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Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia

Primary Purpose

Aplastic Anemia, Drug Effect

Status

Not yet recruiting

Phase

Phase 2

Locations

China

Study Type

Interventional

Intervention

Hetrombopag

About this trial

This is an interventional treatment trial for Aplastic Anemia

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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.

Exclusion Criteria:

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;

Sites / Locations

Peking Union Medical College Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

treatment group

Arm Description

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.

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

ORR at 3 Months

ORR will be calculated after 3 months of treatment by measuring platelet, reticulocyte, neutrophil and transfusion independence.

Volume of Platelet Transfusions

Volume of Platelet Transfusions every month

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

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.

Full Information

NCT ID

NCT05018936

First Posted

August 19, 2021

Last Updated

August 19, 2021

Sponsor

Peking Union Medical College Hospital

1. Study Identification

Unique Protocol Identification Number

NCT05018936

Brief Title

Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

August 2021

Overall Recruitment Status

Not yet recruiting

Study Start Date

October 1, 2021 (Anticipated)

Primary Completion Date

October 1, 2023 (Anticipated)

Study Completion Date

December 1, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Peking Union Medical College Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Aplastic Anemia, Drug Effect

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2, Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

treatment group

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Hetrombopag

Intervention Description

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.

Primary Outcome Measure Information:

Title

ORR at 6 Months

Description

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

Time Frame

6 month

Secondary Outcome Measure Information:

Title

ORR at 3 Months

Description

ORR will be calculated after 3 months of treatment by measuring platelet, reticulocyte, neutrophil and transfusion independence.

Time Frame

3 month

Title

Volume of Platelet Transfusions

Description

Volume of Platelet Transfusions every month

Time Frame

12 months

Title

percentage of side effects at 12 months

Description

percentage of side effects would be recorded during the study and be calculated according to CTCAE 5.0 at 12 months

Time Frame

12 months

Title

Percentage of patients with clonal evolution at 12 months

Description

Percentage of patients with clonal evolution would be evaluated by bone marrow biopsy at 12 months follow up.

Time Frame

12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

75 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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. Exclusion Criteria: 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;

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Bing Han, Docter

Phone

+8613601059938

hanbing_li@sina.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Bing Han, Master

Organizational Affiliation

Peking Union Medical College Hospital

Official's Role

Study Director

Facility Information:

Facility Name

Peking Union Medical College Hospital

City

Beijing

ZIP/Postal Code

100730

Country

China

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

individual participant data would be accepted upon request

IPD Sharing Time Frame

10 years

IPD Sharing Access Criteria

email request

Citations:

PubMed Identifier

33632264

Citation

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.

Results Reference

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Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia

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