Efficacy and safety of idursulfase beta in the treatment of mucopolysaccharidosis II: a phase 3, two-part study compared to a historical placebo cohort.

IF 6.6 1区医学 Q1 GENETICS & HEREDITY

Genetics in Medicine Pub Date : 2025-05-21 DOI:10.1016/j.gim.2025.101460

Young Bae Sohn, Aram Yang, Min-Sun Kim, Jinsup Kim, Jae Seong Kim, Youngsin Oh, Dong-Kyu Jin

{"title":"Efficacy and safety of idursulfase beta in the treatment of mucopolysaccharidosis II: a phase 3, two-part study compared to a historical placebo cohort.","authors":"Young Bae Sohn, Aram Yang, Min-Sun Kim, Jinsup Kim, Jae Seong Kim, Youngsin Oh, Dong-Kyu Jin","doi":"10.1016/j.gim.2025.101460","DOIUrl":null,"url":null,"abstract":"Purpose: To investigate the efficacy and safety of idursulfase beta (0.5 mg/kg weekly) in the treatment of mucopolysaccharidosis II (MPS II), compared with a historical placebo from a previous idursulfase trial (TKT024).Methods: The study comprised two sequential parts. In Part 1, a randomized, double-blind study, idursulfase or idursulfase beta were given for 52 weeks. In the open, single cohort Part 2 study, additional participants received idursulfase beta for 52 weeks. Data from the idursulfase beta groups from Parts 1 & 2 were pooled for comparisons with the historical placebo group (n=32). The primary endpoint was a change in the 6-minute walk test (6-MWT) at week 53.Results: Participants in the idursulfase beta groups (n=24) were male Asians (mean age, 12.0 years). Idursulfase beta was superior to placebo in 6-MWT improvement (62.2 m vs. 7.3 m, p < 0.0001). Decrease in urine glycosaminoglycan excretion (-71.13% vs. 21.39%, p < 0.0001) and reduction in the liver (-26.67% vs. -0.80%, p < 0.0001) and spleen volumes (-26.46% vs. 7.2%, p < 0.0001) were significant. The safety profile of idursulfase beta was satisfactory.Conclusion: Idursulfase beta is a safe and effective treatment option in MPS II, addressing crucial somatic ailments presented by patients.","PeriodicalId":12717,"journal":{"name":"Genetics in Medicine","volume":" ","pages":"101460"},"PeriodicalIF":6.6000,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Genetics in Medicine","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1016/j.gim.2025.101460","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"GENETICS & HEREDITY","Score":null,"Total":0}

引用次数: 0

Abstract

Purpose: To investigate the efficacy and safety of idursulfase beta (0.5 mg/kg weekly) in the treatment of mucopolysaccharidosis II (MPS II), compared with a historical placebo from a previous idursulfase trial (TKT024).

Methods: The study comprised two sequential parts. In Part 1, a randomized, double-blind study, idursulfase or idursulfase beta were given for 52 weeks. In the open, single cohort Part 2 study, additional participants received idursulfase beta for 52 weeks. Data from the idursulfase beta groups from Parts 1 & 2 were pooled for comparisons with the historical placebo group (n=32). The primary endpoint was a change in the 6-minute walk test (6-MWT) at week 53.

Results: Participants in the idursulfase beta groups (n=24) were male Asians (mean age, 12.0 years). Idursulfase beta was superior to placebo in 6-MWT improvement (62.2 m vs. 7.3 m, p < 0.0001). Decrease in urine glycosaminoglycan excretion (-71.13% vs. 21.39%, p < 0.0001) and reduction in the liver (-26.67% vs. -0.80%, p < 0.0001) and spleen volumes (-26.46% vs. 7.2%, p < 0.0001) were significant. The safety profile of idursulfase beta was satisfactory.

Conclusion: Idursulfase beta is a safe and effective treatment option in MPS II, addressing crucial somatic ailments presented by patients.

查看原文本刊更多论文

idursulase β治疗粘多糖病II的疗效和安全性：一项与历史安慰剂队列比较的3期两部分研究

目的：研究idursulase β（每周0.5 mg/kg）治疗粘多糖病II （MPS II）的有效性和安全性，并与先前idursulase试验（TKT024）的历史安慰剂进行比较。方法：本研究分为两部分。在第一部分的随机双盲研究中，idursulfase或idursulfase β给药52周。在开放的单队列第2部分研究中，额外的参与者接受了52周的idursulfase beta治疗。将第1部分和第2部分的idursulase β组数据与历史安慰剂组（n=32）进行汇总比较。主要终点是53周时6分钟步行试验（6-MWT）的变化。结果：idursulase β组参与者（n=24）为亚洲男性（平均年龄12.0岁）。idursulase β在6-MWT改善方面优于安慰剂（62.2 m对7.3 m, p < 0.0001）。尿糖胺聚糖排泄量（-71.13% vs. 21.39%, p < 0.0001）、肝脏（-26.67% vs. -0.80%, p < 0.0001）和脾脏体积（-26.46% vs. 7.2%, p < 0.0001）均显著减少。idursulase β的安全性令人满意。结论：idursulase β是一种安全有效的治疗MPS II的选择，可解决患者出现的关键躯体疾病。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Genetics in Medicine 医学-遗传学

CiteScore

15.20

自引率

6.80%

发文量

857

审稿时长

1.3 weeks

期刊介绍： Genetics in Medicine (GIM) is the official journal of the American College of Medical Genetics and Genomics. The journal''s mission is to enhance the knowledge, understanding, and practice of medical genetics and genomics through publications in clinical and laboratory genetics and genomics, including ethical, legal, and social issues as well as public health. GIM encourages research that combats racism, includes diverse populations and is written by authors from diverse and underrepresented backgrounds.