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

Sponsor
Institute of Hematology & Blood Diseases Hospital (Other)
Overall Status
Not yet recruiting
CT.gov ID
NCT06021977
Collaborator
(none)
33
1
1
28
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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

Study Type:
Interventional
Anticipated Enrollment :
33 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase II, Single Center, Open-label Trial to Determine the Safety and Efficacy of Zanubrutinib in Refractory/Relapsed Autoimmune Hemolytic
Anticipated Study Start Date :
Sep 1, 2023
Anticipated Primary Completion Date :
Sep 30, 2025
Anticipated Study Completion Date :
Dec 31, 2025

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

  1. 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

  1. Incidence of adverse events and severe adverse events [within 24 weeks]

  2. Relapse free survival rate [within 48 weeks]

Eligibility Criteria

Criteria

Ages Eligible for Study:
6 Years to 70 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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.

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.
Responsible Party:
Fang Liwei, Physician of Regenerative Medical Center, Institute of Hematology & Blood Diseases Hospital
ClinicalTrials.gov Identifier:
NCT06021977
Other Study ID Numbers:
  • IHBDH-IIT20230711
First Posted:
Sep 1, 2023
Last Update Posted:
Sep 1, 2023
Last Verified:
Aug 1, 2023
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:

Study Results

No Results Posted as of Sep 1, 2023