Luspatercept Plus CsA vs CsA for the Treatment of Newly Diagnosed Non-Transfusion-Dependent NSAA

June 2, 2026 updated by: Bing Han, Peking Union Medical College Hospital

A Randomized, Controlled Trial Comparing the Safety and Efficacy of Luspatercept Plus Cyclosporine Versus Cyclosporine Alone for the Treatment of Newly Diagnosed Non-transfusion-dependent Non-severe Aplastic Anemia (NSAA)

In a randomized, controlled clinical trial, the efficacy and safety of rodsipil combined with cyclosporine versus cyclosporine alone in the treatment of newly diagnosed non-transfusion-dependent NSAA were compared.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Detailed Description

Conduct a comparative evaluation of the effectiveness and safety of Luspatercept combined with cyclosporine versus cyclosporine monotherapy in the treatment of newly diagnosed non-transfusion-dependent non-severe aplastic anemia (NSAA). Patients were randomized in a 1:1 ratio and assigned to one of two groups: Group A, Luspatercept combined with cyclosporine: received Luspatercept (1.0 mg/kg, subcutaneous injection every 3 weeks), cyclosporine (3-5mg/kg/day), adjusted based on hematological parameters, for at least 6 months to assess efficacy. Effective patients continued to receive cyclosporine treatment for at least 1.5 years, with a gradual reduction in dosage; Group B, cyclosporine: received 3-5mg/kg/day, adjusted based on hematological parameters, for at least 6 months to assess efficacy, with effective patients continuing to receive cyclosporine treatment for at least 1.5 years, with a gradual reduction in dosage. Hgb below 60g/L was allowed, or in emergency conditions, blood transfusion was allowed. Platelets below 20×10^9/L or with obvious bleeding tendency were allowed to receive platelet transfusion. If neutrophil count was below 1.0×10^9/L, G-CSF was allowed until neutrophil count recovered to above 1.0×10^9/L. Symptoms, treatment-related adverse events, signs, blood transfusion volume, and laboratory tests (including reticulocyte count) were recorded at least every 3 months for the first 3 months, and every 6 months thereafter until 6 months, and bone marrow aspiration, biopsy, and chromosome examination were performed at least every 6 months to observe efficacy and safety.

Study Type

Interventional

Phase

  • Phase 4

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 Municipality
      • Beijing, Beijing Municipality, China, 100730
        • 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

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Age ≥ 18 years
  2. Hemoglobin level of 6-10 g/dL
  3. Definition of NSAA: Patients with AA diagnosis but not SAA or VSAA diagnosis (at least two of the following conditions can be diagnosed as AA: (i) Hemoglobin < 100 g/L; (ii) Platelet count < 50×10^9/L; (iii) Neutrophil count < 1.5×10^9/L. SAA diagnosis criteria include less than 25% (or 25-50%, but residual hematopoietic cells < 30%) of bone marrow cells, plus at least two of the following conditions: (i) Neutrophil count < 0.5×10^9/L; (ii) Platelet count < 20×10^9/L; (iii) Retroperitoneal lymph node count < 20×10^9/L. VSAA meets the criteria for SAA, but with neutrophil count < 0.2×10^9/L. (British guidelines, 2015))
  4. No active infection
  5. No other concurrent neoplasms (except in situ carcinoma)
  6. Baseline liver and renal function within 1.5 times of normal value
  7. No pregnancy or breastfeeding
  8. Agree to sign informed consent form
  9. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.

Exclusion Criteria:

  1. Congenital aplastic anemia
  2. Presence of chromosomal aberrations
  3. Cytogenetic evidence of clonal hematological myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML)
  4. PNH clone ≥50%
  5. Previous use of alemtuzumab, any ATG, or any dose of cyclosporine for immunosuppressive treatment
  6. Previous hematopoietic stem cell transplant (HSCT)
  7. Uncontrolled infection or bleeding under standard treatment
  8. Allergy to rituximab, cyclosporine, or excipients
  9. History of allergy to polyethylene glycol (PEG) 80
  10. Active infection or cirrhosis of the liver or portal hypertension due to HIV, HCV, or HBV
  11. Screening QTcF (Fridericia QT corrected formula) less than 450 milliseconds or less than 480 milliseconds of bundle branch block determined by three ECG averages, and assessed on-site; unstable angina; uncontrolled hypertension (>180/100 mmHg); pulmonary hypertension
  12. Any malignant tumor within 5 years, except local basal cell carcinoma; previous thromboembolic event, history of myocardial infarction or stroke (including antiphospholipid syndrome); currently using anticoagulants
  13. Pregnant or lactating women
  14. Participated in another clinical trial 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: Luspatercept combined with cyclosporine
Administered Luspatercept (1.0 mg/kg, subcutaneous injection every 3 weeks), and Cyclosporine (3-5mg/kg/day) adjusted according to hematological parameters, for at least 6 months to evaluate efficacy. Effective patients will continue to receive Cyclosporine treatment for at least 1.5 years, followed by a gradual reduction in dosage.
Drug: Luspatercept
Luspatercept (dose of 1.0 mg/kg, subcutaneous injection every 3 weeks) Cyclosporine (3-5mg/kg/day)
Other Names:
  • cyclosporine
Drug: cyclosporine
Cyclosporine (3-5mg/kg/day)
Experimental: cyclosporine
Give cyclosporine 3-5mg/kg/day, adjust the dose based on the blood count, and administer it for at least 6 months to evaluate the efficacy. If effective, the patient will continue to receive cyclosporine treatment for at least 1.5 years, followed by a gradual reduction in dosage.
Drug: cyclosporine
Cyclosporine (3-5mg/kg/day)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
overall response rate (ORR)
Time Frame: 6 month
Proportion of patients who achieved complete response, partial response and hematological response
6 month

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
overall response rate (ORR)
Time Frame: 12 month
Proportion of patients who achieved complete response, partial response and hematological response
12 month
adverse event rate
Time Frame: 12 month
Proportion of patients with adverse events
12 month

Collaborators and Investigators

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

Sponsor

Peking Union Medical College Hospital

Investigators

  • Principal Investigator: Bing Bing, PhD, Peking Union Medical College Hospital

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 (Estimated)

May 1, 2024

Primary Completion (Estimated)

May 1, 2025

Study Completion (Estimated)

December 1, 2025

Study Registration Dates

First Submitted

May 16, 2024

First Submitted That Met QC Criteria

May 16, 2024

First Posted (Actual)

May 22, 2024

Study Record Updates

Last Update Posted (Actual)

June 4, 2026

Last Update Submitted That Met QC Criteria

June 2, 2026

Last Verified

June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • LC-001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

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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