Avatrombopag in the Treatment of Adult Immune Thrombocytopenia With Autoantibodies

Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorInstitute of Hematology & Blood Diseases Hospital, China

About this trial

This prospective, open-label, single-center, one-arm clinical trial aims at evaluating the efficacy and safety of avatrombopag in Chinese adult Immune Thrombocytopenia (ITP) patients with autoantibodies fail (due to intolerance or resistance) to eltrombopag or herombopag treatment.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

The patients have provided written informed consent prior to enrollment.

Men and women greater than or equal to 18 years of age.

Diagnosed as ITP secondary to connective tissue diseases (including but not limited to systemic lupus erythematosus, Sjogren's syndrome and rheumatoid arthritis), primary ITP with positive antinuclear antibody but not up to the diagnostic criteria of connective tissue diseases, primary Evans syndrome, Evans syndrome secondary to connective tissue diseases, and primary ITP with positive Coomb's test but not up to the diagnostic criteria of Evans syndrome.

Platelet count<30 ×10^9/L at screening.

Disqualifiers

Patients with active thyroid disease requiring treatment.

Patients with any prior history of arterial or venous thrombosis within 3 months, and with any of the following risk factors: cancer, Factor V Leiden, ATIII deficiency, or patients who are using anticoagulants or antiplatelet drugs at the beginning of screening.

Those who had received rituximab within 3 months.

Patients who had failed to respond to the previous use of Avatrombopag (40mg once a day for more than 4 weeks).

Trial design

Design model

Single group

Treatments tested in this trial

  • Avatrombopag

    Drug

    The subjects will receive avatrombopag treatment with an initial dose of 40mg once a day. Platelet counts will be obtained weekly during the first 4 weeks and then every 2 weeks until week 12 after treatment. The dose adjustment range is 20 mg per week to 40 mg per day to maintain the platelet level between 30×10\^9/L and 200×10\^9/L. If the platelet count does not reach to 30×10\^9/L after taking avatrombopag 40mg once a day for 4 consecutive weeks, the treatment will be stopped.

Treatment groups

52 Participants
are divided into 1 treatment group
Group A: Treatment groupExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Platelet response

Percentage of participants achieving a platelet count \>=30×10\^9/L and at least doubling of the baseline count within 12 weeks of treatment.

Time frame
From the start of study treatment (Day 1) up to the end of week 12.

Secondary outcomes

1

Platelet response

Proportion of subjects who achieve response (R) within 1, 2, 4 and 8 weeks of treatment.

Time frame
From the start of study treatment (Day 1) up to the end of week 12.
2

Platelet response

Proportion of subjects who achieve complete response (CR) within 4, 8 and 12 weeks of treatment.

Time frame
From the start of study treatment (Day 1) up to the end of week 12.
3

Duration of platelet response

Proportion of subjects with a platelet count \>=30×10\^9/L for at least 4 consecutive weeks during the 12 week treatment period without remedial treatment.

Time frame
From the start of study treatment (Day 1) up to the end of week 12.
4

Platelet response

Percentage of participants achieving a platelet count \>=50×10\^9/L within 12 weeks of treatment.

Time frame
From the start of study treatment (Day 1) up to the end of week 12.

Other outcomes

1

Incidence of Toxicity

The proportion of subjects with specific pre-defined toxicity, including headache, fever, nausea and abdominal pain, and unpredictable toxicity.

Time frame
From the start of study treatment (Day 1) up to the end of week 12.

Sponsors and contacts

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