Using avatrombopag to treat pediatric immune thrombocytopenia

A Prospective, One-arm and Open Clinical Study to Assess Efficacy and Safety of Avatrombopag in the Treatment of Pediatric Primary Immune Thrombocytopenia

This study is testing if a new medication called avatrombopag can help children aged 6 to 18 with immune thrombocytopenia who didn't do well on their previous treatment.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Ages: 6 Years to 18 Years
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (Academic / other)
• Drugs / interventions: rituximab
• Locations: 1 site (Tianjin)
• Trial ID: NCT06281327 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of avatrombopag in children aged 6 to 18 years with primary immune thrombocytopenia (ITP) who have previously been treated with eltrombopag. The study focuses on patients who switched to avatrombopag due to poor efficacy, excessive platelet fluctuation, intolerance, or other reasons. Participants will be monitored for their platelet counts and any adverse effects during the treatment period. The goal is to determine if avatrombopag can provide a more effective treatment option for these patients.

Who should consider this trial

Good fit: Ideal candidates are children aged 6-18 years diagnosed with primary immune thrombocytopenia who have previously received eltrombopag and switched to avatrombopag.

Not a fit: Patients with secondary thrombocytopenia or significant organ dysfunction may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a more effective option for managing pediatric immune thrombocytopenia, improving platelet counts and reducing bleeding risks.

How similar studies have performed: While there have been studies on eltrombopag, this approach with avatrombopag specifically for pediatric ITP is relatively novel.

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.

Where this trial is running

Tianjin

• Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College — Tianjin, China (Recruiting)

Study contacts

• Principal investigator: Lei Zhang, MD — Institute of Hematology & Blood Diseases Hospital, China
• Study coordinator: Lei Zhang, MD

Conditions Immune Thrombocytopenia; Treatment