Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia

NCT05018936 | Withdrawn Phase 2 DMC

• 1 other identifier: HBP-NSAA-001
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

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

Outcome Measures

Primary Outcomes (1)

• 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

  6 month

Secondary Outcomes (4)

• ORR at 3 Months

  3 month

• Volume of Platelet Transfusions

  12 months

• percentage of side effects at 12 months

  12 months

• Percentage of patients with clonal evolution at 12 months

  12 months

Study Arms (1)

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.

Drug: Hetrombopag

Interventions

Hetrombopag DRUG

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.

treatment group

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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 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;

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, 100730, China

Location

Related Publications (1)

• 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.

  PMID: 33632264 BACKGROUND

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

hetrombopag

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Officials

• Bing Han, Master

  Peking Union Medical College Hospital

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Docter

Study Record Dates

• First Submitted: August 19, 2021
• First Posted: August 24, 2021
• Study Start: October 1, 2021
• Primary Completion: October 1, 2023
• Study Completion: December 1, 2023
• Last Updated: June 8, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: 10 years
• Access Criteria: email request

Locations

CN (1)