A phase III clinical trial of PXT3003 in Chinese patients with Charcot-Marie-Tooth disease type 1A
Abstract
Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common inherited peripheral neuropathy and currently lacks disease-modifying therapy. PXT3003, a low-dose oral combination targeting PMP22 overexpression, has shown efficacy in two trials, while one recent confirmatory trial failed to meet its primary efficacy endpoints. In this trial, eligible participants aged 16 to 65 years with genetically confirmed mild-to-moderate CMT1A were randomly assigned to receive oral PXT3003 or placebo twice daily for 15 months. The primary endpoint was the change in the overall neuropathy limitations scale (ONLS) total score from baseline to month 15. At month 15, mean ONLS change from baseline was −0.268 (SD: 0.82) in the PXT3003 group versus 0.013 (SD: 0.65) in the placebo group, with a between-group difference of −0.249 (95% confidence interval [CI]: −0.467 to −0.030; p = 0.0257). Significant improvements were observed in the ONLS leg subscore and ankle-dorsiflexion strength. These findings support PXT3003 as a promising therapeutic option for alleviating limb symptoms in patients with CMT1A.
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Authors: Xiaoxuan Liu, Wanqian Cao, Hongli Li, Chen Sun, Jingzheng Hao, Yuwei Da, Baoyu Yuan, Ning Wang, Shan Wu, Jun Liu, Zuneng Lu, Qi Fang, Zhi‐Ying Wu, Liying Yuan, Jiaoting Jin, Haishan Jiang, Yaling Liu, Mingming Ma, Wei Ling, Jianglong Tu, Xiaoli Yao, Yuan Sun, Xuefan Yu, Yiming Liu, Di Zhong, Yun Xu, Yuming Xu, Runxiu Zhu, Dongsheng Fan, Ruxu Zhang
Institutions: Hebei Medical University, Peking University, Sun Yat-sen University, Central South University, Third Xiangya Hospital, Fujian Medical University, Second Affiliated Hospital of Zhejiang University, Nanfang Hospital, Southern Medical University, Soochow University, Second Hospital of Hebei Medical University, Harbin Medical University, Wuhan University, Renmin Hospital of Wuhan University, Peking University Third Hospital, Guiyang Medical University, Affiliated Hospital of Guizhou Medical University, First Affiliated Hospital of Harbin Medical University, The First Affiliated Hospital, Sun Yat-sen University, Anhui Medical University, Capital Medical University, First Affiliated Hospital of Fujian Medical University, China Medical University, First Affiliated Hospital of Anhui Medical University, Jilin University, First Affiliated Hospital of Soochow University, First Hospital of China Medical University, Nanjing Drum Tower Hospital, Inner Mongolia People's Hospital, Henan Provincial People's Hospital, Ruijin Hospital, National Health and Family Planning Commission, Nanchang University, First Affiliated Hospital of Zhengzhou University, Qilu Hospital of Shandong University, Zhongda Hospital Southeast University, Second Affiliated Hospital of Nanchang University, First Affiliated Hospital of Xi'an Jiaotong University, First Hospital of Jilin University, Tianjin Institute of Pharmaceutical Research (China)