The Safety and Efficacy of Zanubrutinib in Refractory/Relapsed Autoimmune Hemolytic Anemia
Study Details
Study Description
Brief Summary
The sample size of this study is calculated based on Simon's two-stage design. The first stage of the study enrolled a cohort of 12 patients. If after 12 weeks at least 6 patients achieved a response, then enrollment was expanded to a total of 26 patients. The null hypothesis was unaccepted if more than 14 of 26 patients achieved the response. Accounting for a 20% dropout rate, the estimated final sample size was 33 patients.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Zanubrutinib Each recruited subject will accept Zanubrutinib treatment. |
Drug: Zanubrutinib
Zanubrutinib was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity.
|
Outcome Measures
Primary Outcome Measures
- Overall response rate [within 12 weeks]
The percentage of patients achieved complete response, complete response with incomplete hemolysis recovery, and partial response.
Secondary Outcome Measures
- Incidence of adverse events and severe adverse events [within 24 weeks]
- Relapse free survival rate [within 48 weeks]
Eligibility Criteria
Criteria
Inclusion Criteria:
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Age from 6 to 70
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Diagnosis of Coombs-negative AIHA
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Diagnosis of warm AIHA, mixed AIHA or Evans syndrome.
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Meets the criteria of relapsed / refractory AIHA
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ECOG ≤ 3
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Willing and able to comply with the requirements for this study and written informed consent.
Exclusion Criteria:
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Diagnosis of any of the following diseases: Cold agglutinin disease, cold agglutinin syndrome, mixed AIHA, paroxysmal cold hemoglobinuria (PCH).
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Diagnosis of active stage of connective tissue disease.
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History of lymphoproliferative tumors or any other malignant.
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Diagnosis of other inherited or acquired hemolytic diseases.
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Secondary AIHA caused by drugs or infection.
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Previously received organ or stem cell transplantation.
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History of thrombosis or organ infarction.
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Received rituximab within 8 weeks before enrollment.
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Previously treated with BTK inhibitor ≥ 2 weeks.
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Received low-molecular-weight heparin or warfarin within 1 week before enrollment and need to continue the drug treatment.
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Received CYP3A4 Enzyme Inhibitors or Inducers within 1 week before enrollment and need to continue the drug treatment.
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Uncontrolled systemic fungal, bacterial, or viral infection (defined as ongoing signs/symptoms related to the infection without improvement despite appropriate antibiotics, antiviral therapy, and/or other treatment), known human immunodeficiency virus (HIV), known evidence of active infectious hepatitis B, and/or known evidence of active hepatitis C.
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Any severe and/or uncontrolled medical conditions: refractory hypertension, clinically significant cardiac diseases, renal diseases, liver diseases and metabolic diseases, etc.
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History of mental illness.
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Participation in another clinical trial within 4 weeks before the start of this trial.
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Pregnant or breast-feeding patients.
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Patients considered ineligible for the study by the investigator for reasons other than the above.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Regenerative Medicine Center | Tianjin | Tianjin | China | 300131 |
Sponsors and Collaborators
- Institute of Hematology & Blood Diseases Hospital
Investigators
- Principal Investigator: Liwei Fang, MD, Institute of Hematology & Blood Diseases Hospital
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- IHBDH-IIT20230711