Luspatercept for the Treatment of Anemia Following Allogeneic Hematopoietic Stem Cell Transplantation(Allo-HSCT)

Official title

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

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.

Interventions

• Drug Luspatercept
  Study Treatment and Dosing Schedule According to the protocol, subcutaneous injections will be administered at a dose of 1 mg/kg on day +7 and day +28 post-transplantation. Inject into the upper arm, thigh, or abdomen. Doses requiring a larger volume (\>1.2 mL) should be divided into similar volumes in separate syringes and injected at different sites. Use a new syringe and needle for each separate injection. Missed Dose: A dosing window of ±3 days is allowed, i.e., within day +7 ±3 days and da

Primary outcome measures

• 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).]

Secondary outcome measures (3)

• Time to Granulocyte Engraftment [Time frame: One month after allo-HSCT (i.e., 3 weeks after the first dose)]
• Time to Platelet Engraftment [Time frame: One month after allo-HSCT (i.e., 3 weeks after the first dose)]
• Incidence of drug-related adverse reactions [Time frame: From baseline through 2 months post-administration of Luspatercept]

Eligibility criteria

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. 2. 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. 9. 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

China · 1 center

• Zhujiang Hospital — Guangzhou

Identifiers

NCT: NCT07362095 · 2025-KY-242

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗