{
  "abstract": "Abstract Objective To assess the efficacy and safety of CM313, an anti-CD38 monoclonal antibody, in adults with persistent or chronic primary immune thrombocytopenia.Design Multicentre, randomised, placebo controlled, phase 2 trial.Setting Five hospitals in China, 16 January to 11 June 2024.Participants 45 patients aged ≥18 years with persistent or chronic immune thrombocytopenia who failed to respond to, or relapsed after, glucocorticoid treatment but had previously responded to standard first line treatment.Interventions Patients were randomised to receive intravenous CM313 (16 mg/kg) or placebo weekly for eight weeks.Main outcome measures The primary outcome was overall response rate (at least two consecutive platelet counts ≥30×10 9/L, a minimum doubling from baseline, and no bleeding) at week 8. A secondary outcome was time to the first two consecutive platelet counts ≥50×109/L.Results Of 56 patients screened, 45 were randomised to receive CM313 (n=30) or placebo (n=15). At week 8, the overall response rate was higher in the CM313 group (83%; 25/30) compared with placebo group (20%; 3/15): difference 63.3% (95% confidence interval 33.7% to 81.3%; P<0.001). The median time to a platelet count of ≥50×10 9/L was one week in the CM313 group but was not reached in the placebo group (P<0.001). The median cumulative response duration for platelet counts ≥50×109/L was 18 weeks in the CM313 group versus three weeks in the placebo group (P=0.004). Treatment emergent adverse events occurred in 87% (26/30) of patients in the CM313 group and 80% (12/15) in the placebo group, with infusion related reactions and petechiae being the most common.Conclusion CM313 showed a favourable safety profile and encouraging efficacy in adults with persistent or chronic primary immune thrombocytopenia, characterised by rapid increase in platelet count, sustained platelet responses, and manageable adverse events.Trial registration ClinicalTrials.gov NCT06199089.",
  "authors": [
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Yunfei Chen"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Yanmei Xu"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Jiawen Dai"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Ting Sun"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Huiyuan Li"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Zhengjie Hua"
    },
    {
      "affiliations": [
        "Department of Hematology, Second Affiliated Hospital of Kunming Medical University, Kunming, China"
      ],
      "name": "Zeping Zhou"
    },
    {
      "affiliations": [
        "Affiliated Cancer Hospital of Zhengzhou University, Henan Cancer Hospital, Zhengzhou, China"
      ],
      "name": "Hu Zhou"
    },
    {
      "affiliations": [
        "North China University of Science and Technology Affiliated Hospital, Tangshan, China"
      ],
      "name": "Zhenyu Yan"
    },
    {
      "affiliations": [
        "Department of Hematology, Oncology Center, Tianjin People’s Hospital, Tianjin, China"
      ],
      "name": "Xingli Zhao"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Feng Xue"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Wei Liu"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Xiaofan Liu"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Rongfeng Fu"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Wentian Wang"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Ying Chi"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Huan Dong"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Mankai Ju"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Xinyue Dai"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Wenjing Gu"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Xiaolei Pei"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Renchi Yang"
    },
    {
      "affiliations": [
        "State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Tianjin Key Laboratory of Gene Therapy for Blood Diseases, CAMS Key Laboratory of Gene Therapy for Blood Diseases, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, Tianjin, China",
        "Tianjin Institutes of Health Science, Tianjin 301600, China"
      ],
      "name": "Lei Zhang"
    }
  ],
  "title": "Anti-CD38 monoclonal antibody CM313 for primary immune thrombocytopenia: multicentre, randomised, placebo controlled, phase 2 trial",
  "uid": "93d98dc1-de10-5fe7-ae14-8abcd7df76b9"
}