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Efficacy and safety of JMT103 in patients with unresectable or surgically-challenging giant cell tumor of bone: a multicenter, phase Ib/II study

Hairong Xu, Yong Zhou, Li Liang, Jingnan Shen, Wangjun Yan, Jin Wang, Jianmin Li, Xiaojing Zhang, Gang Huang, Wenzhi Bi, Zheng Guo, Yanbin Xiao, Jianhua Lin, Weitao Yao, Zhichao Tong, Wenxian Zhou, Guochuan Zhang, Zhaoming Ye, Dong Wang, Jilong Yang, Zhengfu Fan, Caigang Liu, Guofan Qu, Qing Zhang, Feng Wei, Weifeng Liu, Chongqi Tu, Hong Li, Jing Yuan and Xiaohui Niu ()
Additional contact information
Hairong Xu: Capital Medical University
Yong Zhou: West China Hospital, Sichuan University
Li Liang: Peking University Third Hospital
Jingnan Shen: The First Affiliated Hospital of Sun Yat-Sen University
Wangjun Yan: Fudan University Shanghai Cancer Center
Jin Wang: Sun Yat-Sen University Cancer Center
Jianmin Li: Qilu Hospital of Shandong University
Xiaojing Zhang: Liaoning Cancer Hospital and Institute
Gang Huang: Hunan Cancer Hospital
Wenzhi Bi: The Fourth Medical Center, Chinese PLA General Hospital
Zheng Guo: The Second Affiliated Hospital of Air Force Medical University
Yanbin Xiao: Yunnan Cancer Hospital
Jianhua Lin: The First Affiliated Hospital of Fujian Medical University
Weitao Yao: The Affiliated Cancer Hospital of Zhengzhou University, Henan Cancer Hospital
Zhichao Tong: Xi’an Jiaotong University
Wenxian Zhou: Guangxi Medical University Cancer Hospital
Guochuan Zhang: The Third Hospital of Hebei Medical University
Zhaoming Ye: The Second Affiliated Hospital of Zhejiang University School of Medicine
Dong Wang: Affiliated Cancer Hospital of Guizhou Medical University
Jilong Yang: Tianjin Medical University Cancer Institute & Hospital
Zhengfu Fan: Peking University Cancer Hospital
Caigang Liu: Shengjing Hospital of China Medical University
Guofan Qu: Harbin Medical University Cancer Hospital
Qing Zhang: Capital Medical University
Feng Wei: Peking University Third Hospital
Weifeng Liu: Capital Medical University
Chongqi Tu: West China Hospital, Sichuan University
Hong Li: CSPC Pharmaceutical Group Limited
Jing Yuan: CSPC Pharmaceutical Group Limited
Xiaohui Niu: Capital Medical University

Nature Communications, 2024, vol. 15, issue 1, 1-9

Abstract: Abstract This was a multicenter, single-arm, open-label, phase Ib/II study (NCT04255576), aimed to evaluate the efficacy and safety of JMT103 in patients with unresectable or surgically-challenging giant cell tumor of bone (GCTB). JMT103 (2 mg/kg) was administered subcutaneously every four weeks, with loading doses on days 8 and 15. The primary endpoint was the objective tumor response rate (OTR) based on best response, defined as the proportion of patients who achieved elimination of at least 90% of the giant cells or radiologic complete or partial response per the modified Inverse Choi density/size (mICDS) or modified European Organization for Research and Treatment of Cancer (mEORTC) within 12 weeks. Secondary endpoints included objective response rate (ORR) per mICDS and mEORTC, and safety. A total of 139 patients were enrolled, and 135 were analyzed for efficacy. OTR, determined by the independent review committee (IRC) was 93.3% (95% CI 87.7-96.9). Treatment-related adverse events occurred in 90 (64.7%) patients, with hypophosphatemia and hypocalcemia being the most common. No serious treatment-related adverse events were observed. Thus, JMT103 demonstrates potential as a therapeutic option for GCTB.

Date: 2024
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DOI: 10.1038/s41467-024-53686-4

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