Health TrialsbyHealthPanel

Avatrombopag in the Treatment of Pediatric Immune Thrombocytopenia

ConditionsImmune ThrombocytopeniaTreatment
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age6-18
SponsorInstitute of Hematology & Blood Diseases Hospital, China

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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;

Disqualifiers

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;

Trial design

Design model

Single group

Treatments tested in this trial

  • 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 groups

60 Participants
are divided into 1 treatment group
Group A: Treatment group: AvatrombopagExperimental treatment 1 intervention

Trial outcomes

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.

Time frame
12 weeks

Secondary outcomes

1

Treatment response-1

Number of participants achieving a platelet count ≥ 50×10\^9/L at week 1,2,4,6,8,12 in absence of rescue therapy.

Time frame
12 weeks
2

Treatment response-2

Number of participants achieving a platelet count ≥ 100×10\^9/L at week 1,2,4,6,8,12 in absence of rescue therapy.

Time frame
12 weeks
3

Time to Response

The time required from the start of treatment to the first time a subject's platelet count was greater than or equal to 30×10\^9/L and at least a two-fold increase from the baseline platelet count without bleeding in absence of rescue therapy.

Time frame
12 weeks
4

Persistent response

Persistent response defined as proportion of subjects with a platelet count ≥ 30 × 10\^9/L and at least 2-fold from baseline without bleeding for 4 consecutive weeks or more within 12 weeks after initial administration in absence of rescue therapy.

Time frame
12 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Institute of Hematology & Blood Diseases Hospital, China

Lead sponsor

Henan Cancer Hospital

Collaborator

Tianjin Medical University Second Hospital

Collaborator

Tianjin Children's Hospital

Collaborator

The Second Affiliated Hospital of Kunming Medical University

Collaborator

Source ClinicalTrials.gov
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