Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia

Aplastic Anemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05018936
• Other study ID #: HBP-NSAA-001
• Status: Not yet recruiting
• Phase: Phase 2/Phase 3
• Start date: October 1, 2021
• Est. completion date: December 1, 2023

Study information

• Verified date: August 2021
• Source: Peking Union Medical College Hospital
• Contact: Bing Han, Docter
• Phone: +8613601059938
• Email: hanbing_li[@]sina.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 40
• Est. completion date: December 1, 2023
• Est. primary completion date: October 1, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

Inclusion Criteria:

1. Patients diagnosed to be non-severe aplastic anemia

2. Patients with platelet count < 30×109/L and have at least one of the followings:

?absolute neutrophil count < 1.5×109/L, ?platelet count < 50×109/L, ? hemoglobin level < 100g/L

3. 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);

4. Eastern Cooperative Oncology Group (ECOG) performance status 0-2;

5. Patients able to understand and comply with protocol requirements and instructions and have signed and dated informed consent.

Exclusion Criteria:

1. Congenital aplastic anemia;

2. Presence of chromosomal aberration;

3. Evidence of a clonal hematologic bone marrow disorder (MDS, AML) on cytogenetics;

4. Presence with PNH clone =50%;

5. Patients received HSCT before;

6. Uncontrolled infection or bleeding with standard treatment;

7. Allergic to Hetrombopag or accessories;

8. HIV, HCV or HBV active infection or liver cirrhosis or portal hypertension;

9. 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;

10. Have any concomitant malignancies within 5 years expect for local basal cell carcinoma of the skin;

11. Past history of thromboembolic event, heart attack or stroke (including anti-phospholipid antibody syndrome) and current use of anticoagulants;

12. Pregnant or nursing (lactating) woman;

13. Have attended other clinical trials within 3 months;

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Aplastic Anemia
• Drug Effect

Intervention

Drug:
• Hetrombopag
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.

Locations

Country	Name	City	State
China	Peking Union Medical College Hospital	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Peking Union Medical College Hospital

Country where clinical trial is conducted

China, 

References & 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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	ORR at 3 Months	ORR will be calculated after 3 months of treatment by measuring platelet, reticulocyte, neutrophil and transfusion independence.	3 month
Secondary	Volume of Platelet Transfusions	Volume of Platelet Transfusions every month	12 months
Secondary	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	12 months
Secondary	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.	12 months