The Safety and Efficacy of Zanubrutinib in Refractory/Rel... | NCT06021977

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

NCT06021977

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Status

Unknown status

Last Update Posted

Nov 29, 2023Actual

Enrollment

33Estimated

Phase

Phase 2

Conditions

Refractory/Relapsed Autoimmune Hemolytic Anemia

Interventions

Zanubrutinib

Countries

China

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Nov 2023

Overall Recruitment Status or Expanded Access Status

Unknown status

Last Known Status

Recruiting

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Sep 1, 2023Actual

Primary Completion Date

Sep 30, 2025Estimated

Completion Date

Dec 31, 2025Estimated

First Submitted Date

Aug 27, 2023

First Submission Date that Met QC Criteria

Aug 27, 2023

First Posted Date

Sep 1, 2023Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Nov 26, 2023

Last Update Posted Date

Nov 29, 2023Actual

Description Module

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

33Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Zanubrutinib	Experimental	Each recruited subject will accept Zanubrutinib treatment.	Drug: Zanubrutinib

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Zanubrutinib	Drug	Zanubrutinib was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity.

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Overall response rate	The percentage of patients achieved complete response, complete response with incomplete hemolysis recovery, and partial response.	within 12 weeks

Secondary Outcomes

Measure	Description	Time Frame
Incidence of adverse events and severe adverse events		within 24 weeks
Relapse free survival rate		within 48 weeks

Other Outcomes

Not provided

Eligibility Module

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Years

Maximum Age

70 Years

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Name	Role	Phone	Extension	Email
Weiwang Li, PhD	Contact	15332132036		liweiwang@ihcams.ac.cn
Jingyu Zhao, MPH	Contact	13752253515		zhaojingyu@ihcams.ac.cn

ID	Term
D000743	Anemia, Hemolytic
D000740	Anemia
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases