An Exploratory Study on the Safety and Efficacy of Luspatercept in the Treatment of Anemia Following Allogeneic Hematopoietic Stem Cell Transplantation for Acute Leukemia
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 46
- 试验地点
- 1
- 主要终点
- Proportion of patients with reduced transfusion requirement
研究概览
简要总结
This is a single-center, single-arm, prospective exploratory study designed to evaluate the safety and efficacy of luspatercept for the treatment of anemia following allogeneic hematopoietic stem cell transplantation in patients with acute leukemia. A total of 46 eligible subjects are planned to be enrolled. The primary efficacy endpoint is the proportion of patients achieving a hemoglobin increase of ≥1.5 g/dL at 8 weeks post-transplantation (without transfusion support) compared to baseline (the average of 3 days prior to the first dose). Secondary endpoints include assessing the impact of luspatercept on time to hematopoietic engraftment (neutrophil and platelet) and recording safety indicators such as adverse events, graft-versus-host disease, and infections. Subjects will receive subcutaneous luspatercept at 1 mg/kg on day +7 and day +28 post-transplantation.
详细描述
This is a single-center, single-arm, prospective, exploratory clinical study. The study plans to enroll 46 acute leukemia patients who have undergone allo-HSCT. All enrolled subjects will receive subcutaneous luspatercept at a dose of 1 mg/kg on day +7 (±3 days) and day +28 (±3 days) post-transplantation.
Study Procedures:
The study includes a screening period, treatment follow-up visits (for drug administration), and observation follow-up visits. Intensive follow-up within 2 months post-transplant includes assessments of vital signs, physical examinations, serial hematological and biochemical tests, transfusion records, engraftment status, T-lymphocyte subset analysis (day 28), and disease-related evaluations (e.g., minimal residual disease testing, week 8). Adverse events will be continuously monitored and recorded.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 70 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinically diagnosed with acute leukemia.
- •Has undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT).
- •Expected to survive for more than 3 months from the date of signing the informed consent form.
- •Willing and able to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures, and has provided written informed consent.
- •Women of childbearing potential must have a negative serum pregnancy test within prior to enrollment. Female subjects who are surgically sterile or postmenopausal for at least 2 years are considered not of childbearing potential. Male and female subjects of reproductive potential must agree to use effective contraception throughout the study period.
- •Hemoglobin (Hb) level < 80 g/L.
排除标准
- •1. Inadequate organ function, defined as:
- •Creatinine clearance < 60 mL/min;
- •Left ventricular ejection fraction (LVEF) < 55%;
- •Oxygen saturation (SpO₂) < 92% on room air;
- •Total bilirubin > 2 × the upper limit of normal (ULN);
- •Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 × ULN.
- •Poorly controlled hypertension, defined as repeated systolic blood pressure ≥140 mmHg and/or diastolic blood pressure ≥90 mmHg despite adequate antihypertensive therapy, or a history of hypertensive crisis or hypertensive encephalopathy.
- •3. History of other malignancies (except for acute leukemia), unless the subject has been disease-free for ≥5 years. However, subjects with the following history/concomitant conditions are eligible:
- •Basal or squamous cell carcinoma of the skin;
- •Carcinoma in situ of the cervix;
- •Carcinoma in situ of the breast;
- •Incidental histological finding of prostate cancer (T1a or T1b as defined by the TNM staging system);
- •Solid tumors considered by the investigator to have no other known active disease.
- •4. Major surgery within 8 weeks prior to enrollment. The subject must have fully recovered from any prior surgery.
- •5. History of cerebrovascular accident (including ischemic, embolic, and hemorrhagic), transient ischemic attack, deep vein thrombosis (including proximal and distal), pulmonary or arterial embolism, arterial thrombosis, or other venous thrombosis within 6 months prior to enrollment. Note: Prior superficial thrombophlebitis is not an exclusion criterion.
- •6. Uncontrolled epilepsy, history of cerebral ischemia/hemorrhage, cerebellar disease, or other active central nervous system disorders.
- •7. Cardiac disease within 6 months prior to enrollment, including: myocardial infarction, uncontrolled angina, acute decompensated heart failure, New York Heart Association (NYHA) Class III-IV heart failure, or uncontrolled arrhythmia (as determined by the investigator).
- •8. Uncontrolled active systemic fungal, bacterial, or viral infection.
- •Evidence of human immunodeficiency virus (HIV) infection, active hepatitis B, and/or active hepatitis C.
- •10. History of pure red cell aplasia (PRCA) and/or anti-erythropoietin antibody.
- •11. Any condition or concomitant medication that may interfere with the interpretation of study data.
- •12. Known hypersensitivity to luspatercept or any of its excipients.
研究组 & 干预措施
Single-Arm Group
All participants receive subcutaneous luspatercept at 1 mg/kg on day +7 and day +28 post-transplantation.
干预措施: Luspatercept (Drug)
结局指标
主要结局
Proportion of patients with reduced transfusion requirement
时间窗: 8 weeks post-Allo-HSCT (without transfusion support) compared to the pre-first-dose baseline (average Hb level from the three days prior to the first dose).
Proportion of patients achieving an increase in Hb of ≥1.5 g/dL at 8 weeks post-Allo-HSCT (without transfusion support) compared to the pre-first-dose baseline (average Hb level from the three days prior to the first dose).
次要结局
- Time to Granulocyte Engraftment(One month after allo-HSCT (i.e., 3 weeks after the first dose))
- Time to Platelet Engraftment(One month after allo-HSCT (i.e., 3 weeks after the first dose))
- Incidence of drug-related adverse reactions(From baseline through 2 months post-administration of Luspatercept)
研究者
Liang Zhao
Professor
Zhujiang Hospital