Avatrombopag in the Treatment of Pediatric Immune Thrombocytopenia

NCT ID: NCT06281327

Last Updated: 2025-02-24

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 60 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-03-01
• Study Completion Date: 2025-12-31

Brief 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.

Detailed 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.

Conditions

Immune Thrombocytopenia; Treatment

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment group: Avatrombopag

Sixty subjects will be enrolled with the indicated treatment dose of avatrombopag

Group Type: EXPERIMENTAL

Avatrombopag

Intervention Type: DRUG

After enrollment, all subjects receive Avatrombopag treatment. The initial dose of Avatrombopag administration was an oral 10 mg (<30kg) or 20mg (≥30kg) once daily in all participants. ps. For subjects weighing ≥30kg, the dose can be started from 40mg once daily if the platelet count is < 10×10^9/L or if there is severe bleeding or risk of bleeding.

Complete blood count including platelet count was done once a week. The dose of Avatrombopag was adjusted according to the subject platelet count during the period from week 1 to week 24. If the platelet count was less than 30×10^9/L for 2 weeks, avatrobopag was gradually increased. If the platelet count was greater than 150 x 10^9/L, avatrobopag was gradually reduced. If the platelet count >250×10^9/L, avatrobopag will be stoped until the platelet count <100×10^9/L.

Interventions

Avatrombopag

After enrollment, all subjects receive Avatrombopag treatment. The initial dose of Avatrombopag administration was an oral 10 mg (<30kg) or 20mg (≥30kg) once daily in all participants. ps. For subjects weighing ≥30kg, the dose can be started from 40mg once daily if the platelet count is < 10×10^9/L or if there is severe bleeding or risk of bleeding.

Complete blood count including platelet count was done once a week. The dose of Avatrombopag was adjusted according to the subject platelet count during the period from week 1 to week 24. If the platelet count was less than 30×10^9/L for 2 weeks, avatrobopag was gradually increased. If the platelet count was greater than 150 x 10^9/L, avatrobopag was gradually reduced. If the platelet count >250×10^9/L, avatrobopag will be stoped until the platelet count <100×10^9/L.

Intervention Type: DRUG

Eligibility Criteria

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 to avatrombopag treatment because of ineffectiveness (platelet count < 30×10^9/L after eltrombopag treatment, or platelet count increased less than 2 times of the basic value, 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 Criteria

• Secondary thrombocytopenia caused by various reasons, such as connective tissue disorders, 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.), or with 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 the reliability of platelet count measurements；
• Any medical history or condition that the investigator deems unsuitable for participation in the study.

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Henan Cancer Hospital

OTHER_GOV

Sponsor Role: collaborator

Tianjin Medical University Second Hospital

OTHER

Sponsor Role: collaborator

Tianjin Children's Hospital

OTHER

Sponsor Role: collaborator

The Second Affiliated Hospital of Kunming Medical University

OTHER

Sponsor Role: collaborator

Institute of Hematology & Blood Diseases Hospital, China

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Lei Zhang, MD

Role: PRINCIPAL_INVESTIGATOR

Institute of Hematology & Blood Diseases Hospital, China

Locations

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

Tianjin, , China

Site Status: RECRUITING

Countries

China

Central Contacts

Lei Zhang, MD

Role: CONTACT

zhanglei1@ihcams.ac.cn

+86 13502118379

Ting Sun, MD

Role: CONTACT

sunting@ihcams.ac.cn

+8615822339131

Facility Contacts

Lei Zhang, MD

Role: primary

zhanglei1@ihcams.ac.cn

+8602223909009

Ting Sun, MD

Role: backup

sunting@ihcams.ac.cn

+8602223909009

Other Identifiers

IIT2023062

Identifier Type: -

Identifier Source: org_study_id