NCT06199089

A Randomized, Double-blind, Placebo-controlled, and Multi-center Clinical Study of CM313 in the Treatment of Immune Thrombocytopenia

A Randomized, Double-blind, Placebo-controlled, and Multi-center Clinical Study to Assess Safety and Efficacy of Anti-Human CD38 Monoclonal Antibody CM313 in the Treatment of Primary Immune Thrombocytopenia

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 45 (actual)
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

To evaluate the efficacy and safety of CM313 in the treatment of immune thrombocytopenia in patients who have failed glucocorticoid therapy.

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 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. CM313, 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 similar CD38 monoclonal antibody drugs, such as daratumumab, in the treatment of autoimmune diseases, including membranous nephropathy, systemic lupus erythematosus (SLE) and ITP, are also being carried out simultaneously. We 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. Therefore, the investigators designed this clinical trial to evaluate the efficacy and safety of CM313 in the treatment of immune thrombocytopenia in patients who have failed glucocorticoid therapy.

Conditions

• Immune Thrombocytopenia
• Treatment

Interventions

Type	Name	Description
DRUG	CM313 Injection	Intravenous CM313 administration This study adopts a randomized, double-blind, placebo-controlled design method. A total of 45 subjects were randomly assigned to experimental group (CM313, n = 30) and placebo comparator group (Placebo, n = 15) at a ratio of 2:1. Subjects assigned to experimental group were treated with CD38 monoclonal antibody (CM313: 16mg/kg/w) for 8 weeks. The first stage is the main research stage (d1-w8), which is the core treatment period. The subjects will receive intravenous infusion of 16mg/kg CM313 once a week for 8 weeks to observe the efficacy and safety during treatment. The second stage (w9-w24) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy of CM313 after treatment.
DRUG	Placebo Injection	Intravenous Placebo administration This study adopts a randomized, double-blind, placebo-controlled design method. A total of 45 subjects were randomly assigned to experimental group (CM313, n = 30) and placebo comparator group (Placebo, n = 15) at a ratio of 2:1. Subjects assigned to placebo comparator group were treated with placebo of CM313 once a week for 8 weeks. The first stage is the main research stage (d1-w8), which is the core treatment period. The subjects will receive intravenous infusion of placebo of CM313 once a week for 8 weeks to observe the efficacy and safety during treatment. The second stage (w9-w24) is the stage of withdrawal from the visit, mainly to observe the safety and continuous efficacy after placebo administration.

Timeline

• Start date: 2024-01-16
• Primary completion: 2024-11-28
• Completion: 2024-12-01
• First posted: 2024-01-10
• Last updated: 2024-12-30

Locations

1 site across 1 country: China