A Study of Zanubrutinib in Patients With ITP

A Study of Zanubrutinib in Patients With Primary Immune Thrombocytopenia (ITP)

This project was undertaken to evaluate the efficacy and safety of BTK inhibitor Zanubrutinib for the secondary treatment of adults with primary immune thrombocytopenia (ITP).

Study Overview

• Status: Completed
• Conditions: Immune Thrombocytopenia; Bruton Tyrosine Kinase
• Intervention / Treatment: Drug: Zanubrutinib

Detailed Description

The investigators are undertaking a prospective trial of 10 adults with ITP in China. Zanubrutinib is administered as 80 mg po. qd for 6 weeks. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

• Study Type: Interventional
• Enrollment (Actual): 20
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100010
      • Peking University Insititute of Hematology, Peking University People's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Confirmed primary refractory ITP
• Platelet counts <30×10^9/L or with bleeding symptoms
• Willing and able to sign written informed consent

Exclusion Criteria:

• Secondary thrombocytopenia
• Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit
• HIV infection or hepatitis B virus or hepatitis C virus infections
• Malignancy
• Severe medical condition (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia)
• Nursing or pregnant patients
• Patients who are deemed unsuitable for the study by the investigator

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Zanubrutinib; Zanubrutinib 80mg po qd 6 weeks	Drug: Zanubrutinib; Zanubrutinib 80mg po qd 6 weeks

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall response (OR)	The maintenance of platelet count ≥ 30 x 10^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-week follow-up.	6 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Complete response (CR)	Complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding.	6 weeks
Time to response	The time from starting treatment to time of achievement of Response.	6 weeks
Bleeding events	The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss.	6 weeks
Side Effects	Potential side effects include leukopenia, nausea and diarrhea, infectious diseases, etc .	6 weeks

Collaborators and Investigators

• Sponsor: Peking University People's Hospital
• Investigators: Principal Investigator: Xiao-Hui Zhang, MD, Peking University People's Hospital, Peking University Institute of Hematology, National Clinical Research Center for Hematologic Disease, Beijing Key Laboratory of Hematopoietic Stem Cell Transplantation, Collaborative Innovation Center of Hematology

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2022
• Primary Completion (Actual): October 30, 2022
• Study Completion (Actual): October 30, 2022

Study Registration Dates

• First Submitted: January 24, 2022
• First Submitted That Met QC Criteria: March 6, 2022
• First Posted (Actual): March 15, 2022

Study Record Updates

• Last Update Posted (Estimated): October 10, 2023
• Last Update Submitted That Met QC Criteria: October 7, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia; Molecular Mechanisms of Pharmacological Action; Enzyme Inhibitors; Antineoplastic Agents; Protein Kinase Inhibitors; Zanubrutinib
• Other Study ID Numbers: PKU-ITP032

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No