Avatrombopag in the Treatment of Pediatric Immune Thrombocytopenia

NCT06281327 | Recruiting Phase 2 DMC

• 1 other identifier: IIT2023062
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

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.

Conditions

Immune Thrombocytopenia; Treatment

Outcome Measures

Primary Outcomes (1)

• Overall efficacy response after AVA treatment within 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.

  12 weeks

Secondary Outcomes (12)

• Treatment response-1

  12 weeks

• Treatment response-2

  12 weeks

• Time to Response

  12 weeks

• Persistent response

  12 weeks

• Emergency treatment

  12 weeks

Study Arms (1)

Treatment group: Avatrombopag

EXPERIMENTAL

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

Drug: Avatrombopag

Interventions

Avatrombopag 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.

Treatment group: Avatrombopag

Eligibility Criteria

• Age: 6 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead
• Henan Cancer Hospital: collaborator
• Tianjin Medical University Second Hospital: collaborator
• Tianjin Children's Hospital: collaborator
• The Second Affiliated Hospital of Kunming Medical University: collaborator

Study Sites (1)

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

Tianjin, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

avatrombopag

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Lei Zhang, MD

  Institute of Hematology & Blood Diseases Hospital, China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Lei Zhang, MD

CONTACT

+86 13502118379; zhanglei1@ihcams.ac.cn

Ting Sun, MD

CONTACT

+8615822339131; sunting@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 20, 2024
• First Posted: February 28, 2024
• Study Start: March 1, 2024
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: February 24, 2025

Record last verified: 2025-02

Locations

CN (1)