Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia

Phase 2

• Conditions: Drug Effect; Aplastic Anemia
• Interventions: Drug: Hetrombopag

• Registration Number: NCT05018936
• Lead Sponsor: Peking Union Medical College Hospital

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2021-08
• Study First Submitted: 2021-08-19
• Study First Submitted QC: 2021-08-19
• Last Update Submitted: 2021-08-19
• Study First Posted: 2021-08-24
• Last Update Posted: 2021-08-24
• Study Start: 2021-10-01
• Primary Completion: 2023-10-01
• Study Completion: 2023-12-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 40

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: ①absolute neutrophil count < 1.5×109/L, ②platelet count < 50×109/L, ③ 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;

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
treatment group	Hetrombopag	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.

Primary Outcome Measures

Name	Time	Method
ORR at 6 Months	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

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Peking Union Medical College Hospital; 🇨🇳 Beijing, China