Back to results

Efficacy and Safety of Avatrombopag Combined With IST for the Treatment of HAAA and SAA With Abnormal Liver Function

Primary Purpose

Aplastic Anemia

Status

Recruiting

Phase

Not Applicable

Locations

China

Study Type

Interventional

Intervention

Avatrombopag 20 MG Oral Tablet

About this trial

This is an interventional treatment trial for Aplastic Anemia focused on measuring Avatrombopag, aplastic anemia, abnormal liver function, hepatitis

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

patients with V/SAA with a definite diagnosis.
age between 18-70 years, male or female.
Subjects must complete all screening assessments as outlined in the trial protocol.
Able to swallow or administer the drug orally.
No prior application of TPO receptor agonists (including Thrombopoietin, Eltrombopag, Hetrombopag, etc.) or application of TPO receptor agonists for treatment with ≤ 5 total doses and ≤ 7 days of TPO receptor agonist drugs such as Eltrombopag, Hetrombopag, etc.
Diagnosis as HAAA or abnormal liver function. ALT and AST more than 1.5 times of upper limit.
Informed consent must be signed prior to the start of all specific study procedures, in consideration of the patient's condition, or by a member of the patient's immediate family if the patient's signature is not conducive to the treatment of the condition.

Exclusion Criteria:

Known diagnosis of congenital hematopoietic failure disorders (e.g. Fanconi anemia) and other causes of allogeneic cytopenias and bone marrow hypoproliferative disorders (e.g. hemolytic PNH, hypoproliferative MDS/AML, autoantibody-mediated allogeneic cytopenias, etc.);
Patients with uncontrolled bleeding and/or infection despite standard treatment.
Patients with previous history of hematopoietic stem cell transplantation; previous history of thrombosis.
Patients with concurrent malignancy or potential cancer on immunosuppressive therapy.
Those who are considered unsuitable for enrollment by the investigator.

Sites / Locations

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical SciencesRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Avatrombopag+CsA+ p-ATG

Arm Description

Patients received p-ATG for 5 consecutive days (day 1-5), at a dose of 20 mg/kg/day. CSA is started at 3 mg/kg orally in two doses. Concentrations maintained at 200-250 ng/ml to achieve maximum efficacy and then tapered by 25 mg every 3 months; Avatrombopag: 40 mg orally once daily for a total of 12 weeks. A total of 39 patients were expected to be included.

Outcomes

Primary Outcome Measures

CR rate at 12 weeks of treatment

Percentage of the total number of patients receiving treatment who received a complete response at 12 weeks of treatment

ncidence of Treatment-Emergent Adverse Events as assessed by information on Common Toxicity Criteria (CTC) AE grading at 12 weeks of treatment

Incidence of Treatment-Emergent AE by CTCAE

Secondary Outcome Measures

OR rate at 12 weeks of treatment

Percentage of the total number of patients receiving treatment who received a response at 12 weeks of treatment

Full Information

NCT ID

NCT05571332

First Posted

August 28, 2022

Last Updated

October 4, 2022

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

1. Study Identification

Unique Protocol Identification Number

NCT05571332

Brief Title

Efficacy and Safety of Avatrombopag Combined With IST for the Treatment of HAAA and SAA With Abnormal Liver Function

Official Title

Efficacy and Safety of Avatrombopag Combined With IST for the Treatment of HAAA and SAA With Abnormal Liver Function

Study Type

Interventional

2. Study Status

Record Verification Date

August 2022

Overall Recruitment Status

Recruiting

Study Start Date

June 28, 2022 (Actual)

Primary Completion Date

December 28, 2023 (Anticipated)

Study Completion Date

June 28, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Institute of Hematology & Blood Diseases Hospital, China

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

This is a multicenter, single-arm clinical study. The objective was to evaluate the efficacy and safety of Avatrombopag combined with IST in very/sever aplastic anemia patients with abnormal liver function or HAAA patients treated for the first time. The design was: Patients received p-ATG for 5 consecutive days (day 1-5), at a dose of 20 mg/kg/day. Cyclosporine 3 mg/kg orally in two divided doses, with cyclosporine trough concentrations maintained at 200-250 ng/ml for 3 months to achieve maximum efficacy, and Avatrombopag, which was administered in the dose of 40 mg orally once daily for a total of 12 weeks. Thirty-nine patients are expected to be enrolled in this study. Evaluation endpoint: complete response rate at 12 weeks of treatment.

Detailed Description

This is a multicenter, single-arm clinical study to evaluate the efficacy and safety of Avatrombopag combined with IST as the first-line regimen for aplastic anemia. The patients are diagnosed as hepatitis associated with very sever/sever aplastic anemia(V/SAA) or V/SAA with abnormal liver function before treatment. Patients received p-ATG for 5 consecutive days (day 1-5), at a dose of 20 mg/kg/day. CSA is started at 3 mg/kg orally in two doses. Concentrations maintained at 200-250 ng/ml to achieve maximum efficacy and then tapered by 25 mg every 3 months; Avatrombopag: 40 mg orally once daily for a total of 12 weeks. A total of 39 patients were expected to be included.Complete response rate at 12 weeks of treatment and adverse events are the evaluation endpoint.Secondary study endpoints were: ORR at 12 , CRR and ORR at 24 weeks, survival, and clonal evolution in follow-up.

6. Conditions and Keywords

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

Aplastic Anemia

Keywords

Avatrombopag, aplastic anemia, abnormal liver function, hepatitis

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

39 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Avatrombopag+CsA+ p-ATG

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

Avatrombopag 20 MG Oral Tablet

Other Intervention Name(s)

porcine ATG, Cyclosporine(CsA)

Intervention Description

p-ATG and CsA in combination with Avatrombopag to treat

Primary Outcome Measure Information:

Title

CR rate at 12 weeks of treatment

Description

Percentage of the total number of patients receiving treatment who received a complete response at 12 weeks of treatment

Time Frame

12 weeks of treatment

Title

ncidence of Treatment-Emergent Adverse Events as assessed by information on Common Toxicity Criteria (CTC) AE grading at 12 weeks of treatment

Description

Incidence of Treatment-Emergent AE by CTCAE

Time Frame

12 weeks of treatment

Secondary Outcome Measure Information:

Title

OR rate at 12 weeks of treatment

Description

Percentage of the total number of patients receiving treatment who received a response at 12 weeks of treatment

Time Frame

12 weeks of treatment

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

70 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: patients with V/SAA with a definite diagnosis. age between 18-70 years, male or female. Subjects must complete all screening assessments as outlined in the trial protocol. Able to swallow or administer the drug orally. No prior application of TPO receptor agonists (including Thrombopoietin, Eltrombopag, Hetrombopag, etc.) or application of TPO receptor agonists for treatment with ≤ 5 total doses and ≤ 7 days of TPO receptor agonist drugs such as Eltrombopag, Hetrombopag, etc. Diagnosis as HAAA or abnormal liver function. ALT and AST more than 1.5 times of upper limit. Informed consent must be signed prior to the start of all specific study procedures, in consideration of the patient's condition, or by a member of the patient's immediate family if the patient's signature is not conducive to the treatment of the condition. Exclusion Criteria: Known diagnosis of congenital hematopoietic failure disorders (e.g. Fanconi anemia) and other causes of allogeneic cytopenias and bone marrow hypoproliferative disorders (e.g. hemolytic PNH, hypoproliferative MDS/AML, autoantibody-mediated allogeneic cytopenias, etc.); Patients with uncontrolled bleeding and/or infection despite standard treatment. Patients with previous history of hematopoietic stem cell transplantation; previous history of thrombosis. Patients with concurrent malignancy or potential cancer on immunosuppressive therapy. Those who are considered unsuitable for enrollment by the investigator.

Central Contact Person:

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

Fengkui Zhang, Dr.

Phone

+8602223909229

zhangfenkui@ihcams.ac.cn

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

Wenrui Yang, Dr.

Phone

+8602223909223

yangwenrui@ihcams.ac.cn

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Wenrui Yang

Organizational Affiliation

Anemia Therapeutic center

Official's Role

Study Director

Facility Information:

Facility Name

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

City

Tianjin

State/Province

Tianjin

ZIP/Postal Code

300020

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Fengkui Zhang, Doctor

Phone

8602223909229

fkzhang@ihcams.ac.cn

First Name & Middle Initial & Last Name & Degree

Liping Jing, Doctor

Phone

8602223909223

jingliping@ihcams.ac.cn

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

You can ask for the researcher after completing the experiment

IPD Sharing Time Frame

Follow-up and publication of the paper are planned for December 2024

IPD Sharing Access Criteria

After the paper is written and published

Citations:

PubMed Identifier

18379007

Citation

Young NS, Kaufman DW. The epidemiology of acquired aplastic anemia. Haematologica. 2008 Apr;93(4):489-92. doi: 10.3324/haematol.12855. No abstract available.

Results Reference

result

PubMed Identifier

30504345

Citation

Scheinberg P. Activity of eltrombopag in severe aplastic anemia. Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):450-456. doi: 10.1182/asheducation-2018.1.450.

Results Reference

result

Learn more about this trial

Efficacy and Safety of Avatrombopag Combined With IST for the Treatment of HAAA and SAA With Abnormal Liver Function

We'll reach out to this number within 24 hrs