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

NCT07362095 | Recruiting Phase 1 FDA Drug

• 1 other identifier: 2025-KY-242
• Study Type: interventional
• Target: 46
• Locations: 1 country
• Sites: 1

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.

Conditions

Acute Leukemia

Keywords

Acute leukemia; Allo-HSCT; Anemia; Luspatercept

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• 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

Study Arms (1)

Single-Arm Group

EXPERIMENTAL

All participants receive subcutaneous luspatercept at 1 mg/kg on day +7 and day +28 post-transplantation.

Drug: Luspatercept

Interventions

Luspatercept DRUG

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.

Also known as: Reblozyl®

Single-Arm Group

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• \. 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.
• \. 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.
• \. Major surgery within 8 weeks prior to enrollment. The subject must have fully recovered from any prior surgery.
• \. Uncontrolled epilepsy, history of cerebral ischemia/hemorrhage, cerebellar disease, or other active central nervous system disorders.
• \. 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).

Sponsors & Collaborators

• Zhujiang Hospital: lead

Study Sites (1)

Zhujiang Hospital

Guangzhou, Guangdong, China

RECRUITING

MeSH Terms

Conditions

Anemia

Interventions

luspatercept

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Zhao Liang, Doctor

  Zhujiang Hosptial of Southern Medical University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Zhao Liang, Doctor

CONTACT

86 13631451531; 383331047@qq.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: Luspatercept

Study Record Dates

• First Submitted: January 15, 2026
• First Posted: January 23, 2026
• Study Start: October 1, 2025
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): May 31, 2028
• Last Updated: January 23, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: Upon trial completion

Locations

CN (1)