Evidence map›Paper›PMID 42699772›Full record

ArticleiScience2026

A phase III clinical trial of PXT3003 in Chinese patients with Charcot-Marie-Tooth disease type 1A.

Xiaoxuan Liu, Wanqian Cao, Hongli Li, Chen Sun, Jingzheng Hao, Yuwei Da, Baoyu Yuan, Ning Wang, Shan Wu, Jun Liu and 20 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in iScience, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

30 authors.

Xiaoxuan LiuDepartment of Neurology, Peking University Third Hospital, Beijing 100191, China.
Wanqian CaoDepartment of Neurology, The Third Xiangya Hospital of Central South University, Changsha 410013, China.
Hongli LiCenter of Clinical Research, Genenet Pharmaceutical Co., Ltd., Tianjin, China.
Chen SunCenter of Clinical Research, Genenet Pharmaceutical Co., Ltd., Tianjin, China.
Jingzheng HaoCenter of Clinical Research, Genenet Pharmaceutical Co., Ltd., Tianjin, China.
Yuwei DaNeurology Department, Xuanwu Hospital of Capital Medical University, Beijing, China.
Baoyu YuanDepartment of Neurology, Zhongda Hospital, Southeast University, Nanjing, China.
Ning WangDepartment of Neurology, The First Affiliated Hospital of Fujian Medical University, Fuzhou, China.
Shan WuDepartment of Neurology, Affiliated Hospital of Guizhou Medical University, Guiyang, China.
Jun LiuDepartment of Neurology, Ruijin Hospital, Shanghai Jiaotong University School of Medicine, Shanghai, China.
Zuneng LuDepartment of Neurology, Renmin Hospital of Wuhan University, Wuhan, China.
Qi FangDepartment of Neurology, The First Affiliated Hospital of Soochow University, Suzhou, China.
Zhi-Ying WuDepartment of Neurology, The Second Affiliated Hospital, Zhejiang University School of Medicine, Hangzhou, China.
Liying YuanDepartment of Neurology, The First Hospital of China Medical University, Shenyang, China.
Jiaoting JinDepartment of Neurology, The First Affiliated Hospital of Xi'an Jiaotong University, Xi'an, China.
Haishan JiangDepartment of Neurology, Nanfang Hospital, Southern Medical University, Guangzhou, China.
Yaling LiuDepartment of Neurology, The Second Hospital of Hebei Medical University, Shijiazhuang, China.
Mingming MaDepartment of Neurology, Henan Provincial People's Hospital, Zhengzhou, China.
Ling WeiDepartment of Neurology, The First Affiliated Hospital of Anhui Medical University, Hefei, China.
Jianglong TuDepartment of Neurology, The Second Affiliated Hospital of Nanchang University, Nanchang, China.
Xiaoli YaoDepartment of Neurology, The First Affiliated Hospital of Sun Yat-sen University, Guangzhou, China.
Yuan SunDepartment of Neurology, Qilu Hospital, Cheeloo College of Medicine, Shandong University, Qingdao, China.
Xuefan YuDepartment of Neurology, First Hospital of Jilin University, Changchun, China.
Yiming LiuDepartment of Neurology, Qilu Hospital of Shandong University, Jinan, China.
Di ZhongDepartment of Neurology, The First Affiliated Hospital of Harbin Medical University, Harbin, China.
Yun XuDepartment of Neurology, Nanjing Drum Tower Hospital, Nanjing, China.
Yuming XuDepartment of Neurology, The First Affiliated Hospital of Zhengzhou University, Zhengzhou, China.
Runxiu ZhuDepartment of Neurology, Inner Mongolia People's Hospital, Hohhot, China.
Dongsheng FanDepartment of Neurology, Peking University Third Hospital, Beijing 100191, China.
Ruxu ZhangDepartment of Neurology, The Third Xiangya Hospital of Central South University, Changsha 410013, China.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

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;

Indexed as

Charcot-Marie-Tooth disease type 1Afunctional disabilityperipheral neuropathyphase 3 clinical trialPMP22PXT3003

Identifiers

PMID42699772
PMCPMC13544322

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.