrollment
20 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
treatment group
Arm Type
Experimental
Arm Description
accept Luspatercept treatment
Intervention Type
Drug
Intervention Name(s)
Luspatercept Injectable Product
Other Intervention Name(s)
luspatercept
Intervention Description
1-1.25mg/kg every 3 weeks subcutaneous injection
Primary Outcome Measure Information:
Title
A 33% reduction in the red cell (RBC) transfusion burden for any 12 consecutive weeks within 24 weeks of Luspatercept treatment compared with baseline
Description
the incidence of 33% reduction in RBC transfusion
Time Frame
24 weeks
Title
The incidence of adverse events within 24 consecutive weeks of Luspatercept treatment
Description
incidence of reported adverse events
Time Frame
24weeks
Secondary Outcome Measure Information:
Title
the incidence of 50% reduction in transfusion in any 12 consecutive weeks
Description
the incidence of 50% reduction in transfusion
Time Frame
24 weeks
Title
Changes in mean cumulative transfusion volume from baseline at weeks 1-9, 1-12, and 1-24
Description
volume of transfusion
Time Frame
24 weeks
Title
The rate of transfusion free at any 8 and 12 consecutive weeks in the entire study population
Description
volume of transfusion
Time Frame
24 weeks
Title
Changes in mean serum ferritin (SF) levels in the population from baseline
Description
serum ferritin (SF) levels
Time Frame
24 weeks
Title
Changes of cardiac and liver iron concentrations at 24 weeks from baseline (MRI T2*)
Description
MR T2*
Time Frame
24 weeks
Title
changes in reticulocyte levels from baseline during treatment
Description
blood routine
Time Frame
24 weeks
Title
changes in fetal hemoglobin levels from baseline during treatment
Description
Hemoglobin electrophoresis
Time Frame
24weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Jianpei Fang, MD
Phone
008613682290830
Email
jpfang2005@163.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jianpei Fang, Dr.
Organizational Affiliation
SunYat-senU2H
Official's Role
Study Director
Facility Information:
Facility Name
The second affiliated hospital of Sun Yat-sen University
City
Guangzhou
State/Province
Guangdong
ZIP/Postal Code
510000
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jianpei Fang, M.D.
Phone
+8620-81332003
Email
jpfang2005@163.com
First Name & Middle Initial & Last Name & Degree
Jianpei Fang, M.D.
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing 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
Learn more about this trial
The Safety and Efficiency of Luspatercept in Chinese Adults With Transfusion Dependent β-thalassemia: a Real-world Study
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