Institute of Hematology & Blood Diseases Hospital, China
Phase
Phase 2
Started
2024-03-01
Last updated
2025-02-24
Condition(s) studied
Immune ThrombocytopeniaTreatment
Investigational drug(s) / intervention(s)
Avatrombopag
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.
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
Sex
ALL
Min age
6 Years
Max age
18 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
Overall efficacy response after AVA treatment within 12 weeks — 12 weeks Overall response rate defined as proportion of subjects with a platelet count ≥ 30 × 10\^9/L and at least 2-fold from baseline without bleeding at the meanwhile within 12 weeks after initial administration in absence of rescue therapy.
Trial sites (1)
Facility
City
Region
Status
Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Tianjin
China
Recruiting
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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