Using zanubrutinib to treat antiphospholipid syndrome with low platelet counts

Prospective, Single Arm and Open Clinical Observation of Zanubrutinib in the Treatment of Antiphospholipid Syndrome With Secondary Thrombocytopenia

This study is testing if zanubrutinib can help people with antiphospholipid syndrome and low platelet counts who haven't found relief with other treatments.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China (Academic / other)
• Drugs / interventions: rituximab, cyclophosphamide, zanubrutinib
• Locations: 2 sites (Tianjin, Tianjin Municipality and 1 other locations)
• Trial ID: NCT05199909 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety and efficacy of zanubrutinib in patients diagnosed with antiphospholipid syndrome (APS) who also experience secondary thrombocytopenia. The study aims to enroll 10 patients who have not responded to previous treatments such as glucocorticoids or other immunosuppressants. Zanubrutinib, a Bruton tyrosine kinase inhibitor, may help manage the symptoms of APS by targeting B cell signaling pathways. The trial will assess the impact of this treatment on platelet counts and overall disease management.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older with a diagnosis of antiphospholipid syndrome and severe thrombocytopenia who have not responded to other treatments.

Not a fit: Patients with uncontrollable primary diseases of important organs or those with active infections may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with APS and severe thrombocytopenia, potentially improving their quality of life.

How similar studies have performed: While there have been studies on the use of BTK inhibitors in autoimmune diseases, this specific application in APS with secondary thrombocytopenia is relatively novel.

Eligibility criteria

Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Where this trial is running

Tianjin, Tianjin Municipality and 1 other locations

• Chinese Academy of Medical Science and Blood Disease Hospital — Tianjin, Tianjin Municipality, China (Recruiting)
• Institute of Hematology & Blood Diseases Hospital — Tianjin, Tianjin Municipality, China (Recruiting)

Study contacts

• Principal investigator: Lei Zhang, MD — Chinese Academy of Medical Science and Blood Disease Hospital
• Study coordinator: Yunfei Chen, MD

Conditions Antiphospholipid Syndrome; Thrombocytopenia; Treatment