Clinical trial NCT05462548: 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
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Thalassemia Major
Transfusion-dependent Anemia
Lead sponsor: Sun Yat-sen University
Sponsor class: OTHER
First posted: 2022-07-18
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: 18 years old or above
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: A 33% reduction in the red cell (RBC) transfusion burden for any 12 consecutive weeks within 24 weeks of Luspatercept treatment compared with baseline
timeFrame: 24 weeks
measure: The incidence of adverse events within 24 consecutive weeks of Luspatercept treatment
timeFrame: 24weeks
Secondary outcomes:
measure: the incidence of 50% reduction in transfusion in any 12 consecutive weeks
timeFrame: 24 weeks
measure: Changes in mean cumulative transfusion volume from baseline at weeks 1-9, 1-12, and 1-24
timeFrame: 24 weeks
measure: The rate of transfusion free at any 8 and 12 consecutive weeks in the entire study population
timeFrame: 24 weeks
measure: Changes in mean serum ferritin (SF) levels in the population from baseline
timeFrame: 24 weeks
measure: Changes of cardiac and liver iron concentrations at 24 weeks from baseline (MRI T2*)
timeFrame: 24 weeks
measure: changes in reticulocyte levels from baseline during treatment
timeFrame: 24 weeks
measure: changes in fetal hemoglobin levels from baseline during treatment
timeFrame: 24weeks
Other outcomes:
Locations:
city: Guangzhou
country: China
facility: The second affiliated hospital of Sun Yat-sen University
state: Guangdong
Study officials:
affiliation: SunYat-senU2H
name: Jianpei Fang, Dr.
role: STUDY_DIRECTOR
References: