The Safety and Efficacy of Zanubrutinib in Refractory/Relapsed Autoimmune Hemolytic Anemia

NCT06021977 | Unknown Phase 2

• 1 other identifier: IHBDH-IIT20230711
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 1

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.

Conditions

Refractory/Relapsed Autoimmune Hemolytic Anemia

Outcome Measures

Primary Outcomes (1)

• Overall response rate

  The percentage of patients achieved complete response, complete response with incomplete hemolysis recovery, and partial response.

  within 12 weeks

Secondary Outcomes (2)

• Incidence of adverse events and severe adverse events

  within 24 weeks

• Relapse free survival rate

  within 48 weeks

Study Arms (1)

Zanubrutinib

EXPERIMENTAL

Each recruited subject will accept Zanubrutinib treatment.

Drug: Zanubrutinib

Interventions

Zanubrutinib DRUG

Zanubrutinib was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity.

Zanubrutinib

Eligibility Criteria

• Age: 6 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Regenerative Medicine Center

Tianjin, Tianjin Municipality, 300131, China

RECRUITING

MeSH Terms

Conditions

Anemia, Hemolytic, Autoimmune

Interventions

zanubrutinib

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Liwei Fang, MD

  Institute of Hematology & Blood Diseases Hospital, China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Weiwang Li, PhD

CONTACT

15332132036; liweiwang@ihcams.ac.cn

Jingyu Zhao, MPH

CONTACT

13752253515; zhaojingyu@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator of Regenerative Medical Center

Study Record Dates

• First Submitted: August 27, 2023
• First Posted: September 1, 2023
• Study Start: September 1, 2023
• Primary Completion: September 30, 2025
• Study Completion: December 31, 2025
• Last Updated: November 29, 2023

Record last verified: 2023-11

Locations

CN (1)