NCT05720234

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).

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
53

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Nov 2022

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 10, 2022

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

January 11, 2023

Completed
29 days until next milestone

First Posted

Study publicly available on registry

February 9, 2023

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 30, 2023

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

November 30, 2024

Completed
Last Updated

February 9, 2023

Status Verified

November 1, 2022

Enrollment Period

1.1 years

First QC Date

January 11, 2023

Last Update Submit

February 7, 2023

Conditions

Severe Aplastic Anemia

Keywords

avatrombopag

Outcome Measures

Primary Outcomes (1)

  • 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 (6)

  • Treatment response

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

  • Treatment response

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

  • Supportive treatment

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

  • Incidence of Treatment-Emergent Adverse Events by CTCAE

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

  • Dose-effect relationship

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

  • +1 more secondary outcomes

Study Arms (1)

Treatment group

EXPERIMENTAL

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

Drug: avatrombopag

Interventions

avatrombopagDRUG

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

Also known as: AVA, APAG
Treatment group

Eligibility Criteria

Age12 Years - 60 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

avatrombopag

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow Diseases

Study Officials

  • Xin Zhao

    Institute of Hematology & Blood Diseases Hospital, China

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Xin Zhao, M.D

CONTACT

8613702041366zhaoxin@ihcams.ac.cn

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 11, 2023

First Posted

February 9, 2023

Study Start

November 10, 2022

Primary Completion

November 30, 2023

Study Completion

November 30, 2024

Last Updated

February 9, 2023

Record last verified: 2022-11

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)