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Completed

NCT Number: NCT05399732

Efficacy and Safety in Transfusion Independent Non-severe Aplastic Anemia

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.

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Key information

Conditions

Aplastic Anemia Anemia Anemia, Aplastic Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Peking union medical college hospital

Beijing, China

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • age≥18 year-old;
  • hemoglobin level between 60g/L~10 g/dL;
  • 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;
  • with normal baseline liver and kidney function;
  • with no active infection; are not pregnant or nursing;
  • agree to sign consent forms;
  • Eastern Cooperative Oncology Group (ECOG) performance status 0-2

Exclusion criteria

  • Congenital aplastic anemia;
  • Presence of chromosomal aberration;
  • Evidence of a clonal hematologic bone marrow disorder (MDS, AML) on cytogenetics;
  • Presence with PNH clone ≥50%;
  • Patients received HSCT before;
  • Uncontrolled infection or bleeding with standard treatment;
  • Allergic to luspatercept CsA or accessories;
  • HIV, HCV or HBV active infection or liver cirrhosis or portal hypertension;
  • 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;
  • Have any concomitant malignancies within 5 years expect for local basal cell carcinoma of the skin;
  • Past history of thromboembolic event, heart attack or stroke (including anti-phospholipid antibody syndrome) and current use of anticoagulants;
  • Pregnant or nursing (lactating) woman;
  • Have attended other clinical trials within 3 months

Treatment and study plan

Luspatercept

Drug

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.

cyclosporine

Drug

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

Primary outcomes

  1. 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)

Secondary outcomes

  1. 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

  2. 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.

  3. 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.

Sponsors and collaborators

Lead sponsor

Bing Han

Other

Collaborators

  • Bristol-Myers Squibb

Registry information

Official study title

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).

Important dates

Study start
2022
Primary completion
2025
Study completion
2025
First posted
Jun 1, 2022
Registry last updated
Sep 16, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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