Avatrombopag in the Treatment of Pediatric Immune Thrombocytopenia

Last updated: February 20, 2025
Sponsor: Institute of Hematology & Blood Diseases Hospital, China
Overall Status: Active - Recruiting

Phase

2

Condition

Thrombosis

Dysfunctional Uterine Bleeding

Platelet Disorders

Treatment

Avatrombopag

Clinical Study ID

NCT06281327
IIT2023062
  • Ages 6-18
  • All Genders

Study Summary

To evaluate the safety and efficacy of avatrombopag in the treatment of pediatric primary immune thrombocytopenia in patients who have been treated with eltrombopag before and switched to avatrobopag because of poor efficacy, excessive platelet fluctuation or intolerance, or patient preference, economic reasons, and other reasons.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Age 6-18 years old (including both ends), male and female;

  • Diagnosed with primary immune thrombocytopenia (ITP);

  • Patients who had previously received eltrombopag treatment and then converted toavatrombopag treatment because of ineffectiveness (platelet count < 30×10^9/L aftereltrombopag treatment, or platelet count increased less than 2 times of the basicvalue, or bleeding) or large platelet fluctuation or due to patient preference,economic reasons and other reasons;

  • Cardiac function of the New York Society of Cardiac Function ≤ 2;

  • Understand the study procedure and voluntarily sign the informed consent.

Exclusion

Exclusion Criteria:

  • Secondary thrombocytopenia caused by various reasons, such as connective tissuedisorders, bone marrow hematopoietic failure disease, myelodysplastic syndrome,malignancy, drugs, inherited thrombocytopenia, common variable immune deficiency,lymphoma, etc.;

  • Subjects with primary disease of important organs (liver, kidney, heart, etc.), orwith immune system diseases;

  • Subjects who are known to be allergic to avatrombopag or any of its excipients;

  • Subjects who had used rituximab within the last 3 months;

  • Subjects who underwent splenectomy within the last 3 months;

  • Subjects with a history of abnormal platelet aggregation that may affect thereliability of platelet count measurements;

  • Any medical history or condition that the investigator deems unsuitable forparticipation in the study.

Study Design

Total Participants: 60
Treatment Group(s): 1
Primary Treatment: Avatrombopag
Phase: 2
Study Start date:
March 01, 2024
Estimated Completion Date:
December 31, 2025

Study Description

Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.).

Eltrombopag is currently the only TPO receptor agonist with indications for pediatric immune thrombocytopenia. However, at present, the treatment response of pediatric ITP is not good, and a considerable number of patients need to switched to other TPO receptor agonist, such as avatrombopag, because of poor efficacy, excessive platelet fluctuation or intolerance, or patient preference, economic reasons, and other reasons.

Therefore, the investigators designed this clinical trial to evaluate the efficacy and safety of avatrombopag in the treatment of pediatric immune thrombocytopenia in patients who who have been treated with eltrombopag before and switched to avatrobopag because of different reasons.

Connect with a study center

  • Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College

    Tianjin,
    China

    Active - Recruiting

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.