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

An Exploratory Study on the Safety and Efficacy of Luspatercept in the Treatment of Anemia Following Allogeneic Hematopoietic Stem Cell Transplantation for Acute Leukemia

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.

Study Overview

• Status: Recruiting
• Conditions: Acute Leukemia
• Intervention / Treatment: Drug: Luspatercept

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 Type: Interventional
• Enrollment (Estimated): 46
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Zhao Liang, Doctor; Phone Number: 02061643888 86 13631451531; Email: 383331047@qq.com

Study Locations

• China
  • Guangdong
    • Guangzhou, Guangdong, China
      • Recruiting
      • Zhujiang Hospital
      • Contact: Zhao Liang, Doctor

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Clinically diagnosed with acute leukemia.
2. Has undergone allogeneic hematopoietic stem cell transplantation (allo-HSCT).
3. Expected to survive for more than 3 months from the date of signing the informed consent form.
4. Willing and able to comply with scheduled visits, treatment plans, laboratory tests, and other study procedures, and has provided written informed consent.
5. 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.
6. Hemoglobin (Hb) level < 80 g/L.

Exclusion Criteria:

1. Inadequate organ function, defined as:

1. Creatinine clearance < 60 mL/min;
2. Left ventricular ejection fraction (LVEF) < 55%;
3. Oxygen saturation (SpO₂) < 92% on room air;
4. Total bilirubin > 2 × the upper limit of normal (ULN);
5. 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:

1. Basal or squamous cell carcinoma of the skin;
2. Carcinoma in situ of the cervix;
3. Carcinoma in situ of the breast;
4. Incidental histological finding of prostate cancer (T1a or T1b as defined by the TNM staging system);
5. 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Single-Arm Group; All participants receive subcutaneous luspatercept at 1 mg/kg on day +7 and day +28 post-transplantation.

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 day +28 ±3 days post-transplantation. If delayed, administer as soon as possible and continue with the regular dosing schedule (maintaining at least a 3-week interval between doses). If the ±3-day window is exceeded, the subject will be withdrawn from the study treatment.; Other Names: Reblozyl®

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of patients with reduced transfusion requirement	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).	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

Outcome Measure	Measure Description	Time Frame
Time to Granulocyte Engraftment	Time interval from the day of stem cell infusion to the first day of three consecutive days with an absolute neutrophil count (ANC) greater than 0.5 G/L.	One month after allo-HSCT (i.e., 3 weeks after the first dose)
Time to Platelet Engraftment	Time interval from the day of stem cell infusion to the first day of three consecutive days with an absolute platelet count ≥40 × 10⁹/L.	One month after allo-HSCT (i.e., 3 weeks after the first dose)
Incidence of drug-related adverse reactions	Record the occurrence of all adverse events, including the event type, severity, time of onset, relationship to the drug, and management actions taken. Analyze the incidence of adverse reactions (possibly) related to the drug, and the incidence of Grade ≥3 adverse reactions.	From baseline through 2 months post-administration of Luspatercept

Collaborators and Investigators

• Sponsor: Zhujiang Hospital
• Investigators: Principal Investigator: Zhao Liang, Doctor, Zhujiang Hosptial of Southern Medical University

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2025
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): May 31, 2028

Study Registration Dates

• First Submitted: January 15, 2026
• First Submitted That Met QC Criteria: January 21, 2026
• First Posted (Actual): January 23, 2026

Study Record Updates

• Last Update Posted (Actual): January 23, 2026
• Last Update Submitted That Met QC Criteria: January 21, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Keywords: Anemia; Luspatercept; Allo-HSCT; Acute leukemia
• Additional Relevant MeSH Terms: Hematologic Diseases; Hemic and Lymphatic Diseases; Anemia; luspatercept
• Other Study ID Numbers: 2025-KY-242

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Upon completion of the trial, data will be made public through the publication of academic papers. The original data can be requested via email.
• IPD Sharing Time Frame: Upon trial completion
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes