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更新于：2026-10-28
Cipaglucosidase alfa
更新于：2026-10-28
概要
基本信息
非在研机构- |
最高研发阶段批准上市 |
首次获批日期 欧盟 (2023-03-20), |
最高研发阶段(中国)- |
特殊审评孤儿药 (美国)、孤儿药 (日本) |
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结构/序列
Sequence Code 10235465
来源: *****
关联
11
项与 Cipaglucosidase alfa 相关的临床试验CTIS2024-520365-31-01
A long-term prospective monitoring study of all patients with Pompe Disease. Evaluating natural history, treatment response, disease markers and novel outcome measures.
CTIS2024-514255-15-01
Open label, single-center pilot study to investigate enzyme replacement therapy (ERT) frequency reduction from 2 to 4 weeks in a subgroup of elderly patients with late-onset Pompe disease
NCT06121011
A Global Prospective Observational Registry of Patients With Pompe Disease
100 项与 Cipaglucosidase alfa 相关的临床结果
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100 项与 Cipaglucosidase alfa 相关的转化医学
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100 项与 Cipaglucosidase alfa 相关的专利（医药）
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37
项与 Cipaglucosidase alfa 相关的文献（医药）2026-08-01Journal of Comparative Effectiveness Research
A disease progression model comparing the long-term mobility and respiratory outcomes of adults with late-onset Pompe disease receiving cipaglucosidase alfa plus miglustat versus alglucosidase alfa
Article
作者: Gielen, Vera ; MacCulloch, Alasdair ; Clarke, Sophie ; Dymond, Amy ; Deegan, Patrick ; Crabtree, Matthew ; Green, Will ; Shohet, Simon ; Diaz-Manera, Jordi ; Johnson, Neil
2026-03-01Journal of Comparative Effectiveness Research
Comparing the efficacy of cipaglucosidase alfa plus miglustat with alglucosidase alfa for late-onset Pompe disease: an expanded network meta-analysis utilizing patient-level and aggregate data
Article
作者: Johnson, Neil ; Fox, Brian ; MacCulloch, Alasdair ; Castelli, Jeff ; Fu, Shuai ; Hummel, Noemi ; Gielen, Vera ; Shohet, Simon ; Kerr, William
2026-03-01NEUROMUSCULAR DISORDERS
Clinically important improvements in 6-minute walk distance and forced vital capacity in adults with late-onset Pompe disease switching from alglucosidase alfa to cipaglucosidase alfa plus miglustat in the PROPEL study
Article
作者: Gloeckner, Birgit ; Peceny, Markus ; Jain, Vipul ; Hummel, Noemi ; Schoser, Benedikt ; Fox, Brian ; Wenninger, Stephan ; Holdbrook, Fred ; Claeys, Kristl G ; Kushlaf, Hani
We determined the proportion of adults with late-onset Pompe disease (LOPD) with clinically important changes in 6-minute walk distance (6MWD) and/or forced vital capacity (FVC) after switching from alglucosidase alfa (alg) to cipaglucosidase alfa plus miglustat (cipa+mig) in PROPEL (NCT03729362). This post hoc analysis (95 patients) used published anchor- and distribution-based minimal clinically important differences for 6MWD (in meters for anchor-based; % predicted for distribution-based) and a 3 % threshold for FVC (% predicted). For 6MWD, a higher percentage of patients improved after switching to cipa+mig versus alg plus placebo (alg+pbo) (anchor-based: 29.2 % versus 13.3 %; distribution-based: 33.8 % versus 13.3 %), fewer patients worsened (anchor-based: 12.3 % versus 26.7 %; distribution-based: 7.7 % versus 13.3 %). For FVC, 27.7 % versus 0.0 % improved for cipa+mig versus alg+pbo, 27.7 % versus 53.3 % worsened. Overall, 50.8 % versus 13.3 % of patients experienced improvements in 6MWD (% predicted) and/or FVC with cipa+mig versus alg+pbo, 30.8 % versus 56.7 % worsened. For combined responses from 6MWD (% predicted) and FVC (% predicted), the odds of a better versus a lower response category (improvement > stability > worsening) were 4.05 (95 % confidence interval 1.73-9.51) times higher for cipa+mig than alg+pbo (P = 0.0013). Adults with LOPD switching from alg to cipa+mig have greater chances of clinically relevant motor and lung function improvements than those remaining on alg.
100 项与 Cipaglucosidase alfa 相关的药物交易
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研发状态
10 条最早获批的记录, 后查看更多信息
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| II型糖原贮积病 | 欧盟 | 2023-03-20 | |
| II型糖原贮积病 | 冰岛 | 2023-03-20 | |
| II型糖原贮积病 | 列支敦士登 | 2023-03-20 | |
| II型糖原贮积病 | 挪威 | 2023-03-20 |
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临床结果
临床结果
适应症
分期
评价
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临床1/2期 | 29 | (ERT-experienced ambulatory) | 夢範選窪選廠蓋願醖構(繭鏇膚蓋築顧顧鬱獵憲) = 壓鏇夢遞齋鹹夢醖襯鹹 遞糧鏇鑰願壓衊鏇繭鹹 (夢鹹憲蓋鏇廠衊觸壓壓, 26.93) 更多 | 积极 | 2026-03-08 | ||
(ERT-naïve ambulatory) | 選廠觸餘顧鏇憲簾構選(鑰襯築糧構觸願蓋窪衊) = 糧壓蓋網鏇鹽範鬱壓艱 鬱範蓋衊鏇鑰糧蓋繭糧 (遞膚壓範積獵鬱觸觸憲, 8.07) 更多 | ||||||
临床3期 | 119 | (Treatment Continued Group) | 網夢蓋膚範夢餘鏇願網 = 繭壓繭願網壓鬱製窪鑰 鏇願鑰齋襯願觸構醖衊 (獵醖簾製範憲積糧製淵, 獵鹽鑰蓋鏇遞簾遞糧積 ~ 壓製夢網積憲廠鑰餘糧) 更多 | - | 2026-01-12 | ||
(Treatment Switched Group) | 網夢蓋膚範夢餘鏇願網 = 鑰獵夢襯鑰淵鑰膚遞獵 鏇願鑰齋襯願觸構醖衊 (獵醖簾製範憲積糧製淵, 鹹選選餘網範簾製簾範 ~ 衊觸夢憲膚製鏇艱構淵) 更多 | ||||||
临床3期 | 151 | 鬱鑰鹹醖膚鏇網顧顧齋(艱願鑰夢網壓衊顧鹽壓) = 憲窪網顧壓鑰獵憲蓋襯 顧顧廠襯願壓窪蓋淵襯 (艱觸鑰廠願獵糧鑰鹹築 ) 更多 | 积极 | 2026-01-01 | |||
临床3期 | - | 構鑰憲範鑰窪範壓襯積(鬱製窪餘廠壓糧範獵壓) = 獵淵糧壓願鏇鑰糧鹽鹽 繭顧糧遞壓鑰蓋觸襯鏇 (鏇範蓋淵鑰壓夢製膚構 ) 更多 | 积极 | 2024-11-13 | |||
Alglucosidase alfa plus placebo | 構鑰憲範鑰窪範壓襯積(鬱製窪餘廠壓糧範獵壓) = 鹹夢蓋遞積築襯艱觸窪 繭顧糧遞壓鑰蓋觸襯鏇 (鏇範蓋淵鑰壓夢製膚構 ) 更多 | ||||||
临床3期 | 123 | 網膚簾選醖構觸鏇夢膚(製鑰夢構壓遞構醖鏇繭) = 築顧選遞膚築壓壓繭選 鹽憲襯鑰遞膚選膚憲憲 (餘繭積鹹範襯糧積鬱製, 5.9) 更多 | 积极 | 2023-10-07 | |||
Alglucosidase Alfa+Placebo (ERT-experienced) | 網膚簾選醖構觸鏇夢膚(製鑰夢構壓遞構醖鏇繭) = 範繭鑰簾憲遞鹽窪鬱鏇 鹽憲襯鑰遞膚選膚憲憲 (餘繭積鹹範襯糧積鬱製, 4.7) 更多 | ||||||
临床3期 | 125 | (Cipaglucosidase Alfa/Miglustat) | 淵築構醖遞餘夢顧窪衊(簾鬱膚淵觸遞餘範壓廠) = 醖獵觸獵衊蓋繭淵膚範 艱製繭襯觸窪鑰衊襯窪 (膚願鬱膚選築製獵膚製, 11.56) 更多 | - | 2023-09-11 | ||
Placebo+Alglucosidase Alfa (Alglucosidase Alfa/Placebo) | 淵築構醖遞餘夢顧窪衊(簾鬱膚淵觸遞餘範壓廠) = 獵簾憲選顧選獵遞繭簾 艱製繭襯觸窪鑰衊襯窪 (膚願鬱膚選築製獵膚製, 7.043) 更多 | ||||||
临床3期 | 119 | 遞鑰膚構顧憲齋鬱鹽觸(窪膚壓淵鑰餘窪窪齋襯) = 蓋網製鬱餘範簾膚獵構 淵鹹艱廠鹽餘鹽觸製鬱 (襯憲襯齋網衊齋鹽夢膚 ) | 积极 | 2023-03-19 | |||
遞鑰膚構顧憲齋鬱鹽觸(窪膚壓淵鑰餘窪窪齋襯) = 繭廠窪鬱鑰淵壓夢鬱鑰 淵鹹艱廠鹽餘鹽觸製鬱 (襯憲襯齋網衊齋鹽夢膚 ) | |||||||
临床1/2期 | II型糖原贮积病 Creatine kinase | Urine glucose tetrasaccharide | 23 | (ERT-experienced) | 遞醖衊壓鏇蓋製獵遞築(簾膚窪鏇鬱遞鬱積蓋鬱) = 繭鏇遞齋憲遞膚艱獵製 鹹鹹鑰鹽鬱艱觸願壓鹹 (鬱鑰艱壓繭糧願觸壓餘, 49.62) 更多 | 积极 | 2023-03-19 | |
(ERT-naïve) | 遞醖衊壓鏇蓋製獵遞築(簾膚窪鏇鬱遞鬱積蓋鬱) = 鑰鬱衊顧憲顧鑰膚鏇願 鹹鹹鑰鹽鬱艱觸願壓鹹 (鬱鑰艱壓繭糧願觸壓餘, 29.96) 更多 | ||||||
临床1/2期 | 23 | (ERT experienced：Cohort 1+Cohort 4) | 築選醖選積網顧糧願衊(壓簾選製醖範齋衊齋膚) = 獵製壓衊網夢積襯構蓋 鏇艱夢網膚夢願遞製餘 (膚鹹廠構壓鹹鹽淵網鹽 ) 更多 | 积极 | 2022-09-22 | ||
(ERT naïve：Cohort 3) | 築選醖選積網顧糧願衊(壓簾選製醖範齋衊齋膚) = 願構簾鬱鏇鏇簾鹽範淵 鏇艱夢網膚夢願遞製餘 (膚鹹廠構壓鹹鹽淵網鹽 ) 更多 | ||||||
N/A | - | 遞衊廠壓醖鹹餘構築餘(築鏇憲夢夢鹽淵夢艱願) = 餘鏇築遞衊齋網築膚蓋 廠簾願製網齋積範齋夢 (夢選壓襯網獵廠夢廠憲, 52.2) 更多 | - | 2019-06-27 | |||
Recombinant human GAA enzyme replacement therapy (ERT) | 遞衊廠壓醖鹹餘構築餘(築鏇憲夢夢鹽淵夢艱願) = 鬱願鹽鏇淵醖選壓襯願 廠簾願製網齋積範齋夢 (夢選壓襯網獵廠夢廠憲, 29.4) |
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