Evaluating Zanubrutinib for Autoimmune Hemolytic Anemia
A Phase II, Single Center, Open-label Trial to Determine the Safety and Efficacy of Zanubrutinib in Refractory/Relapsed Autoimmune Hemolytic
This study is testing if a new drug called Zanubrutinib can help people with tough-to-treat autoimmune hemolytic anemia feel better.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 33 (estimated) |
| Ages | 6 Years to 70 Years |
| Sex | All |
| Sponsor | Institute of Hematology & Blood Diseases Hospital, China Academic / other |
| Drugs / interventions | rituximab |
| Locations | 1 site (Tianjin, Tianjin) |
| Trial ID | NCT06021977 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the safety and efficacy of Zanubrutinib in patients with refractory or relapsed autoimmune hemolytic anemia (AIHA). The study employs a Simon's two-stage design, initially enrolling 12 patients, with the potential to expand to 26 based on response rates after 12 weeks. The goal is to determine if more than 14 out of 26 patients achieve a positive response, which would indicate the treatment's effectiveness. The trial is designed to accommodate a dropout rate, aiming for a final sample size of 33 participants.
Who should consider this trial
Good fit: Ideal candidates include individuals aged 6 to 70 diagnosed with Coombs-negative AIHA or warm AIHA who have relapsed or refractory conditions.
Not a fit: Patients with cold agglutinin disease, active connective tissue diseases, or those who have received certain prior treatments may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from refractory or relapsed autoimmune hemolytic anemia.
How similar studies have performed: While this approach is novel for AIHA, similar studies using BTK inhibitors have shown promise in other hematological conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Tianjin, Tianjin
- Regenerative Medicine Center — Tianjin, Tianjin, China (Recruiting)
Study contacts
- Principal investigator: Liwei Fang, MD — Institute of Hematology & Blood Diseases Hospital, China
- Study coordinator: Weiwang Li, PhD
- Email: liweiwang@ihcams.ac.cn
- Phone: 15332132036
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.