A Two-center, Randomized, Double-blind, Placebo-controlled Study of Intravenous Plasma-purified Alpha-1 Antitrypsin for Hospitalized Patients With COPD Exacerbations (AECOPD Study)

Intravenous augmentation therapy with purified preparations of AAT (Alpha1-antitrypsin) derived from human plasma is a well consolidated specific therapeutic option to treat the severe deficient state of AAT. Prolastin is used to restore the balance between AAT and elastases in the lung and consequently to prevent a further deterioration in the pulmonary emphysema. Recently, in patients with COVID-19, without genetically lowered AAT levels with moderate to severe ARDS, treatment with AAT was demonstrated to be safe, feasible, and biochemically efficacious as an anti-inflammatory therapeutic therapy.
The aim of the study, based on biological plausibility, is to evaluate the safety and efficacy (from a biological perspective) of the administration of intravenous plasma-purified AAT as an anti-inflammatory treatment for patients admitted to hospital because of a COPD exacerbation leading to an acute or an acute on chronic respiratory failure.
Thirty-six adult patients hospitalized because of a COPD exacerbation leading to an acute or an acute on chronic respiratory failure will be enrolled by the two sites involved in the study, the Pneumology Unit of Fondazione IRCCS Policlinico San Matteo (Pavia, Italy) and the Pneumology Unit of IRCCS Istituto Clinico Humanitas, Rozzano (Milano, Italy).
Participants will be randomized 2:1 in the active treatment group (standard treatment + IV administration of Prolastin) or in the placebo group (standard treatment+ IV administration of 0.9% sodium chloride).
To address the clinical question from a biological perspective, we will investigate the decrease in inflammatory markers in the active treated group in comparison with the placebo group.
The primary objective is to demonstrate a significant reduction in systemic inflammation by IV Prolastin administered once at 120 mg per kilogram of body weight in patients with moderate to severe AECOPD, as assessed by the change in plasma concentration of IL6 at 7 days after randomization, in the active treatment group with respect to placebo group.
Secondary outcomes are:
1. Difference in change in plasma concentration of IL-1b, IL-5, IL-8, IL-10, and soluble TNF receptor 1 (sTNFR1), CRP at 7 days after randomization.
2. Differences in AAT antielastase activity, the amount of active elastase, the AAT levels in serum at baseline and at 7 days after randomization.
3. Difference in treatment failure rate. Treatment failure is a composite endpoint of need for either NIV or CPAP or need of ETI or need of transfer to ICU or in-hospital death after randomization
4. Impact of AECOPD on overall health, daily life, and perceived well-being in patients with obstructive airways disease by the change of St. George's Respiratory Questionnaire (SGRQ) score from Day 7 to follow-up (30 days after hospital discharge).
5. Differences in type and number of AEs and SAEs in the two groups
The expected duration of subject participation is from randomization to 30 days after hospital discharge, with a follow up phone contact.

开始日期2025-10-01

申办/合作机构-

CTIS2024-515894-80-00

/ Recruiting临床3期

Safety and clinical parameter assessment of a 12-week home self-infusion therapy with Prolastin® in patients with severe alpha-1-antitrypsin (AATD) deficiency. The “Sunshine Study”

开始日期2025-07-01

申办/合作机构-

CTIS2024-517613-33-00

/ Not yet recruiting临床2期IIT

A two-center, randomized, double-blind, placebo-controlled study of intravenous plasma-purified alpha-1 antitrypsin for hospitalized patients with COPD exacerbations (AECOPD study)

开始日期2025-02-01

申办/合作机构

Fondazione IRCCS Policlinico San Matteo

100 项与 Alpha1-proteinase inhibitor(Grifols SA) 相关的临床结果

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100 项与 Alpha1-proteinase inhibitor(Grifols SA) 相关的转化医学

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100 项与 Alpha1-proteinase inhibitor(Grifols SA) 相关的专利（医药）

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项与 Alpha1-proteinase inhibitor(Grifols SA) 相关的文献（医药）

2025-12-01·WATER RESEARCH

Development and validation of a new method for predicting spatial and temporal concentration dynamics of active pharmaceutical ingredients in a lake receiving wastewater effluents

Article

作者: Prajapati, Prajwol ; Kjellin, Johan ; Molander, Sverker ; Gustavsson, Mikael ; Kristiansson, Erik ; Karlsson, Anna ; Wiberg, Karin ; Sokolova, Ekaterina

Human consumption of pharmaceuticals leads to continuous emissions of active pharmaceutical ingredients (APIs) to the aquatic environment, primarily via wastewater treatment plants (WWTPs). However, temporal and spatial patterns of environmental API concentrations are challenging to assess using conventional chemical measurements. Chemical risk assessments are, consequently, typically based on low-resolution data and often overlook API concentrations of environmental concern. We, therefore, developed a new method, combining emission and hydrodynamic modeling to estimate spatiotemporal variations. The method was applied in a case study in a Swedish lake receiving water from a WWTP (capacity 200,000 person equivalents), including >500 prescription APIs. The emission model was validated using ten different APIs measured monthly in the WWTP effluent, and of 103 measured API concentrations 102 were predicted within a factor of 10. The full method was validated against 321 historical measurements from the lake, covering 20 different APIs where 233 (73%) and 319 (99%) of the predicted concentrations were within a factor of 10 and 100 of the measurements. Thus, our method enables predictions of environmental concentrations of APIs, accurate enough to supplement and guide environmental monitoring, directly from human prescription data. Furthermore, we demonstrate that API concentrations vary by orders of magnitude over time and space, directly impacting risk management and monitoring.

2025-11-02·MEDICAL TEACHER

Investigation of the mechanism of ambivalent professional identity formation: A directed content analysis of reflective assignments

Article

作者: Yin, Zhao ; Bao, Xiaoyue ; Hu, Youhong ; Du, Jiayi ; Loraine, Cook ; Yin, Xuejun ; Chen, Zhimin ; Jiang, Shuai ; Yang, Yongjie ; Zhang, Qiufang ; Jia, Xuedong ; Zhang, Wan

BACKGROUND:

Ambivalent professional identity (API)-the coexistence of identification and dis-identification with one's profession-undermines motivation and satisfaction. Research on API among medical interns remains limited. This study applies social cognitive theory (SCT) to explore the formation mechanism of API in Chinese medical interns and proposes strategies to enhance professional identity.

METHODS:

Conducted at the First Affiliated Hospital of Zhengzhou University, the study involved 1,054 medical interns from six institutions and five specialties. In October 2023, all interns submitted reflective writings during the early internship phase. Directed content analysis was used to examine 772 valid submissions.

RESULTS:

Six common themes were identified and categorized into three SCT-based stages: conflictive learning experiences, ambivalent reinforcements, and API formation. Through observation and practice, interns encountered contrasting experiences related to patient outcomes, professional behavior, automation, feedback, task performance, and risk. These shaped mixed perceptions of their roles. Reinforcement processes triggered ambivalent emotions-achievement alongside self-doubt-culminating in API marked by increased responsibility and career interest, yet coupled with anxiety and powerlessness.

CONCLUSION:

SCT effectively explains API formation in medical interns. Ambivalent learning experiences, mediated by contradictory reinforcements, shape identity development, highlighting SCT's relevance in healthcare education.

2025-11-01·JOURNAL OF PHARMACEUTICAL AND BIOMEDICAL ANALYSIS

Structure elucidation and quantification of the active pharmaceutical ingredient in a non-approved drug and in cat serum using QTRAP-MS/MS and ZenoTOF-MS/MS

Article

作者: Koletzko, Berthold ; Horak, Jeannie ; Zwicklbauer, Katharina ; Tu, Luke ; Lescrinier, Eveline ; von Both, Ulrich ; Krentz, Daniela ; Hartmann, Katrin ; Alberer, Martin

Feline infectious peritonitis (FIP) is a fatal systemic disease in cats caused by a feline coronavirus (FCoV). Recent studies have shown that Xraphconn, an unapproved veterinary drug, has significantly increased survival rates in cats with FIP. However, the lack of transparent information regarding the chemical structure and quantity of the active pharmaceutical ingredient (API) necessitates a thorough investigation of this drug. In this study, the API from Xraphconn's plant-based matrix was successfully isolated and purified using weak cation-exchange solid-phase extraction (WCX-SPE). Preliminary structure elucidation was conducted using mass spectra simulations, multiple reaction monitoring-information-dependent acquisition-enhanced product ion (MRM-IDA-EPI) scans from a 6500+ QTRAP-MS, and both collision-induced dissociation (CID) and electron-activated dissociation (EAD) fragmentation techniques with the ZenoTOF 7600. Conclusive structural identification of the Xraphconn API was achieved by ¹³C and ¹H nuclear magnetic resonance spectroscopy. A 4-minute RP HPLC-MS/MS method was optimized to achieve satisfactory separation of the API peak from an isobaric contaminant. The limit of detection (LOD) and limit of quantification (LOQ) were established at 2.11 nM and 6.39 nM in water, and 7.21 nM and 21.84 nM in plasma, respectively. The intra- and inter-day accuracy was found to range between 96 % and 105 %, with a coefficient of variation (CV%) of ≤ 5.51 %. The API content was determined to be 21.56 ± 0.98 mg per tablet employing ¹³C₅-GS-441524 as an internal standard to account for potential matrix effects. The API concentration was measured in plasma and serum samples collected at four different time points from six treated cats.

项与 Alpha1-proteinase inhibitor(Grifols SA) 相关的新闻（医药）

2024-09-12

·Healthcare Digital

Grifols & Orange Business Launch Egypt Plasma Project

Grifols' Cairo project is part of a national initiative to achieve self-sufficiency in plasma-derived medicines. Grifols & Orange Business Complete landmark phase of Data Centre for Plasma Derivatives Facility, designed to boost healthcare in Egypt Global healthcare company Grifols is working with French telecoms giant Orange to create a sustainable supply of essential therapeutics for life-threatening diseases in Egypt. Orange Business – the enterprise division of telecoms company, Orange Group – has completed the initial stage of a new data centre and communications infrastructure in Cairo, for Grifols Egypt for Plasma Derivatives (GEPD). GEPD is a joint venture between Grifols and Egypt's National Service Projects Organisation (NSPO), which manufactures military and civilian products and provides contracting services. The project is part of a national initiative to achieve self-sufficiency in plasma-derived medicines -- vital treatments extracted from blood plasma for various serious medical conditions. Partnership to boost Egyptian healthcare Grifols, a specialist in plasma-derived medicines, is seeking to interconnect critical healthcare facilities, including a plasma laboratory, co-location data centre, plasma warehouse, 20 plasma donation centres, and a state-of-the-art manufacturing facility. Orange Business is providing co-location services, which allows GEPD to house its servers in Orange's data centre. The company is also supplying communication services and support with equipment procurement. The networked healthcare infrastructure is designed to ensure efficient operations and “the timely delivery of plasma-derived medicines to patients in need”, said Dr Ahmed Serag, Chief Strategy and Projects Officer at GEPD. Kristof Symons, CEO International at Orange Business, said: "Our technical skills, experienced resources, and global knowledge were pivotal in enabling Grifols to replicate its global business model in Egypt. This project shows how Orange Business can support its customers in creating a positive impact in today's society." About Grifols Multinational healthcare company Grifols was founded in 1940 in Barcelona, Spain employs 24,000 people worldwide, and specialises in the production of plasma-derived medicines and the development of diagnostic solutions. Its core activities include plasma collection, the manufacture of protein therapies derived from plasma, and fractionation, a process that separates a mixture into two or more fractions with different chemical and physical properties. Grifols is conducting clinical trials for its Alzheimer's treatment , a plasma-based therapy aimed at slowing cognitive decline in patients with mild to moderate Alzheimer's disease. The company is also researching treatments for liver diseases and immunodeficiencies. Among Grifols' products are Gamunex, an immune globulin therapy used to treat primary immunodeficiency, and Prolastin, used in the treatment of alpha-1 antitrypsin deficiency. The firm also produces albumin solutions for fluid replacement and coagulation factors for haemophilia treatment. In the diagnostic sector, Grifols offers blood typing systems and infectious disease screening tools. The company operates a network of plasma donation centres, primarily in the US, which form a crucial part of its supply chain for plasma-derived therapies.

2024-08-04

·药明康德

使90%阿尔茨海默病患者认知功能稳定或改善的NK细胞疗法；旨在功能性治愈乙肝的创新疗法进入临床…… | 一周盘点

▎药明康德内容团队编辑本期看点 1. 治疗阿尔茨海默病（AD）的自然杀伤（NK）细胞疗法SNK01在一项1期临床试验中使90%患者的认知功能稳定或有改善。 2. FDA批准了能在体内制造嵌合抗原受体（CAR）-T细胞的创新疗法UB-VV111的IND申请，预计将于今年年底前启动1期临床试验。 3. 有望功能性治愈乙型肝炎病毒（HBV）感染的重组多克隆抗体疗法GIGA-2339获许可进入临床。药明康德内容团队整理 SNK01：公布1期临床试验的新数据 NKGen Biotech公布了其在研NK细胞疗法SNK01治疗阿尔茨海默病患者的临床试验数据。SNK01是一款自体、非基因工程改造的NK细胞产品，它具有增强的细胞毒性，活化受体表达率超过90%，可从任何供体中稳定生产。该候选疗法能够有效降低AD患者脑脊液（CSF）中的α-突触核蛋白（α-syn）水平，该蛋白的升高被认为与认知能力下降有关。目前，已批准的AD疗法均未将α-syn作为靶点。此前公布的1期试验结果显示，SNK01除了先前披露的对淀粉样蛋白β（Aβ）和神经炎症生物标志物的积极影响外，对晚期AD患者的认知功能也具有临床疗效。在10名可评估的AD患者中，90%患者的阿尔茨海默病综合评分（ADCOMS，一种反映患者认知功能的评分）较基线时有改善或保持稳定。此次公布的结果显示，尽管70%的受试者接受的是相对低剂量的SNK01治疗，仍有60%的受试者（6/10）的CSF α-syn水平较基线时有所下降。第11周时，6名患者中有5名受试者的α-syn下降与ADCOMS的稳定/下降相对应。安全性方面，没有观察到与治疗相关的不良事件。AD受试者对SNK01的耐受性良好，在所有测试剂量下均未观察到剂量限制性毒性。该公司表示，这些数据值得在更大剂量、更长疗程、更大规模的2期试验中进一步研究。 UB-VV111：IND申请获得FDA许可 Umoja Biopharma宣布，美国FDA已批准其在研CD19靶向原位生成（in-situ generated）CAR-T细胞疗法UB-VV111的IND申请，用以治疗血液恶性肿瘤。Umoja预计将在2024年底前启动1期试验并进行首位患者给药。根据新闻稿，UB-VV111可能是血液学人体试验中首个原位生成CD19靶向CAR-T细胞疗法。 UB-VV111是一款通过VivoVec平台开发的体内CAR-T细胞疗法。UB-VV111包含一个表面经过工程化的病毒包膜，以及编码CD19靶向CAR和雷帕霉素激活细胞因子受体（RACRTM）的转基因，旨在于患者体内扩增UB-VV111工程化CAR-T细胞。该疗法是VivoVec基因递送平台进入临床的首个管线。 GIGA-2339：IND申请获得FDA许可 GigaGen公司宣布，美国FDA已批准其IND申请，将于2024年第四季度启动一项1期试验，以评估该公司首款用于治疗HBV感染的重组多克隆抗体疗法GIGA-2339。GIGA-2339采用GigaGen的下一代平台开发，含有1000多种全人重组抗HBV抗体，旨在复制人体的自然免疫反应。GIGA-2339的效力是血浆衍生HBV药物的2000多倍，涵盖了广泛的循环HBV变异。在小鼠模型中，GIGA-2339中和并清除了HBV的病毒DNA及其抗原。通过这种独特的作用机制，GIGA-2339有可能清除病毒颗粒并激活免疫反应，从而提供HBV感染患者的功能性治愈。 Sabirnetug（ACU193）：公布1期临床试验的新数据 Acumen Pharmaceuticals公司公布了sabirnetug（ACU193）用于治疗AD的1期临床试验的新结果。Sabirnetug是一种选择性靶向毒性可溶性β淀粉样蛋白寡聚体（AβOs）的人源化单克隆抗体。Sabirnetug能够结合AβOs，由于其独特的结合特性，sabirnetug有可能在产生淀粉样蛋白相关成像异常（ARIA，一种抗体疗法产生的常见主要副作用）风险较低的情况下提供治疗益处，因为sabirnetug可以在不直接靶向淀粉样斑块的情况下阻断AβOs的毒性作用。此次公布的结果显示，接受sabirnetug给药3次可显著降低患者脑脊液中突触前和突触后蛋白水平，这与sabirnetug抑制AβOs突触结合的作用机制一致。VAMP2是一种与突触损伤相关的生物标志物，在所有多剂量递增组中均显著降低，似乎是该研究中发现的对sabirnetug最敏感的生物标志物。Acumen公司计划在正在进行的为期18个月的2期临床试验中评估生物标志物的长期变化及其与临床结果的关系，以进一步证实sabirnetug的作用机制。此外，Acumen公司还宣布了sabirnetug皮下制剂治疗早期AD的1期临床试验已完成首例受试者给药。 Pepinemab：公布1b/2a期临床试验数据 Vaccinex公司公布了pepinemab治疗AD的1b/2期试验数据的良好初步结果。Pepinemab是一种人源化IgG4单克隆抗体，旨在阻断SEMA4D与plexin-B1受体结合，从而避免引发细胞内肌动蛋白细胞骨架的崩溃，以及进一步导致的大脑中星形胶质细胞和其他胶质细胞以及免疫组织中树突状细胞的稳态功能丧失。此次公布的结果证实了以激活星形胶质细胞为目标的新型作用机制有望延缓AD从早期轻度认知障碍发展为老年痴呆症的进程。该研究结果与此前pepinemab治疗亨廷顿病的2期临床试验结果类似，因为AD与亨廷顿病在神经免疫病理学方面具有相似性。 RGX-202：公布1/2期临床试验的新数据 REGENXBIO公司宣布，其在研杜氏肌营养不良症（DMD）基因疗法RGX-202在治疗1至11岁DMD患者的1/2期临床试验中获得了积极的中期安全性和疗效数据。在接受关键性临床试验推荐剂量（剂量水平2）的RGX-202治疗的5.8岁和8.5岁患者中，接受治疗后三个月时微抗肌营养不良蛋白表达水平分别为健康对照组的77.2%和46.5%。该试验中按剂量和年龄分类的微抗肌营养不良蛋白表达结果如下表所示： ▲AFFINITY DUCHENNE试验中接受不同剂量RGX-202治疗患者的微抗肌营养不良蛋白表达水平（图片来源：参考资料[1]） RGX-202是一种一次性基于腺相关病毒载体（AAV）的基因疗法，它编码包含天然抗肌营养不良蛋白C端（CT）结构域的微抗肌营养不良蛋白，以更好地模拟天然蛋白的功能。临床前研究中，CT结构域已被证明可以保护肌肉免受收缩引起的应力，并提高其自我修复的能力。 XPro：公布1期临床试验数据 INmune Bio公司公布了其下一代肿瘤F坏死因子（TNF）抑制剂XPro用于治疗AD的1b期临床试验结果。XPro的作用与现有的TNF抑制剂不同，它能中和可溶性TNF（sTNF），而不影响跨膜TNF（tmTNF）或TNF受体。XPro可在不抑制免疫的情况下减轻神经炎症，从而对神经系统疾病患者产生潜在的实质性益处。此次公布的结果显示，接受XPro治疗12周后，患者的突触蛋白发生了显著变化，而突触蛋白对神经元之间的交流至关重要。 NTLA-3001：获批在英国启动1/2期临床试验 Intellia Therapeutics公司宣布其临床试验申请（CTA）获得英国药品与保健品管理局（MHRA）的授权，可启动一项1/2期研究以评估NTLA-3001治疗α1抗胰蛋白酶缺乏症（AATD）相关的肺部疾病的效果。AATD是一种罕见的遗传性疾病，最常见的表现是由于α1抗胰蛋白酶（AAT）蛋白水平不足导致的肺功能障碍。NTLA-3001是一种全身给药的体内一次性基因编辑疗法，它旨在通过CRISPR/Cas9技术精确插入编码AAT蛋白的SERPINA1基因的健康拷贝，单次给药就有可能使功能性AAT蛋白永久表达并恢复到治疗水平。这种方法旨在改善患者的治疗效果，包括消除每周静脉注射AAT的疗法或严重病例对肺移植的需求。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看） [1] REGENXBIO ANNOUNCES NEW POSITIVE DATA FROM AFFINITY DUCHENNE® TRIAL OF RGX-202. Retrieved August 1, 2024, from https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-announces-new-positive-data-affinity-duchenner-trial [2] Acumen Pharmaceuticals Presents Patient Experience and Biomarker Data from Phase 1 INTERCEPT-AD Study at the Alzheimer’s Association International Conference (AAIC®) 2024. Retrieved August 2, 2024, from https://www.globenewswire.com/news-release/2024/07/28/2919969/0/en/Acumen-Pharmaceuticals-Presents-Patient-Experience-and-Biomarker-Data-from-Phase-1-INTERCEPT-AD-Study-at-the-Alzheimer-s-Association-International-Conference-AAIC-2024.html [3] Acumen Pharmaceuticals Announces First Subject Dosed in Phase 1 Study of Subcutaneous Sabirnetug (ACU193) for Early Alzheimer’s Disease. Retrieved August 2, 2024, from https://investors.acumenpharm.com/news-releases/news-release-details/acumen-pharmaceuticals-announces-first-subject-dosed-phase-1 [4] NKGen Biotech Presents New Positive SNK01 Biomarker Data at the 2024 Alzheimer’s Association International Conference. Retrieved August 2, 2024, from https://nkgenbiotech.com/nkgen-biotech-presents-new-positive-snk01-biomarker-data-at-the-2024-alzheimers-association-international-conference/ [5] Umoja Biopharma Announces FDA Clearance of IND Application for UB-VV111, a CD19 Directed in situ CAR T for Hematologic Malignancies. Retrieved July 31, 2024 from https://www.umoja-biopharma.com/news/umoja-biopharma-announces-fda-clearance-of-ind-application-for-ub-vv111-a-cd19-directed-in-situ-car-t-for-hematologic-malignancies/ [6] Intellia Therapeutics Receives Authorization to Initiate Phase 1/2 Clinical Trial of NTLA-3001 for the Treatment of Alpha-1 Antitrypsin Deficiency. Retrieved August 2, 2024, from https://www.globenewswire.com/news-release/2024/07/30/2920893/0/en/Intellia-Therapeutics-Receives-Authorization-to-Initiate-Phase-1-2-Clinical-Trial-of-NTLA-3001-for-the-Treatment-of-Alpha-1-Antitrypsin-Deficiency.html [7] Vaccinex Reports Positive Data for SIGNAL-AD Phase 1b/2 trial of Pepinemab in Alzheimer’s Disease. Retrieved August 2, 2024, from https://www.globenewswire.com/news-release/2024/07/31/2921918/34858/en/Vaccinex-Reports-Positive-Data-for-SIGNAL-AD-Phase-1b-2-trial-of-Pepinemab-in-Alzheimer-s-Disease.html [8] Citryll Completes Patient Dosing In The Repeat Dose Stage Of The Phase 1 Trial. Retrieved August 2, 2024, from https://citryll.com/newsitems/citryll-completes-patient-dosing-in-the-repeat-dose-stage/ [9] GigaGen Receives FDA Clearance of IND Application for Phase 1 Trial of Recombinant Polyclonal for HBV Treatment, GIGA-2339. Retrieved August 2, 2024, from https://www.globenewswire.com/news-release/2024/07/31/2921826/0/en/GigaGen-Receives-FDA-Clearance-of-IND-Application-for-Phase-1-Trial-of-Recombinant-Polyclonal-for-HBV-Treatment-GIGA-2339.html [10] Compugen Announces FDA Clearance of IND for COM503 for the Treatment of Solid Tumors. Retrieved August 2, 2024, from https://www.prnewswire.com/news-releases/compugen-announces-fda-clearance-of-ind-for-com503-for-the-treatment-of-solid-tumors-302208442.html [11] Georgiamune Announces First Patient Dosed in A First-In-Class Phase 1 Cancer Treatment Trial of GIM-531. Retrieved August 2, 2024, from https://www.globenewswire.com/news-release/2024/07/30/2921055/0/en/Georgiamune-Announces-First-Patient-Dosed-in-A-First-In-Class-Phase-1-Cancer-Treatment-Trial-of-GIM-531.html [12] INmune Bio Inc. Announces New Phase 1 Study Data Demonstrating Dose-Dependent Effect of XPro™ on Proteins that Regulation Synapses in Alzheimer’s Patients. Retrieved August 2, 2024, from https://www.inmunebio.com/index.php/newsroom/2024-news/muneioncnnouncesewhase1tudyataemonstrati20240729060507 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

细胞疗法临床申请临床1期免疫疗法临床2期

2023-11-15

·GlobeNewswire-Health Care

Grifols Completes Cohort 1 in Clinical Study of Alpha-1 15%, Evaluating First-in-Human Subcutaneous Dosing Option for Patients with Alpha1-Antitrypsin Deficiency

Study designed to demonstrate impact of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% (Alpha-1 15%), an alpha1 antitrypsin treatment, compared with Liquid Alpha1-Proteinase Inhibitor (Human) intravenousThis first in-human subcutaneous approach to treating alpha1-antitrypsin deficiency, if proven successful in clinical trials, could give patients the convenience and flexibility to administer their medication from home BARCELONA, Spain, Nov. 15, 2023 (GLOBE NEWSWIRE) -- Grifols (MCE: GRF, MCE: GRF.P NASDAQ: GRFS), one of the world’s leading producers of plasma-derived medicines, today announced that it has completed Cohort 1 of its Phase 1/2 study (NCT04722887) evaluating Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% (Alpha-1 15%), a subcutaneous (SC) alpha1 antitrypsin (AAT) treatment being compared to Liquid Alpha1-Proteinase Inhibitor (Human) intravenous (IV). Alpha1-antitrypsin deficiency (also known as alpha-1) is an underdiagnosed1 genetic disorder that occurs when a patient has low levels of AAT, a protective protein that safeguards the lungs. Augmentation therapy with IV AAT is the standard medical treatment option for patients with severe AAT deficiency and emphysema. If proven successful in clinical trials, a SC option could provide alpha-1 patients the ability to independently administer AAT therapy from home, allowing for greater convenience and flexibility. In this multi-center, single-dose and repeat-dose study over eight weeks, Cohort 1 has been completed and demonstrated no safety issues with Alpha-1 15% that would prevent the study from moving forward into Cohort 2. “A subcutaneous option would be a first for alpha-1 patients, providing more freedom when it comes to managing their AAT deficiency by allowing patients to administer their medication from the comfort of their own home. We are pleased to announce this milestone during Alpha-1 Awareness Month, contributing to raising further visibility about this rare disease,” said Jörg Schüttrumpf, Chief Scientific Innovation Officer, Grifols. “We look forward to moving this study into Cohort 2. Grifols has a strong commitment to the alpha-1 community and continues to innovate to find additional treatment options for patients living with this disease, in addition to more convenient alpha-1 testing alternatives such as AlphaID™ At Home, our recently launched direct-to-consumer genetic health risk service.” “During Alpha-1 Awareness month, we are excited to share this exciting news to the Alpha-1 community of a new clinical trial advancement in Alpha-1. We are thrilled that Grifols has completed Cohort 1 in clinical study of Alpha-1 15% and moving forward into Cohort 2. This study evaluating first-in-human subcutaneous dosing option for Alpha-1 patients could provide a new option that could easily be administered from their own homes,” said Scott Santarella, Alpha-1 Foundation President and CEO. About Grifols Grifols is a global healthcare company founded in Barcelona in 1909 committed to improving the health and well-being of people around the world. A leader in essential plasma-derived medicines and transfusion medicine, the company develops, produces and provides innovative healthcare services and solutions in more than 110 countries. Patient needs and Grifols’ ever-growing knowledge of many chronic, rare and prevalent conditions, at times life-threatening, drive the company’s innovation in both plasma and other biopharmaceuticals to enhance quality of life. Grifols is focused on treating conditions across a broad range of therapeutic areas: immunology, hepatology and intensive care, pulmonology, hematology, neurology and infectious diseases. A pioneer in the plasma industry, Grifols continues to grow its network of donation centers, the world’s largest with over 390 across North America, Europe, Africa and the Middle East and China. As a recognized leader in transfusion medicine, Grifols offers a comprehensive portfolio of solutions designed to enhance safety from donation to transfusion, in addition to clinical diagnostic technologies. It provides high-quality biological supplies for life-science research, clinical trials, and for manufacturing pharmaceutical and diagnostic products. The company also supplies tools, information and services that enable hospitals, pharmacies and healthcare professionals to efficiently deliver expert medical care. Grifols, with more than 24,000 employees in more than 30 countries and regions, is committed to a sustainable business model that sets the standard for continuous innovation, quality, safety and ethical leadership. In 2022, Grifols’ economic impact in its core countries of operation was EUR 9.6 billion. The company also generated 193,000 jobs, including indirect and induced. The company’s class A shares are listed on the Spanish Stock Exchange, where they are part of the Ibex-35 (MCE:GRF). Grifols non-voting class B shares are listed on the Mercado Continuo (MCE:GRF.P) and on the U.S. NASDAQ through ADRs (NASDAQ:GRFS). For more information, please visit www.grifols.com MEDIA CONTACT:Media Press Officemedia@grifols.com Tel. +34 93 571 00 02 INVESTORS: Investors Relations Department & Sustainabilityinversores@grifols.com - investors@grifols.com Tel. +34 93 571 02 21 LEGAL DISCLAIMER The facts and figures contained in this report that do not refer to historical data are “future projections and assumptions”. Words and expressions such as “believe”, “hope”, “anticipate”, “predict”, “expect”, “intend”, “should”, “will seek to achieve”, “it is estimated”, “future” and similar expressions, insofar as they relate to the Grifols group, are used to identify future projections and assumptions. These expressions reflect the assumptions, hypotheses, expectations and predictions of the management team at the time of writing this report, and these are subject to a number of factors that mean that the actual results may be materially different. The future results of the Grifols group could be affected by events relating to its own activities, such as a shortage of supplies of raw materials for the manufacture of its products, the appearance of competitor products on the market, or changes to the regulatory framework of the markets in which it operates, among others. At the date of compiling this report, the Grifols group has adopted the necessary measures to mitigate the potential impact of these events. Grifols, S.A. does not accept any obligation to publicly report, revise or update future projections or assumptions to adapt them to events or circumstances subsequent to the date of writing this report, except where expressly required by the applicable legislation. This document does not constitute an offer or invitation to buy or subscribe shares in accordance with the provisions of the following Spanish legislation: Royal Legislative Decree 4/2015, of 23 October, approving recast text of Securities Market Law; Royal Decree Law 5/2005, of 11 March and/or Royal Decree 1310/2005, of 4 November, and any regulations developing this legislation. In addition, this document does not constitute an offer of purchase, sale or exchange, or a request for an offer of purchase, sale or exchange of securities, or a request for any vote or approval in any other jurisdiction. The information included in this document has not been verified nor reviewed by the external auditors of the Grifols group 1 American Thoracic Society; European Respiratory Society. American Thoracic Society/European Respiratory Society statement: standards for the diagnosis and management of individuals with alpha-1 antitrypsin deficiency. Am J Respir Crit Care Med. 2003;168(7):818-900. doi:10.1164/rccm.168.7.818

100 项与 Alpha1-proteinase inhibitor(Grifols SA) 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	Alpha1-proteinase inhibitor(Grifols SA)	B02AB02	DB00058

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
α1-抗胰蛋白酶缺乏症	日本	OrphanPacific, Inc.	2021-01-22
肺气肿	美国	Grifols Therapeutics, Inc.	1987-12-02

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
HIV感染	临床3期	美国	Grifols Biologicals, Inc.	2012-04-01
新型冠状病毒感染	临床2期	西班牙	Instituto Grifols SA	2020-07-29
1型糖尿病	临床2期	美国	Grifols Therapeutics, Inc.	2014-03-01
囊性纤维化	临床2期	-	Grifols Therapeutics, Inc.	2003-12-01
气肿	临床2期	丹麦	Grifols Therapeutics, Inc.	2003-12-01
气肿	临床2期	瑞典	Grifols Therapeutics, Inc.	2003-12-01
气肿	临床2期	英国	Grifols Therapeutics, Inc.	2003-12-01

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT02947087 (CTgov)	临床1/2期	慢性胰腺炎	48	Prolastin-C (Prolastin-C)	糧醖顧網鬱餘糧獵糧鹹(蓋構獵鹹壓製獵夢遞鹽) = 醖廠製願廠網廠鹽壓醖網膚構齋淵網繭膚醖窪 (簾鹽艱膚網齋淵鹹鑰構, 42) 更多	-	2023-05-09
				Placebo (Placebo)	糧醖顧網鬱餘糧獵糧鹹(蓋構獵鹹壓製獵夢遞鹽) = 蓋鑰獵蓋範鹽製鬱夢鏇網膚構齋淵網繭膚醖窪 (簾鹽艱膚網齋淵鹹鑰構, 78) 更多
NCT04547140 (CTgov)	临床2期	新型冠状病毒感染	57	Standard Medical Treatment	鹹夢窪餘鬱餘鑰製遞夢 = 範願簾窪網觸範齋醖餘憲鹹蓋餘醖築鏇窪繭夢 (鬱淵襯窪醖觸蓋襯壓廠, 繭窪積齋夢遞顧網繭醖 ~ 憲襯獵艱鏇遞艱選網遞) 更多	-	2023-03-22
JPRN-JapicCTI-163194 \| NCT02870348 (GTI1401-OLE, Pubmed \| Respir Investig) 人工标引	临床1/2期	α1-抗胰蛋白酶缺乏症	4	Alpha1-proteinase inhibitor	夢糧襯窪繭憲遞衊齋艱(襯醖觸繭鬱壓繭鏇鏇繭) = 鏇憲積繭顧淵膚窪襯築蓋鹹窪簾鏇鬱觸衊遞鏇 (遞蓋淵淵鬱鑰窪醖蓋夢 ) 更多	积极	2022-08-12
NCT02870348 (GTI1401-OLE, CTgov)	临床1/2期	α1-抗胰蛋白酶缺乏症	4	Alpha-1 MP	艱繭醖範壓膚糧艱壓觸 = 觸艱醖蓋選鹽夢繭廠鹽積繭蓋構鑰觸獵壓鹽鹽 (衊鑰獵積製憲憲鹹鹽築, 網糧製餘鏇積衊製醖憲 ~ 築衊積膚糧簾範遞廠鏇) 更多	-	2022-03-21
NCT01731691 (CTgov)	临床2/3期	HIV感染	12	α1 Proteinase Inhibitor (α1 Proteinase Inhibitor in HIV Disease)	築範顧觸獵築醖膚選遞(築鹽遞衊觸選齋艱夢簾) = 繭壓製構遞顧網鑰鹽鑰願積鹽廠選壓醖廠願鹽 (廠範醖鏇觸鬱繭築鏇構, 103) 更多	-	2020-08-31
				Placebos (Placebo in HIV Disease)	築範顧觸獵築醖膚選遞(築鹽遞衊觸選齋艱夢簾) = 遞醖積製鬱觸窪膚夢顧願積鹽廠選壓醖廠願鹽 (廠範醖鏇觸鬱繭築鏇構, 180) 更多
NCT02093221 (CTgov)	临床2期	1型糖尿病	76	180 mg/kg Alpha1-PI (Alpha1-PI 180 mg/kg/wk, 26 Weeks)	艱構製製積廠餘鹽繭襯(遞積艱顧顧構遞餘獵齋) = 憲選襯顧壓衊憲簾糧鏇選壓製築夢蓋選鑰餘觸 (艱製顧構憲範衊醖製膚, 27.7184) 更多	-	2018-04-19
				180 mg/kg Alpha1-PI (180 mg/kg/wk Alpha1-PI, 13 Weeks)	艱構製製積廠餘鹽繭襯(遞積艱顧顧構遞餘獵齋) = 淵鑰鹽淵鹽鬱齋醖壓衊選壓製築夢蓋選鑰餘觸 (艱製顧構憲範衊醖製膚, 28.9450) 更多
NCT02282527 (CTgov)	临床2/3期	α1-抗胰蛋白酶缺乏症	32	Alpha1-PI (Liquid Alpha₁-PI)	壓願膚膚淵積觸襯願網(願艱壓壓鬱齋壓窪膚餘) = 鬱醖觸簾繭餘窪蓋簾艱積獵衊淵獵憲鏇醖淵蓋 (醖顧壓簾衊觸蓋憲獵觸, 23.041) 更多	-	2017-03-13
				Prolastin-C (Prolastin-C)	壓願膚膚淵積觸襯願網(願艱壓壓鬱齋壓窪膚餘) = 遞鹹願繭築艱齋廠顧齋積獵衊淵獵憲鏇醖淵蓋 (醖顧壓簾衊觸蓋憲獵觸, 25.230) 更多
NCT01936896 (VCU-α1RT \| VCU-Alpha1RT, CTgov)	临床1/2期	ST段抬高心肌梗死 \| 急性心肌梗塞	10	Alpha 1-Antitrypsin	繭製網膚繭衊廠積鏇淵(壓選製餘壓顧醖鏇艱膚) = 範鹹繭鑰積淵壓夢憲鏇憲願窪廠範壓艱製襯憲 (窪衊襯鬱網艱築淵壓壓, 鑰糧製糧窪簾願製積艱 ~ 齋廠鏇壓網壓積餘艱構) 更多	-	2016-02-12
NCT01213043 (SPARK, CTgov)	临床2期	α1-抗胰蛋白酶缺乏症 \| 肺气肿	30	Prolastin-C (60 mg/kg Prolastin-C)	鹽廠願壓積襯憲窪膚淵 = 鹽遞憲築範窪構齋鹽窪構獵鹽獵構餘膚壓築壓 (築窪齋窪構鹹壓壓構醖, 鹽簾繭壓築蓋觸築製選 ~ 襯襯鏇壓憲淵繭艱襯範) 更多	-	2013-05-20
				Prolastin-C (120 mg/kg Prolastin-C)	鹽廠願壓積襯憲窪膚淵 = 廠簾壓糧鹽餘簾壓鏇窪構獵鹽獵構餘膚壓築壓 (築窪齋窪構鹹壓壓構醖, 襯襯積鹹鹽憲顧壓鬱構 ~ 鬱窪鏇鹽壓鏇製構積蓋) 更多