Evidence map›Paper›PMID 40760696›Full record

Trial reportOrphanet journal of rare diseases2025

A phase 4, open-label, multicenter study of the safety and efficacy of agalsidase beta in Chinese patients with Fabry disease.

Hong Ren, Wei Zhang, Yan Ouyang, Junhong Guo, Hong Xu, Jie Ma, Xiaoping Luo, Xiaoxia Pan, Yun Yuan, Wei Zhang and 5 more

Registry-linked trialAbstract readMulticenter StudyClinical Trial, Phase IV
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Orphanet journal of rare diseases, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05054387 (A Phase 4, Open Label, Safety and Efficacy Study of Fabrazyme®), which is not on this map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing 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.

NCT05054387 phase4completednot on this map

A Phase 4, Open Label, Safety and Efficacy Study of Fabrazyme® (Agalsidase Beta) as Enzyme Replacement Therapy in Chinese Participants With Fabry Disease

TypeinterventionalSponsorGenzyme, a Sanofi CompanyRan2021 to 2023Enrolled22ConditionsFabry DiseaseArmsAgalsidase beta
3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Article
  2. 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

15 authors.

Hong Ren *Department of Nephrology, Institute of Nephrology, Ruijin Hospital, The Medical School of Shanghai Jiao Tong University, No. 197 Ruijin Er Road, Shanghai, China.
Wei Zhang *Department of Neurology, Peking University First Hospital, No. 8 Xishiku Street, Beijing, China.
Yan Ouyang *Department of Nephrology, Institute of Nephrology, Ruijin Hospital, The Medical School of Shanghai Jiao Tong University, No. 197 Ruijin Er Road, Shanghai, China.
Junhong GuoDepartment of Neurology, First Hospital of Shanxi Medical University, No. 85 South Jiefang Road, Taiyuan, Shanxi, China.
Hong XuDepartment of Nephrology, Children's Hospital of Fudan University, No. 399 Wanyuan Road, Shanghai, China.
Jie MaDepartment of Nephrology, Peking Union Medical College Hospital, No.1 Shuaifuyuan Wangfujing Dongcheng District, Beijing, China.
Xiaoping LuoDepartment of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, No.1095 Jiefang Avenue, Wuhan, Hubei, China.
Xiaoxia PanDepartment of Nephrology, Institute of Nephrology, Ruijin Hospital, The Medical School of Shanghai Jiao Tong University, No. 197 Ruijin Er Road, Shanghai, China.
Yun YuanDepartment of Neurology, Peking University First Hospital, No. 8 Xishiku Street, Beijing, China.
Wei ZhangDepartment of Neurology, First Hospital of Shanxi Medical University, No. 85 South Jiefang Road, Taiyuan, Shanxi, China.
Qian ShenDepartment of Nephrology, Children's Hospital of Fudan University, No. 399 Wanyuan Road, Shanghai, China.
Bin LiMedical Department of Rare Disease & Rare Blood Disorders, Sanofi, No. 1228 Middle Yan'an Road, Shanghai, China.
Qiqi FengMedical Department of Rare Disease & Rare Blood Disorders, Sanofi, No. 1228 Middle Yan'an Road, Shanghai, China.
Shi LiuMedical Department of Rare Disease & Rare Blood Disorders, Sanofi, No. 1228 Middle Yan'an Road, Shanghai, China.
Nan ChenDepartment of Nephrology, Institute of Nephrology, Ruijin Hospital, The Medical School of Shanghai Jiao Tong University, No. 197 Ruijin Er Road, Shanghai, China. cnrj100@126.com.ORCID http://orcid.org/0009-0008-7388-6339

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundThis is the first phase 4 study evaluating safety and efficacy of enzyme replacement therapy (ERT) in Chinese patients with Fabry disease, and exploring the impact of COVID-19 infection on the prognosis of Fabry disease under ERT. METHODS AND

resultsEligible patients received an infusion of agalsidase beta (1.0 mg/kg/2w) for up to 48 weeks. The primary endpoint was the safety of agalsidase beta. The endpoints of efficacy included changes in plasma globotriaosylceramide (GL-3), globotriaosylsphingosine (Lyso-GL-3), symptoms and estimated glomerular filtration rate (eGFR) from baseline to week 48. A post-hoc subgroup analysis was conducted by age group (< 30 years and ≥ 30 years) and in patients with or without COVID-19 infection. All 22 patients completed the study and 14 of them were infected by COVID-19. Treatment-related adverse events (AEs) and infusion-associated reactions (IARs) were reported in 8 participants (36.4%). Mean plasma GL-3 (-34.6%) and Lyso-GL-3 (-60.3%) levels decreased from baseline to week 48. Thirteen participants (59.1%) experienced improved specific symptoms at week 48. There were no meaningful changes in eGFR during the study, and the overall population showed an annual eGFR slope of 0.43 mL/min/1.73 m

conclusionsThis study demonstrates that agalsidase beta is safe and effective in Chinese patients with Fabry disease, and suggestes that COVID-19 infection may potentially impact the renal prognosis for Fabry disease.

trial registrationClinicalTrials.gov, NCT05054387. Registered 09 September 2021, https://clinicaltrials.gov/study/NCT05054387.

Indexed as

alpha-GalactosidaseEnzyme Replacement TherapyFabry DiseaseAdolescentAdultAgedChinaCOVID-19East Asian PeopleFemaleGlomerular Filtration RateGlycolipidsHumansIsoenzymesMaleMiddle Agedagalsidase betaalpha-Galactosidaseglobotriaosylceramideglobotriaosyl lysosphingolipidGlycolipidsIsoenzymesSphingolipidsTrihexosylceramidesAgalsidase betaCOVID-19Fabry diseaseInfusion-associated reactionsKidney function

Identifiers

PMID40760696
PMCPMC12323282

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Registered trials

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.