Efficacy and Safety in Transfusion Independent Non-severe Aplastic Anemia

September 9, 2025 updated by: Bing Han

A Randomized, Case Controlled Clinical Trial Evaluating the Efficiency and Safety of Luspatercept Plus Cyclosporine Versus Cyclosporine in Newly Diagnosed Transfusion Independent Non-severe Aplastic Anemia (NSAA).

Aplastic anemia (AA) is a rare bone marrow failure disease characterized by bone marrow hypocellularity and peripheral blood pancytopenia. AA is divided into severe AA (SAA) and non-severe AA (NSAA) based on the degree of cytopenia. The first line therapy for SAA or transfusion dependent NSAA is either immunosuppression therapy (IST) or hematopoietic stem cell transplantation (HSCT). Little attention has been paid to patients with anemia but not transfusion dependent, whose quality of life is significantly impaired due to the anemia and other complications.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Recombined human erythropoietin (rhEPO) has been shown to increase the erythroid response and response rate when combined with IST for patients with newly diagnosed AA, either SAA or NSAA. Different from rhEPO, luspatercept is a recombinant fusion protein that binds to select transforming growth factor β superfamily ligands and enhances late-stage erythropoiesis, and has been shown the promising efficiency in the erythropoiesis in patients with lower risk myelodysplastic syndrome (MDS) in the phase II and III clinical trials. This randomized control study aimed to compare the 6-month efficacy and safety of the combination of luspatercept and cyclosporine versus cyclosporine monotherapy in patients with newly diagnosed transfusion independent NSAA.

Study Type

Interventional

Enrollment (Actual)

58

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
      • Beijing, China
        • Peking Union Medical College Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. age≥18 year-old;
  2. hemoglobin level between 60g/L~10 g/dL;
  3. newly diagnosed patients have at least one of the followings: #absolute neutrophil count <1.5×109/L, #platelet count < 30×109/L, # hemoglobin level < 100g/L;
  4. with normal baseline liver and kidney function;
  5. with no active infection; are not pregnant or nursing;
  6. agree to sign consent forms;
  7. Eastern Cooperative Oncology Group (ECOG) performance status 0-2

Exclusion Criteria:

  1. Congenital aplastic anemia;
  2. Presence of chromosomal aberration;
  3. Evidence of a clonal hematologic bone marrow disorder (MDS, AML) on cytogenetics;
  4. Presence with PNH clone ≥50%;
  5. Patients received HSCT before;
  6. Uncontrolled infection or bleeding with standard treatment;
  7. Allergic to luspatercept CsA or accessories;
  8. HIV, HCV or HBV active infection or liver cirrhosis or portal hypertension;
  9. Patient with QTcF (Fridericia's QT correction formula) at screening <450 msec, or<480 msec with bundle branch block, as determined via the mean of a triplicate ECG and assessed at site, unstable angina pectoris, uncontrolled hypertension(>180/100mmHg)#pulmonary artery hypertension;
  10. Have any concomitant malignancies within 5 years expect for local basal cell carcinoma of the skin;
  11. Past history of thromboembolic event, heart attack or stroke (including anti-phospholipid antibody syndrome) and current use of anticoagulants;
  12. Pregnant or nursing (lactating) woman;
  13. Have attended other clinical trials within 3 months

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: efficiency and safety in luspatercept plus cyclosporine
luspatercept is at a dose of 1.0 mg per kilogram of body weight, administered subcutaneously every 3 weeks，and cyclosporine is at a dose of 3~5mg/kg /day for at least 6 months.
Drug: Luspatercept
Patients in each group will be treated for at least 6 months and continue the treatment for an additional 6 months unless disease progress or have intolerable side effects.
Other Names:
  • ciclosporin
Drug: Cyclosporine
Cyclosporine was administered at a 3-5 mg/(kd/d) and maintained at a 100-200 ng/ml trough plasma concentration.
Other Names:
  • Ciclosporin
  • Cyclosporin
Active Comparator: controll group in cyclosporine alone
cyclosporine is at a dose of 3~5mg/kg /day for at least 6 months.
Drug: Cyclosporine
Cyclosporine was administered at a 3-5 mg/(kd/d) and maintained at a 100-200 ng/ml trough plasma concentration.
Other Names:
  • Ciclosporin
  • Cyclosporin

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
overall response rate (ORR)
Time Frame: 6 month
Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR); HR defined as a hemoglobin increase from baseline of ≥1.5 g/dL for ≥2 weeks (in the absence of RBC transfusions)
6 month

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Hematologic response-erythroid（HR-E)
Time Frame: 6 month
HR is defined as a hemoglobin increase from baseline of ≥1.5 g/dL for ≥2 weeks (in the absence of RBC transfusions
6 month
side effects
Time Frame: 1 year
Safety analyses include assessments of the incidence and severity of adverse events; all adverse events that occurred or worsened during the treatment period will be reported, as well as adverse events that occurred later but are considered by the investigator to be related to the trial drug.
1 year
predictive factors
Time Frame: 6 month
Predictors analyses will evaluate the relationship between the effect of these two treatments with molecular mutations PIGA and BCOR and BCORL1 and EPO level.
6 month

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Bing Han

Collaborators

Bristol-Myers Squibb

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 19, 2022

Primary Completion (Actual)

February 1, 2025

Study Completion (Actual)

April 15, 2025

Study Registration Dates

First Submitted

May 24, 2022

First Submitted That Met QC Criteria

May 30, 2022

First Posted (Actual)

June 1, 2022

Study Record Updates

Last Update Posted (Estimated)

September 16, 2025

Last Update Submitted That Met QC Criteria

September 9, 2025

Last Verified

September 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HanB-NSAA-lus

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

individual participant data would be accepted upon request

IPD Sharing Time Frame

10years

IPD Sharing Access Criteria

email request

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • CSR

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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