An Exploratory Study on the Safety and Efficacy of Luspatercept in the Treatment of Anemia Following Allogeneic Hematopoietic Stem Cell Transplantation for Acute Leukemia
Overview
- Phase
- Phase 1
- Status
- Recruiting
- Sponsor
- Zhujiang Hospital
- Enrollment
- 46
- Locations
- 1
- Primary Endpoint
- Proportion of patients with reduced transfusion requirement
Overview
Brief Summary
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.
Detailed Description
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 70 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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.
Exclusion Criteria
- •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;
Arms & Interventions
Single-Arm Group
All participants receive subcutaneous luspatercept at 1 mg/kg on day +7 and day +28 post-transplantation.
Intervention: Luspatercept (Drug)
Outcomes
Primary Outcomes
Proportion of patients with reduced transfusion requirement
Time Frame: 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).
Secondary Outcomes
- 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)
Investigators
Liang Zhao
Professor
Zhujiang Hospital