NCT07063199

Anti-CD38 Antibody Treating Elderly Patients With Primary Immune Thrombocytopenia (ITP)

A Single-arm, Open-label Clinical Study Evaluating the Safety and Efficacy of Daratumumab in Elderly Patients With Primary 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: 60 Years
• Healthy volunteers: Not accepted

Summary

To evaluate the safety and efficacy of Anti-CD38 Antibody in the treatment of elderly patients with primary immune thrombocytopenia in patients who have failed multiple treatment.

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 recurrent and refractory ITP patients, which is one of the problems that have attracted more attention and need to be solved urgently.The elderly are at a higher risk of developing immune thrombocytopenia, and they often have cardiovascular risk factors. They usually cannot tolerate treatment regimens such as hormones and immunosuppressants. The main pathogenesis of ITP is the loss of platelet autoantigen immune tolerance, which leads to abnormal activation of humoral and cellular immunity. It is characterized by antibody mediated platelet destruction and insufficient platelet production by megakaryocytes. The residual long-term autoreactive plasma cells may be a source of therapeutic resistance to autoimmune cytopenia. Antiplatelet specific plasma cells have been detected in the spleen of patients with rituximab refractory ITP. Therefore, the strategy of simply eliminating B cells may not work, because LLPC will continue to produce pathogenic antibodies. However, targeting LLPC becomes a new strategy to treat autoimmune diseases. Daratumumab, a kind of anti-CD38 antibody, is a new type of monoclonal antibody targeting CD38. It targets plasma cells and has carried out some clinical studies in multiple myeloma, with good therapeutic effects. In addition, the clinical trials of daratumumab in the treatment of autoimmune diseases, including membranous nephropathy, systemic lupus erythematosus (SLE) and ITP, are also being carried out simultaneously. The investigators assume that autologous reaction LLPC may be the cause of treatment failure in some ITP patients. Therefore, the use of CD38 monoclonal antibody to clear long-term surviving plasma cells in ITP patients may be a new strategy for treating ITP patients.Our hospital has conducted and completed a clinical trial of daratumumab for the treatment of adult ITP. The dosage was 16mg/kg/w for a total of 8 weeks. The results showed that for refractory patients who had previously received multiple lines of treatment with poor efficacy, daratumumab could rapidly increase platelet counts. The median onset time was about 1 week. At 24 weeks of follow-up, the effective rate was approximately 60%, and no serious adverse events occurred. The efficacy and safety were confirmed, but the number of elderly patients included was relatively small. Therefore, the investigators designed this clinical trial to evaluate the safety and efficacy of daratumumab in the treatment of elderly patients with immune thrombocytopenia in patients who failed multiple treatment.

Conditions

• Immune Thrombocytopenia
• Treatment

Interventions

Type	Name	Description
DRUG	Daratumumab Injection	This study adopts a prospective, single arm, open design method. Thirty subjects were enrolled in the study and were treated with CD38 monoclonal antibody (Daratumumab 16mg/kg/w) for 4 weeks. The first stage is the main research stage (d1-w4), which is the core treatment period. The subjects will receive intravenous infusion of 16mg/kg Daratumumab once a week for 4 weeks to observe the safety and efficacy during treatment. The second stage (w5-w24) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy of Daratumumab after treatment.

Timeline

• Start date: 2025-08-01
• Primary completion: 2026-08-01
• Completion: 2026-08-01
• First posted: 2025-07-14
• Last updated: 2025-07-14

Locations

1 site across 1 country: China