Clinical trial NCT06021977: The Safety and Efficacy of Zanubrutinib in Refractory/Relapsed Autoimmune Hemolytic Anemia
Official title: A Phase II, Single Center, Open-label Trial to Determine the Safety and Efficacy of Zanubrutinib in Refractory/Relapsed Autoimmune Hemolytic
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Refractory/Relapsed Autoimmune Hemolytic Anemia
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
First posted: 2023-09-01
Enrollment: 33
Sex: ALL
Minimum age (years): 6
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Overall response rate
timeFrame: within 12 weeks
Secondary outcomes:
measure: Incidence of adverse events and severe adverse events
timeFrame: within 24 weeks
measure: Relapse free survival rate
timeFrame: within 48 weeks
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Regenerative Medicine Center
state: Tianjin
Study officials:
affiliation: Institute of Hematology & Blood Diseases Hospital, China
name: Liwei Fang, MD
role: PRINCIPAL_INVESTIGATOR
References: