دورية أكاديمية
Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease:an open-label phase I/II study (ATB200-02)
| العنوان: | Long-term safety and efficacy of cipaglucosidase alfa plus miglustat in individuals living with Pompe disease:an open-label phase I/II study (ATB200-02) |
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| المؤلفون: | Byrne, Barry J., Schoser, Benedikt, Kishnani, Priya S., Bratkovic, Drago, Clemens, Paula R., Goker-Alpan, Ozlem, Ming, Xue, Roberts, Mark, Vorgerd, Matthias, Sivakumar, Kumaraswamy, van der Ploeg, Ans T., Goldman, Mitchell, Wright, Jacquelyn, Holdbrook, Fred, Jain, Vipul, Benjamin, Elfrida R., Johnson, Franklin, Das, Sheela Sitaraman, Wasfi, Yasmine, Mozaffar, Tahseen |
| المصدر: | Byrne , B J , Schoser , B , Kishnani , P S , Bratkovic , D , Clemens , P R , Goker-Alpan , O , Ming , X , Roberts , M , Vorgerd , M , Sivakumar , K , van der Ploeg , A T , Goldman , M , Wright , J , Holdbrook , F , Jain , V , Benjamin , E R , Johnson , F , Das , S S , Wasfi , Y & Mozaffar , T 2023 , ' Long-term safety and efficacy of cipaglucosidase alfa plus miglustat .... |
| سنة النشر: | 2023 |
| الوصف: | Cipaglucosidase alfa plus miglustat (cipa + mig) is a novel, two-component therapy for Pompe disease. We report data from the Phase I/II ATB200-02 study for up to 48 months of treatment. Four adult cohorts, including one non-ambulatory ERT-experienced (n = 6) and three ambulatory cohorts, (two enzyme replacement therapy [ERT]-experienced cohorts [2–6 years (n = 11) and ≥ 7 years (n = 6)]), one ERT-naïve cohort (n = 6), received 20 mg/kg intravenous-infused cipa plus 260 mg oral mig biweekly. Change from baseline (CFBL) for multiple efficacy endpoints at 12, 24, 36, and 48 months, pharmacodynamics, pharmacokinetics, safety, and immunogenicity data were assessed. Six-minute walking distance (% predicted) improved at 12, 24, 36, and 48 months: pooled ambulatory ERT-experienced cohorts, mean(± standard deviation [SD]) CFBL: 6.1(± 7.84), n = 16; 5.4(± 10.56), n = 13; 3.4(± 14.66), n = 12; 5.9(± 17.36), n = 9, respectively; ERT-naïve cohort: 10.7(± 3.93), n = 6; 11.0(± 5.06), n = 6; 9.0(± 7.98), n = 5; 11.7(± 7.69), n = 4, respectively. Percent predicted forced vital capacity was generally stable in ERT-experienced cohorts, mean(± SD) CFBL − 1.2(± 5.95), n = 16; 1.0(± 7.96), n = 13; − 0.3(± 6.68), n = 10; 1.0(± 6.42), n = 6, respectively, and improved in the ERT-naïve cohort: 3.2(± 8.42), n = 6; 4.7(± 5.09), n = 6; 6.2(± 3.35), n = 5; 8.3(± 4.50), n = 4, respectively. Over 48 months, CK and Hex4 biomarkers improved in ambulatory cohorts. Overall, cipa + mig was well tolerated with a safety profile like alglucosidase alfa. ATB200-02 results show the potential benefits of cipa + mig as a long-term treatment option for Pompe disease. Trial registration number: NCT02675465 January 26, 2016. |
| نوع الوثيقة: | article in journal/newspaper |
| وصف الملف: | application/pdf |
| اللغة: | English |
| العلاقة: | https://pure.eur.nl/en/publications/1650e4fe-e335-4dd2-bde7-ae2621abef72Test |
| DOI: | 10.1007/s00415-023-12096-0 |
| الإتاحة: | https://doi.org/10.1007/s00415-023-12096-0Test https://pure.eur.nl/en/publications/1650e4fe-e335-4dd2-bde7-ae2621abef72Test https://pure.eur.nl/ws/files/121767485/Long-term_safety_and_efficacy_of_cipaglucosidase_alfa_plus_miglustat_in_individuals_living_with_Pompe_disease.pdfTest http://www.scopus.com/inward/record.url?scp=85178872458&partnerID=8YFLogxKTest |
| حقوق: | info:eu-repo/semantics/openAccess |
| رقم الانضمام: | edsbas.A3F66545 |
| قاعدة البيانات: | BASE |
| DOI: | 10.1007/s00415-023-12096-0 |
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