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Aplastic AnemiaPhase 2NCT05399732

Efficacy and Safety in Transfusion Independent Non-severe Aplastic Anemia

A Phase 2 study testing Luspatercept and Cyclosporine in people with Aplastic Anemia, run by Bing Han at 1 site in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 to 80 years
Conditions the study is for
Aplastic Anemia
What is being tested?
Luspatercept and Cyclosporine
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
58 participants
Where
1 siteChina
Study dates
19 Dec 2022to 15 Apr 2025
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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;
Show all 13Show fewer
  • 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

Trial locations and contacts

1 site in 1 country.

China1 site

  • Peking union medical college hospital

    Beijing

Other trials in China

About this study

Official 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).

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.

Read the 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.

Conditions
Aplastic Anemia
Being tested
Luspatercept, Cyclosporine
Keywords
NSAA, luspatercept, ORR, non-transfusion dependent
Medical subject headings
Anemia, Aplastic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LuspaterceptDrug

    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.

  • CyclosporineDrug

    Also called Ciclosporin, Cyclosporin

    Cyclosporine was administered at a 3-5 mg/(kd/d) and maintained at a 100-200 ng/ml trough plasma concentration.

Study groups in NCT05399732
Study groupRoleWhat they receive
efficiency and safety in luspatercept plus cyclosporineExperimentalluspatercept 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.
Receives: Drug: Luspatercept; Drug: Cyclosporine
controll group in cyclosporine aloneActive comparatorcyclosporine is at a dose of 3~5mg/kg /day for at least 6 months.
Receives: Drug: Cyclosporine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. overall response rate (ORR)

    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)

    Measured: 6 month

Secondary outcomes (3)

  1. Hematologic response-erythroid（HR-E)

    HR is defined as a hemoglobin increase from baseline of ≥1.5 g/dL for ≥2 weeks (in the absence of RBC transfusions

    Measured: 6 month

  2. side effects

    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.

    Measured: 1 year

  3. predictive factors

    Predictors analyses will evaluate the relationship between the effect of these two treatments with molecular mutations PIGA and BCOR and BCORL1 and EPO level.

    Measured: 6 month

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Jun 2022
  2. Study start19 Dec 2022
  3. Primary completion1 Feb 2025
  4. Study completion15 Apr 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Cyclosporine soft capsules（25mg） are produced by North China pharmaceutical Group Corporation
Masking
None (open label)
Enrolment
58 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05399732
Sponsor's protocol ID
HanB-NSAA-lus
Status last verified
Sep 2025
First submitted
24 May 2022
Last updated
16 Sep 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: individual participant data would be accepted upon request

Publications

  • Howard SC, Naidu PE, Hu XJ, Jeng MR, Rodriguez-Galindo C, Rieman MD, Wang WC. Natural history of moderate aplastic anemia in children. Pediatr Blood Cancer. 2004 Oct;43(5):545-51. doi: 10.1002/pbc.20131.

    PubMed 15382271

  • Zhang ML, Chen WS, Han B. [Evaluation of the efficacy of cyclosporin A combined with recombined human thrombopoietin for treating patients with non-severe aplastic anemia]. Zhonghua Xue Ye Xue Za Zhi. 2020 Aug 14;41(8):637-642. doi: 10.3760/cma.j.issn.0253-2727.2020.08.004. Chinese.

    PubMed 32942816

  • Chen WS, Zhang ML, Han B. [Evaluation of the Efficacy of Cyclosporin A Combined with Recombined Human Erythropoietin in the Treatment of Patients with Chronic Aplastic Anemia]. Zhongguo Yi Xue Ke Xue Yuan Xue Bao. 2021 Oct;43(5):736-742. doi: 10.3881/j.issn.1000-503X.13201. Chinese.

    PubMed 34728034

  • Furlong E, Carter T. Aplastic anaemia: Current concepts in diagnosis and management. J Paediatr Child Health. 2020 Jul;56(7):1023-1028. doi: 10.1111/jpc.14996. Epub 2020 Jul 3.

    PubMed 32619069

  • Desmond R, Townsley DM, Dunbar C, Young NS. Eltrombopag in aplastic anemia. Semin Hematol. 2015 Jan;52(1):31-7. doi: 10.1053/j.seminhematol.2014.10.002. Epub 2014 Oct 31.

    PubMed 25578417

  • Matsuda K, Koya J, Arai S, Nakazaki K, Nakamura F, Kurokawa M. Cyclosporine Therapy in Patients with Transfusion-independent Non-severe Aplastic Anemia: A Retrospective Analysis. Intern Med. 2019 Feb 1;58(3):355-360. doi: 10.2169/internalmedicine.1372-18. Epub 2018 Aug 24.

    PubMed 30146592

  • Drexler B, Passweg J. Current evidence and the emerging role of eltrombopag in severe aplastic anemia. Ther Adv Hematol. 2021 Mar 3;12:2040620721998126. doi: 10.1177/2040620721998126. eCollection 2021.

    PubMed 33747425

  • Zhang Z, Hu Q, Wang L, Yang C, Chen M, Han B. Efficacy and safety of cyclosporine plus luspatercept versus cyclosporine in newly diagnosed non-transfusion-dependent non-severe aplastic anemia: A prospective randomized trial. BMC Med. 2026 Apr 29;24(1):358. doi: 10.1186/s12916-026-04887-9.

    PubMed 42057135

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Luspatercept and Cyclosporine in people with Aplastic Anemia, run by Bing Han at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 16 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

overall response rate (ORR) (time frame: 6 month). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05399732, which also lists 8 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Sep 2025.

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