Refractory/Relapsed Autoimmune Hemolytic Anemia Clinical Trial
Official title:
A Phase II, Single Center, Open-label Trial to Determine the Safety and Efficacy of Zanubrutinib in Refractory/Relapsed Autoimmune Hemolytic
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.
| Status | Recruiting |
| Enrollment | 33 |
| Est. completion date | December 31, 2025 |
| Est. primary completion date | September 30, 2025 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 6 Years to 70 Years |
| Eligibility | Inclusion Criteria: - Age from 6 to 70 - Diagnosis of Coombs-negative AIHA - Diagnosis of warm AIHA, mixed AIHA or Evans syndrome. - Meets the criteria of relapsed / refractory AIHA - ECOG = 3 - Willing and able to comply with the requirements for this study and written informed consent. Exclusion Criteria: - Diagnosis of any of the following diseases: Cold agglutinin disease, cold agglutinin syndrome, mixed AIHA, paroxysmal cold hemoglobinuria (PCH). - Diagnosis of active stage of connective tissue disease. - History of lymphoproliferative tumors or any other malignant. - Diagnosis of other inherited or acquired hemolytic diseases. - Secondary AIHA caused by drugs or infection. - Previously received organ or stem cell transplantation. - History of thrombosis or organ infarction. - Received rituximab within 8 weeks before enrollment. - Previously treated with BTK inhibitor = 2 weeks. - Received low-molecular-weight heparin or warfarin within 1 week before enrollment and need to continue the drug treatment. - Received CYP3A4 Enzyme Inhibitors or Inducers within 1 week before enrollment and need to continue the drug treatment. - 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. - Any severe and/or uncontrolled medical conditions: refractory hypertension, clinically significant cardiac diseases, renal diseases, liver diseases and metabolic diseases, etc. - History of mental illness. - Participation in another clinical trial within 4 weeks before the start of this trial. - Pregnant or breast-feeding patients. - Patients considered ineligible for the study by the investigator for reasons other than the above. |
| Country | Name | City | State |
|---|---|---|---|
| China | Regenerative Medicine Center | Tianjin | Tianjin |
| Lead Sponsor | Collaborator |
|---|---|
| Institute of Hematology & Blood Diseases Hospital, China |
China,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Overall response rate | The percentage of patients achieved complete response, complete response with incomplete hemolysis recovery, and partial response. | within 12 weeks | |
| Secondary | Incidence of adverse events and severe adverse events | within 24 weeks | ||
| Secondary | Relapse free survival rate | within 48 weeks |
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT04398459 -
The Safety and Efficacy of Ibrutinib in Refractory/Relapsed Autoimmune Hemolytic Anemia
|
Phase 2 |