Aplastic AnemiaPhase 2/3NCT05018936

Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia

A Phase 2/3 study testing Hetrombopag in people with Aplastic Anemia and Drug Effect, run by Peking Union Medical College Hospital at 1 site in China.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record

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Trial summary

Who is it for?: Ages 18 to 75 years
Conditions the study is for: Aplastic Anemia, Drug Effect
What is being tested?: Hetrombopag
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 0 participants
Where: 1 siteChina
Study dates: 1 Oct 2021to 1 Dec 2023
Phase: Phase 2/3
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 75 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Patients diagnosed to be non-severe aplastic anemia
Patients with platelet count < 30×109/L and have at least one of the followings: ①absolute neutrophil count < 1.5×109/L, ②platelet count < 50×109/L, ③ hemoglobin level < 100g/L
Patients have no response or relapsed following at least one treatment course in a period time of > 6 months of immunosuppression containing CsA or CsA+anti-thymocyte globulin (ATG);
Eastern Cooperative Oncology Group (ECOG) performance status 0-2;
Patients able to understand and comply with protocol requirements and instructions and have signed and dated informed consent.

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 Hetrombopag 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, 100730

Other trials in China

About this study

Official title: Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia: a Prospective Single Arm Study

This is a prospective one arm study to explore the efficacy and safety of Hetrombopag in non-severe aplastic anemia. Patients meeting the inclusion and exclusion criteria would be recruited. Treatment of Hetrombopag would be started with 5mg/day. The dosage would be increased by 2.5mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L. The maximum dosage is 15mg/day. All patients would receive treatment for at least 6 months except that the platelet <20×10e9/L at the dosage of 15mg/day for 4 weeks or the platelet ≥200×10e9/L at the dosage of 5mg/week for 2 weeks. The hematological response rate and safety will be recorded and compared at D15, 1month, 1.5month, 2month, 3month, 4month, 5month, 6month, 8month, 10month and 1year.

Conditions: Aplastic Anemia, Drug Effect
Being tested: Hetrombopag
Medical subject headings: Anemia, Aplastic

What participants receive

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

HetrombopagDrug
Hetrombopag starting at 5mg/day and increased by 2.5mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L.

Study groups in NCT05018936
Study group	Role	What they receive
treatment group	Experimental	Hetrombopag would be started with 5mg/day. The dosage would be increased by 2.5mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L. The maximum dosage is 15mg/day. Receives: Drug: Hetrombopag

What is being measured

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

ORR at 6 Months
Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR) at 6 months
Measured: 6 month

Secondary outcomes (4)

ORR at 3 Months
ORR will be calculated after 3 months of treatment by measuring platelet, reticulocyte, neutrophil and transfusion independence.
Measured: 3 month
Volume of Platelet Transfusions
Volume of Platelet Transfusions every month
Measured: 12 months
percentage of side effects at 12 months
percentage of side effects would be recorded during the study and be calculated according to CTCAE 5.0 at 12 months
Measured: 12 months
Percentage of patients with clonal evolution at 12 months
Percentage of patients with clonal evolution would be evaluated by bone marrow biopsy at 12 months follow up.
Measured: 12 months

Timeline

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

1 Oct 20211 Dec 2023

First posted24 Aug 2021
Study start1 Oct 2021 (estimated)
Primary completion1 Oct 2023 (estimated)
Study completion1 Dec 2023 (estimated)

Why it stopped: The ethical application for the institution was not completed, and the research was not carried out.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

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 2/3
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 0 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05018936
Sponsor's protocol ID: HBP-NSAA-001
Status last verified: Jun 2026
First submitted: 19 Aug 2021
Last updated: 8 Jun 2026
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: Yes: individual participant data would be accepted upon request

Publications

Mei H, Liu X, Li Y, Zhou H, Feng Y, Gao G, Cheng P, Huang R, Yang L, Hu J, Hou M, Yao Y, Liu L, Wang Y, Wu D, Zhang L, Zheng C, Shen X, Hu Q, Liu J, Jin J, Luo J, Zeng Y, Gao S, Zhang X, Zhou X, Shi Q, Xia R, Xie X, Jiang Z, Gao L, Bai Y, Li Y, Xiong J, Li R, Zou J, Niu T, Yang R, Hu Y. A multicenter, randomized phase III trial of hetrombopag: a novel thrombopoietin receptor agonist for the treatment of immune thrombocytopenia. J Hematol Oncol. 2021 Feb 25;14(1):37. doi: 10.1186/s13045-021-01047-9.
PubMed 33632264

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
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Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
RecruitingObservational study
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
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Not yet recruitingObservational study
Clinical Genetics Branch Eligibility Screening Survey
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RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)

All Aplastic Anemia trials

Study at a glance

From the registry record, last updated by the sponsor on 8 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia: a Prospective Single Arm Study
ClinicalTrials.gov ID: NCT05018936
Acronym / sponsor ID: HBP-NSAA-001
Conditions: Aplastic Anemia, Drug Effect
Phase: Phase 2/3
Study type: Interventional
Recruitment status: WithdrawnReason stopped: The ethical application for the institution was not completed, and the research was not carried out.
Enrolment: 0 participants (actual)
Lead sponsor: Peking Union Medical College HospitalUniversity, hospital or other
Study start: 1 Oct 2021 (estimated)
Primary completion: 1 Oct 2023 (estimated)
Study completion: 1 Dec 2023 (estimated)
First posted: 24 Aug 2021 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
treatment group	Experimental	Drug: HetrombopagHetrombopag would be started with 5mg/day. The dosage would be increased by 2.5mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L. The maximum dosage is 15mg/day.

Who may qualify

Ages: 18 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (5 listed)

Patients diagnosed to be non-severe aplastic anemia
Patients with platelet count < 30×109/L and have at least one of the followings: ①absolute neutrophil count < 1.5×109/L, ②platelet count < 50×109/L, ③ hemoglobin level < 100g/L
Patients have no response or relapsed following at least one treatment course in a period time of > 6 months of immunosuppression containing CsA or CsA+anti-thymocyte globulin (ATG);
Eastern Cooperative Oncology Group (ECOG) performance status 0-2;
Patients able to understand and comply with protocol requirements and instructions and have signed and dated informed consent.

Key exclusion criteria (13 listed)

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;

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
HetrombopagHetrombopag starting at 5mg/day and increased by 2.5mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L.	Drug	treatment group

Primary outcome

ORR at 6 Months
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) at 6 months

4 secondary outcomes

ORR at 3 Months
Time frame: 3 month
Volume of Platelet Transfusions
Time frame: 12 months
percentage of side effects at 12 months
Time frame: 12 months
Percentage of patients with clonal evolution at 12 months
Time frame: 12 months

Results and safety

The study was withdrawn before enrolling participants, so there are no results.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 8 Jun 2026.
Background: PubMed 33632264Mei H, Liu X, Li Y, Zhou H, Feng Y, Gao G, Cheng P, Huang R, Yang L, Hu J, Hou M, Yao Y, Liu L, Wang Y, Wu D, Zhang L, Zheng C, Shen X, Hu Q, Liu J, Jin J, Luo J, Zeng Y, Gao S, Zhang X, Zhou X, Shi Q, Xia R, Xie X, Jiang Z, Gao L, Bai Y, Li Y, Xiong J, Li R, Zou J, Niu T, Yang R, Hu Y. A multicenter, randomized phase III trial of hetrombopag: a novel thrombopoietin receptor agonist for the treatment of immune thrombocytopenia. J Hematol Oncol. 2021 Feb 25;14(1):37. doi: 10.1186/s13045-021-01047-9.
Individual participant dataSharing planned: Yes. individual participant data would be accepted upon request

Related trials for Aplastic Anemia

RecruitingPhase 1/2
The Safety and Efficacy of Daratumumab in Patients With Refractory Aplastic Anemia
Aplastic Anemia, Refractory
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
RecruitingObservational study
Defining the Natural History of Squamous Cell Carcinoma in Fanconi Anemia
Fanconi Anemia, Inherited Bone Marrow Failure Syndrome
Not yet recruitingObservational study
Clinical Genetics Branch Eligibility Screening Survey
Melanoma, Li-Fraumeni Syndrome
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Bing Han, Master study director

Peking Union Medical College Hospital

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Hetrombopag in people with Aplastic Anemia and Drug Effect, run by Peking Union Medical College Hospital 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 5 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: withdrawn, as of the sponsor's last update on 8 Jun 2026. 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 combined 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.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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 was withdrawn before enrolling participants, so there are no results.

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?

ORR at 6 Months (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 NCT05018936, 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 8 Jun 2026.