A Study of Orelabrutinib in Patients With ITP

Study Overview
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Brief Summary
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This project was undertaken to evaluate the efficacy and safety of BTK inhibitor Orelabrutinib for the secondary treatment of adults with primary immune thrombocytopenia (ITP).

Detailed Description
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The investigators are undertaking a prospective trial of 10 adults with ITP in China. Orelabrutinib is administered as 50 mg po. qd for 6 weeks. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

Official Title
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A Study of Orelabrutinib in Patients With Primary Immune Thrombocytopenia (ITP)

Conditions
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Immune Thrombocytopenia

Intervention / Treatment
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* Drug: Orelabrutinib


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Confirmed primary refractory ITP Platelet counts <30×10^9/L or with bleeding symptoms Willing and able to sign written informed consent Exclusion Criteria: Secondary thrombocytopenia Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit HIV infection or hepatitis B virus or hepatitis C virus infections Malignancy Severe medical condition (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia) Nursing or pregnant patients Patients who are deemed unsuitable for the study by the investigator

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Orelabrutinib<br>Orelabrutinib 50mg po qd 6 weeks | Drug: Orelabrutinib<br>* 50mg po qd 6 weeks<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall response (OR) | The maintenance of platelet count ≥ 30 x 10^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-week follow-up. | 6 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Complete response (CR) | Complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding. | 6 weeks | 
| Time to response | The time from starting treatment to time of achievement of Response. | 6 weeks | 
| Bleeding events | The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss. | 6 weeks | 
| Side effects | Potential side effects include leukopenia, nausea and diarrhea, infectious diseases, etc . | 6 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Bruton's Tyrosine Kinase