Thalassemia Major, Transfusion-dependent Anemia
Conditions
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.
Interventions
1-1.25mg/kg every 3 weeks subcutaneous injection
Sponsors
Sun Yat-sen University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
18 years old or above
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| A 33% reduction in the red cell (RBC) transfusion burden for any 12 consecutive weeks within 24 weeks of Luspatercept treatment compared with baseline | 24 weeks | the incidence of 33% reduction in RBC transfusion |
| The incidence of adverse events within 24 consecutive weeks of Luspatercept treatment | 24weeks | incidence of reported adverse events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The rate of transfusion free at any 8 and 12 consecutive weeks in the entire study population | 24 weeks | volume of transfusion |
| Changes in mean serum ferritin (SF) levels in the population from baseline | 24 weeks | serum ferritin (SF) levels |
| the incidence of 50% reduction in transfusion in any 12 consecutive weeks | 24 weeks | the incidence of 50% reduction in transfusion |
| changes in reticulocyte levels from baseline during treatment | 24 weeks | blood routine |
| changes in fetal hemoglobin levels from baseline during treatment | 24weeks | Hemoglobin electrophoresis |
| Changes of cardiac and liver iron concentrations at 24 weeks from baseline (MRI T2*) | 24 weeks | MR T2\* |
| Changes in mean cumulative transfusion volume from baseline at weeks 1-9, 1-12, and 1-24 | 24 weeks | volume of transfusion |
Countries
China
Contacts
Primary ContactJianpei Fang, MD
Outcome results
None listed