NCT05214391

A Prospective, One-arm and Open Clinical Study of Zanubrutinib in the Treatment of Immune Thrombocytopenia

Safety and Efficacy of Zanubrutinib in the Treatment of Immune Thrombocytopenia

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 30 (estimated)
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

To evaluate the safety and efficacy of zanubrutinib in the treatment of immune thrombocytopenia in 30 patients.

Detailed description

Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second-line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.). ITP is one of the most common hemorrhagic diseases. At present, the treatment response of ITP is not good, and a considerable number of patients need drug maintenance treatment, which seriously affects the quality of life of patients and increases the economic burden of patients. Therefore, there is still a lack of effective treatment for adult ITP, especially for refractory ITP patients, which is one of the problems that have attracted more attention and need to be solved urgently. BTK inhibitors can affect autoimmune diseases (AID) involving B cells and non-B cells through B cell receptor, Fc receptor and RANK receptor signals, such as systemic lupus erythematosus (SLE), multiple sclerosis (MS) and rheumatoid arthritis (RA) .Therefore, small molecule BTK inhibitors can treat autoimmune diseases by targeting B cells. At present, clinical studies on the treatment of immune thrombocytopenia with BTK inhibitor (BRN1008) have been carried out abroad. The preliminary results show that 50% of patients in the treatment ≥ 12 weeks and the initial dose is 400 mg BID group have reached the primary endpoint and maintained platelet response. Zebutinib is a new selective Bruton tyrosine kinase (BTK) inhibitor developed by Baekje Shenzhou Company of China. In November 2019, it was approved by the US Food and Drug Administration to treat adult mantle cell lymphoma (MCL) patients who had received at least one treatment before. Compared with the first generation BTK inhibitors (ibutinib and acalabrutinib), zebutinib has stronger targeting and fewer adverse reactions. Therefore, the investigators designed this clinical trial to provide new treatment options for clinical treatment of ITP.

Conditions

• Immune Thrombocytopenia
• Treatment

Interventions

Type	Name	Description
DRUG	Zanubrutinib	The initial dose is 80mg/day. If the treatment is ineffective after 4 weeks, and under the condition of good safety, the investigator 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.

Timeline

• Start date: 2022-02-15
• Primary completion: 2025-12-31
• Completion: 2025-12-31
• First posted: 2022-01-28
• Last updated: 2025-02-24

Locations

1 site across 1 country: China

Regulatory

• FDA-regulated drug study