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Non Severe Aplastic AnemiaPhase 2NCT05660785

Herombopag Added to Cyclosporine in Non Severe Aplastic Anemia

A Phase 2 study testing Herombopag in people with Non Severe Aplastic Anemia and Untreated, run by Institute of Hematology & Blood Diseases Hospital, China at 4 sites 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

This is a prospective, multicenter, single-arm, phase 2 trial. The aim of this study is to evaluate the efficacy and safety of herombopag combined with cyclosporine for patients with non severe aplastic anemia (NSAA). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Non Severe Aplastic Anemia, Untreated
What is being tested?
Herombopag
Could I receive a placebo?
No, everyone receives an active treatment
How many people
54 participants
Where
4 sitesChina
Study dates
1 Dec 2022to 28 Jan 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 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Willing and able to comply with the requirements for this study and written informed consent.
  • Male or female age ≥ 18 years
  • Diagnosis of untreated non severe aplastic anemia.
  • Platelet counts < 50 x 10^9/L at least 2 times consecutively (time interval ≥ 1 week)

Who cannot join

  • Receive immunosuppressive therapy more than 4 weeks before enrollment
  • Treatment with TPO-RA within 1 week before enrollment
  • Inherited bone marrow failure syndromes
  • Bone marrow fibrosis grade ≥ 2
  • The presence of hemolytic PNH clone
  • The presence of clonal karyotypic abnormalities (del(20q), +8 and -Y are not included in this category)
Show all 21Show fewer
  • Previously treated with TPO-RA ≥ 4 weeks
  • Previously received immunosuppressive therapy ≥ 12 weeks
  • Ferritin > 1000 ng/ml (The increased level of Ferritin led by infection is not included in this category)
  • Have an allergy to eltrombopag or any other part of this medicine.
  • History of radiotherapy and chemotherapy for malignant solid tumors
  • Cytopenia caused by other non-hematologic diseases, including liver cirrhosis, active rheumatic connective tissue disease, and persistence of infectious diseases, etc
  • Abnormal liver function: ALT or AST > 3 ULN, or TBil > 1.5 ULN after treatment.
  • Abnormal kidney function: Creatinine clearance < 30 ml/min, or serum creatinine (sCr) >1.5 ULN
  • Patients with diabetic nephropathy, neuropathy, or eye disease
  • Patients with poorly controlled hypertension or cardiac arrhythmia
  • Patients with congestive heart failure and the NYHA grade ≥ 3 historically or currently, and LVEF < 45% within 4 weeks before enrollment
  • History of arteriovenous thrombosis within 1 year before enrollment
  • Participation in another clinical trial within 4 months before the start of this trial
  • Pregnant or breast-feeding patients
  • Patients considered to be ineligible for the study by the investigator for reasons other than the above

Trial locations and contacts

4 sites in 1 country.

China4 sites

  • The Second Affilated Hospital of Shandong First Medical University

    Tai’an, Shandong

  • Tangshan Central Hospital

    Tangshan, Hebei

  • Regenerative Medicine Center

    Tianjin, Tianjin Municipality

  • Zhoukou Central Hospital

    Zhoukou, Henan

Other trials in China

About this study

Official title: The Efficacy and Safety of Herombopag Combined With Cyclosporine for Patients With Non Severe Aplastic Anemia

This is a prospective, multicenter, single-arm, phase 2 trial. The aim of this study is to evaluate the efficacy and safety of herombopag combined with cyclosporine for patients with non severe aplastic anemia (NSAA).

Read the detailed description

This study aims to improve the 24 weeks response rate. The sample size is calculated based on Simon's two-stage design. The first stage of the study enrolled a cohort of 15 patients. If after 24 weeks at least 9 patients achieved a response, then enrollment was expanded to a total of 43 patients. The null hypothesis was unaccepted if more than 26 of 43 patients achieved the response. Accounting for a 20% dropout rate, the estimated final sample size was 54 patients.

Being tested
Herombopag

What participants receive

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

  • HerombopagDrug

    Also called Cyclosporine A

    Hetrombopag is a TPO receptor agonist approved in China in 2021 for idiopathic thrombocytopenic purpura (ITP) and second-line severe aplastic anemia (SAA). Indications of chemotherapy-induced thrombocytopenia (CIT), pediatric/juvenile ITP and naive severe aplastic anemia are under development. Hetrombopag was granted Orphan Drug Designation by FDA for the treatment of CIT.

    Cyclosporine A is a calcineurin inhibitor, which has an effect on reducing T-cell proliferation and activation, can reverse pancytopenia and alleviate transfusion requirements in NSAA.

Study groups in NCT05660785
Study groupRoleWhat they receive
CsA + HerombopagExperimentalHerombopag combined with cyclosporine
Receives: Drug: Herombopag

What is being measured

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

  1. Overall response rate

    Percentage of patients with hematological response. Hematological response is evaluated by hemoglobin, platelet and neutrophil count in the routine blood test.

    Measured: 24 weeks

Secondary outcomes (6)

  1. Robust response rate

    Percentage of patients with robust response, including complete response, near complete response, very good partial response(VGPR) and Meaningful partial response(mPR). These are evaluated by hemoglobin, platelet and neutrophil count in the routine blood test.

    Measured: 24 weeks

  2. Proportion of patients with abnormal karyotype changes

    The abnormal karyotype was examined by karyotype test

    Measured: Baseline and 24 weeks

  3. Time duration for patients achieving hematological response

    Duration time was calculated from response to relapse.

    Measured: A minimum of 2 years of planned follow-up

  4. Change of the health-related quality of life

    Medical Outcomes Study Questionnaire Short Form 36 Health Survey (SF-36) is used to assess the health-related quality of life of patients. The SF-36 has eight scaled scores; the scores are weighted sums of the questions in each section. Scores range from 0 - 100. Lower scores = more disability, higher scores = less disability

    Measured: Baseline and 24 weeks

  5. Incidence of the adverse event

    Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the adverse event.

    Measured: 24 weeks

  6. Severity of the adverse event

    Use Common Terminology Criteria for Adverse Events (CTCAE) Version 5 to assess the severity.

    Measured: 24 weeks

Timeline

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

  1. First posted21 Dec 2022
  2. Study start1 Dec 2022
  3. Primary completion20 Jan 2025
  4. Study completion28 Jan 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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
54 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05660785
Sponsor's protocol ID
IIT2022049
Status last verified
Jan 2025
First submitted
23 Nov 2022
Last updated
5 Aug 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • Zhang L, Li R, Liang Q, Li W, Pan H, Gao Z, Fang L, Zhao J, Yu X, Kuang Z, Nie N, Li J, Huang J, Zhao X, Ge M, Zheng Y, Li J, Zhang H, Shi J. Hetrombopag Added to Cyclosporine as the First-Line Treatment for Patients With Non-Severe Aplastic Anemia: A Phase 2 Multicenter Trial. Am J Hematol. 2026 Mar;101(3):467-476. doi: 10.1002/ajh.70183. Epub 2026 Jan 5.

    PubMed 41489450

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Herombopag in people with Non Severe Aplastic Anemia and Untreated, run by Institute of Hematology & Blood Diseases Hospital, China at 4 sites 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 4 inclusion and 21 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 5 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 4 sites 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 (time frame: 24 weeks). 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 NCT05660785, which also lists 1 linked publication.

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 5 Aug 2025.

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