Avatrombopag Combined With IST as First-line Treatment fo... | NCT05720234

Studies
Avatrombopag Combined With IST as First-line Treatment for SAA

NCT05720234

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Status

Unknown status

Last Update Posted

Feb 9, 2023Actual

Enrollment

53Estimated

Phase

Phase 2

Conditions

Severe Aplastic Anemia

Interventions

avatrombopag

Countries

China

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Nov 2022

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 10, 2022Actual

Primary Completion Date

Nov 30, 2023Estimated

Completion Date

Nov 30, 2024Estimated

First Submitted Date

Jan 11, 2023

First Submission Date that Met QC Criteria

Feb 7, 2023

First Posted Date

Feb 9, 2023Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 7, 2023

Last Update Posted Date

Feb 9, 2023Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Not provided

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

53Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Treatment group	Experimental	53 subjects will be enrolled with the indicated treatment dose of avatrombopag.	Drug: avatrombopag

Interventions

Name	Type	Description	Arm Group Labels	Other Names
avatrombopag	Drug	Patients with body weight ≥50kg were given 60mg/day and patients with body weight < 50kg were given 40mg/day for 12 weeks.	Treatment group

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Treatment response	Percentage of patients who achieves complete response(CR) at 12 weeks.	From the start of study treatment (Day1) to end of week 12.

Secondary Outcomes

Measure	Description	Time Frame
Treatment response	Percentage of patients achieving hematologic response (OR) at 12 weeks.	From the start of study treatment (Day1) to end of week 12.
Treatment response	Percentage of patients achieving hematologic response and complete response (OR and CR) at 24 weeks.

Other Outcomes

Not provided

Eligibility Module

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

12 Years

Maximum Age

60 Years

Standard Ages

ChildAdult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Xin Zhao, M.D	Contact	8613702041366		zhaoxin@ihcams.ac.cn

Overall Officials

Name	Affiliation	Role
Xin Zhao	Institute of Hematology & Blood Diseases Hospital, China	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Institute of Hematology & Blood Diseases Hospital	Recruiting	Tianjin	Tianjin Municipality	300020	China

ID	Term
D000740	Anemia
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases
D000080983	Bone Marrow Failure Disorders