Safety and Efficacy of Zanubrutinib in the Treatment of Antiphospholipid Syndrome With Secondary Thrombocytopenia

NCT05199909 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: IIT2021048
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 2

Brief Summary

To evaluate the safety and efficacy of zanubrutinib in the treatment of antiphospholipid syndrome with secondary thrombocytopenia in 10 patients.

Conditions

Antiphospholipid Syndrome; Thrombocytopenia; Treatment

Outcome Measures

Primary Outcomes (1)

• Proportion of subjects with a platelet count ≥ 30 × 10^9/L and 50×10^9/L at week 12(Day 85)

  Observe the changes of blood routine platelet count after 12 weeks of treatment, and calculate the proportion and times of subjects ≥ 30 × 10\^9/L and 50 × 10\^9/L.

  12 weeks

Secondary Outcomes (5)

• Persistent platelet response with clinical significance at 24 weeks

  24 weeks

• Number of participants with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale.

  24 weeks

• The occurrence of adverse events during treatment (AE/SAE), treatment-related adverse events (TRAE) and serious adverse events (TRSAE)

  24 weeks

• Evaluate the occurrence of thrombosis during treatment

  24 weeks

• Antiphospholipid antibody titre

  24 weeks

Study Arms (1)

Intervention ( zanubrutinib)

EXPERIMENTAL

10 enrolled patients are picked up to take zanubrutinib at the indicated dose.

Drug: zanubrutinib

Interventions

zanubrutinib DRUG

The initial dose is 80mg/day. If the treatment is ineffective after 4 weeks, and under the condition of good safety, the researcher will judge that the dosage should be added to 80mg twice/day，or a higher dose for oral maintenance. The maximum dose is 160mg twice a day. The duration of zanubrutinib is 24 weeks. In case of intolerable adverse reactions, such as severe infection, severe bleeding, hematopenia, arrhythmia, etc., investigator can reduce the dose of zanubrutinib, or withdraw from clinical trials as appropriate.

Also known as: The treatment of zanubrutinib

Intervention ( zanubrutinib)

Eligibility Criteria

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

You may qualify if:

• Age 18 and above, male or female；
• Diagnosis of antiphospholipid syndrome；
• Failure to receive glucocorticoid treatment in the past (the curative effect cannot be maintained, or recurs, or cannot be tolerated); Can not choose other second-line treatment, such as rituximab, cyclosporine, cyclophosphamide, etc.; Or rituximab, cyclosporine and other treatments are ineffective, relapsed or intolerable;
• Plt \< 30×10\^9/L；
• Liver and kidney function, such as ALT, AST, BUN, SCR \< 1.5 × upper limit of normal value, passing physical examination;
• ECOG physical state score ≤ 2 points；
• Cardiac function of the New York Society of Cardiac Function ≤ 2；
• Signed and dated written informed consent.

You may not qualify if:

• Uncontrollable primary diseases of important organs, such as malignant tumors, liver failure, heart failure, renal failure and other diseases；
• HIV positive;
• Accompanied by uncontrollable active infection, including hepatitis B, hepatitis C, cytomegalovirus, EB virus and syphilis positive;
• Accompanied by extensive and severe bleeding, such as hemoptysis, upper gastrointestinal hemorrhage, intracranial hemorrhage, etc.;
• At present, there are heart diseases, arrhythmias that need treatment or hypertension that researchers judge is poorly controlled;
• Patients with thrombotic diseases such as new pulmonary embolism and unstable period of various arteriovenous thrombosis;
• Those who have received allogeneic stem cell transplantation or organ transplantation in the past;
• Patients with mental disorders who cannot normally obtain informed consent and conduct trials and follow-up;
• Patients whose toxic symptoms caused by pre-trial treatment have not disappeared;
• Other serious diseases that may limit the subject's participation in this test (such as diabetes; Severe cardiac insufficiency; Myocardial obstruction or unstable arrhythmia or unstable angina pectoris in recent 6 months; Gastric ulcer，etc.);
• Patients with septicemia or other irregular severe bleeding;
• Pregnant women, suspected pregnancies (positive pregnancy test for human chorionic gonadotropin in urine at screening) and lactating patients.

Sponsors & Collaborators

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

Study Sites (2)

Chinese Academy of Medical Science and Blood Disease Hospital

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

MeSH Terms

Conditions

Antiphospholipid Syndrome; Thrombocytopenia

Interventions

zanubrutinib

Condition Hierarchy (Ancestors)

Autoimmune Diseases; Immune System Diseases; Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia

Study Officials

• Lei Zhang, MD

  Chinese Academy of Medical Science and Blood Disease Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yunfei Chen, MD

CONTACT

+8618502220788; chenyunfei@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: SPONSOR

Study Record Dates

• First Submitted: December 30, 2021
• First Posted: January 20, 2022
• Study Start: January 25, 2022
• Primary Completion: December 31, 2023
• Study Completion: June 30, 2025
• Last Updated: February 24, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: 12 months to 36 months after study completion.
• Access Criteria: Upon request to PI.

Locations

CN (2)