Avatrombopag Combined With IST as First-line Treatment for SAA

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Primary Purpose

Status

Recruiting

Phase

Phase 2

Locations

China

Study Type

Interventional

Intervention

avatrombopag

About this trial

This is an interventional treatment trial for Severe Aplastic Anemia focused on measuring avatrombopag

Eligibility Criteria

12 Years - 60 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Patients with newly diagnosed severe aplastic anemia. Men and women aged between 12 and 60. Subjects must complete all screening assessments as outlined in the test protocol. Able to swallow or administer orally. Before the start of the research procedure, the patient or guardian should fully understand the research procedure and purpose and sign the informed consent form. If the patient's signature is not conducive to the treatment of the disease, the patient's immediate family should sign the informed consent form. Exclusion Criteria: Congenital bone marrow failure (eg. Fanconi anemia). Accompanied by cytogenetic cloning changes (chromosomal karyotype and FISH detection found somatic cloning abnormalities; Simple -Y abnormality can be included in this study;) . ATG or middle/high-dose cyclophosphamide was used in the past. Previous treatment with cyclosporine or tacrolimus > 6 months. The total course of treatment with TPO receptor agonists (including thrombopoietin, eltrombopag,hetrombopag and avatrombopag) was more than 1 month. Serious infectious diseases (tuberculosis without effective control, pulmonary aspergillosis, viral infections). AIDS patients. Pregnant or breastfeeding, fertile but unwilling to take effective contraceptive measures. Patients with malignant tumors who are not suitable for ATG treatment. A newly diagnosed history of cardio/cerebral vascular thrombosis within 12 months. Those who are assessed as unsuitable for inclusion by the investigator.

Sites / Locations

Institute of Hematology & Blood Diseases HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treatment group

Arm Description

53 subjects will be enrolled with the indicated treatment dose of avatrombopag.

Outcomes

Primary Outcome Measures

Treatment response

Percentage of patients who achieves complete response(CR) at 12 weeks.

Secondary Outcome Measures

Treatment response

Percentage of patients achieving hematologic response (OR) at 12 weeks.

Treatment response

Percentage of patients achieving hematologic response and complete response (OR and CR) at 24 weeks.

Supportive treatment

The time of red blood cell or platelet recovery to transfusion independence.

Incidence of Treatment-Emergent Adverse Events by CTCAE

Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

Dose-effect relationship

Correlation between avatrombopag's serum concentration with total and complete hematological response rate.

Change of CD34+ cell

Change of CD34+ cells' proportion in bone marrow before and after avatrombopag treatment at week 12 and 24.

Full Information

NCT ID

NCT05720234

First Posted

January 11, 2023

Last Updated

February 7, 2023

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

1. Study Identification

Unique Protocol Identification Number

NCT05720234

Brief Title

Avatrombopag Combined With IST as First-line Treatment for SAA

Official Title

The Efficacy and Safety Study of Avatrombopag Combined With IST as First-line Treatment for Severe Aplastic Anemia, A Single-arm, Phase II Clinical Study

Study Type

Interventional

2. Study Status

Record Verification Date

November 2022

Overall Recruitment Status

Recruiting

Study Start Date

November 10, 2022 (Actual)

Primary Completion Date

November 30, 2023 (Anticipated)

Study Completion Date

November 30, 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

No

Studies a U.S. FDA-regulated Device Product

No

5. Study Description

Brief Summary

This single-center study aims to evaluate the early efficacy and safety of avatrombopag combined with immunosuppressive therapy (IST) in the first-line treatment of severe aplastic anemia (SAA).

Detailed Description

This is a single center, single arm, phase II clinical study. Fifty-three patients will be enrolled. Treatment protocol is as follows: 1) Anti-human thymocyte porcine immunoglobulin (P-ATG 20mg/kg/d) or rabbit anti human thymocyte globulin (R-ATG 3.0mg/kg/d) was administered intravenously for 5 days; 2) Cyclosporine (CSA) is given at 3-5 mg/kg.d in divided doses for at least 6 months. The trough concentration is maintained at 150-250 ng/ml. 3) Avatrombopag is given orally at 60 mg once a day for patients with body weight ≥ 50 kg, and 40 mg orally once a day for patients with body weight<50 kg, for a total of 12 weeks.

6. Conditions and Keywords

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

Severe Aplastic Anemia

Keywords

avatrombopag

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

53 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Treatment group

Arm Type

Experimental

Arm Description

53 subjects will be enrolled with the indicated treatment dose of avatrombopag.

Intervention Type

Drug

Intervention Name(s)

avatrombopag

Other Intervention Name(s)

AVA, APAG

Intervention Description

Patients with body weight ≥50kg were given 60mg/day and patients with body weight < 50kg were given 40mg/day for 12 weeks.

Primary Outcome Measure Information:

Title

Treatment response

Description

Percentage of patients who achieves complete response(CR) at 12 weeks.

Time Frame

From the start of study treatment (Day1) to end of week 12.

Secondary Outcome Measure Information:

Title

Treatment response

Description

Percentage of patients achieving hematologic response (OR) at 12 weeks.

Time Frame

From the start of study treatment (Day1) to end of week 12.

Title

Treatment response

Description

Percentage of patients achieving hematologic response and complete response (OR and CR) at 24 weeks.

Time Frame

From the start of study treatment (Day1) to end of week 24.

Title

Supportive treatment

Description

The time of red blood cell or platelet recovery to transfusion independence.

Time Frame

From the start study treatment (Day1) up to transfusion independence.

Title

Incidence of Treatment-Emergent Adverse Events by CTCAE

Description

Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

Time Frame

From the start study treatment (Day1) up to week 12.

Title

Dose-effect relationship

Description

Correlation between avatrombopag's serum concentration with total and complete hematological response rate.

Time Frame

From the start study treatment(Day1) up to week 24.

Title

Change of CD34+ cell

Description

Change of CD34+ cells' proportion in bone marrow before and after avatrombopag treatment at week 12 and 24.

Time Frame

From the start study treatment(Day1) up to end of week 12 and 24.

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Maximum Age & Unit of Time

60 Years

Accepts Healthy Volunteers

No

Eligibility Criteria

Inclusion Criteria: Patients with newly diagnosed severe aplastic anemia. Men and women aged between 12 and 60. Subjects must complete all screening assessments as outlined in the test protocol. Able to swallow or administer orally. Before the start of the research procedure, the patient or guardian should fully understand the research procedure and purpose and sign the informed consent form. If the patient's signature is not conducive to the treatment of the disease, the patient's immediate family should sign the informed consent form. Exclusion Criteria: Congenital bone marrow failure (eg. Fanconi anemia). Accompanied by cytogenetic cloning changes (chromosomal karyotype and FISH detection found somatic cloning abnormalities; Simple -Y abnormality can be included in this study;) . ATG or middle/high-dose cyclophosphamide was used in the past. Previous treatment with cyclosporine or tacrolimus > 6 months. The total course of treatment with TPO receptor agonists (including thrombopoietin, eltrombopag,hetrombopag and avatrombopag) was more than 1 month. Serious infectious diseases (tuberculosis without effective control, pulmonary aspergillosis, viral infections). AIDS patients. Pregnant or breastfeeding, fertile but unwilling to take effective contraceptive measures. Patients with malignant tumors who are not suitable for ATG treatment. A newly diagnosed history of cardio/cerebral vascular thrombosis within 12 months. Those who are assessed as unsuitable for inclusion by the investigator.

Central Contact Person:

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

Xin Zhao, M.D

Phone

8613702041366

Email

zhaoxin@ihcams.ac.cn

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Xin Zhao

Organizational Affiliation

Institute of Hematology & Blood Diseases Hospital, China

Official's Role

Principal Investigator

Facility Information:

Facility Name

Institute of Hematology & Blood Diseases Hospital

City

Tianjin

State/Province

Tianjin

ZIP/Postal Code

300020

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Xin Zhao, M.D

Phone

13820961539

Email

zhaoxin@ihcams.ac.cn

12. IPD Sharing Statement

Plan to Share IPD

No

Severe Aplastic Anemia

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial