Clinical trial NCT06281327: Avatrombopag in the Treatment of Pediatric Immune Thrombocytopenia
Official title: A Prospective, One-arm and Open Clinical Study to Assess Efficacy and Safety of Avatrombopag in the Treatment of Pediatric Primary Immune Thrombocytopenia
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Immune Thrombocytopenia
Treatment
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
Collaborators: name: Henan Cancer Hospital
name: Tianjin Medical University Second Hospital
name: Tianjin Children's Hospital
name: The Second Affiliated Hospital of Kunming Medical University
First posted: 2024-02-28
Enrollment: 60
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Overall efficacy response after AVA treatment within 12 weeks
timeFrame: 12 weeks
Secondary outcomes:
measure: Treatment response-1
timeFrame: 12 weeks
measure: Treatment response-2
timeFrame: 12 weeks
measure: Time to Response
timeFrame: 12 weeks
measure: Persistent response
timeFrame: 12 weeks
measure: Emergency treatment
timeFrame: 12 weeks
measure: Reduction of concomitant drug
timeFrame: 12 weeks
measure: Number of participants with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale.
timeFrame: 12 weeks
measure: Number of participants with clinically significant bleeding as assessed using the bleeding scale for pediatric patients with ITP.
timeFrame: 12 weeks
measure: Health-related quality of life survey of subjects(HRQoL)-1
timeFrame: 12 weeks
measure: Health-related quality of life survey of subjects(HRQoL)-2
timeFrame: 12 weeks
measure: Health-related quality of life survey of subjects(HRQoL)-3
timeFrame: 12 weeks
measure: Health-related quality of life survey of subjects(HRQoL)-4
timeFrame: 12 weeks
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College
Study officials:
affiliation: Institute of Hematology & Blood Diseases Hospital, China
name: Lei Zhang, MD
role: PRINCIPAL_INVESTIGATOR
References: