A Phase 3, Randomized, Open-label, Parallel-arm, Active-controlled, Multicenter Study to Evaluate Safety and Efficacy of ALXN1850 Versus Asfotase Alfa Administered Subcutaneously in Pediatric Participants (2 to < 12 Years of Age) With Hypophosphatasia (HPP) Previously Treated With Asfotase Alfa

The primary purpose of this study is to assess the safety and tolerability of ALXN1850 versus asfotase alfa in pediatric participants with HPP previously treated with asfotase alfa.

开始日期2024-04-02

申办/合作机构

Alexion Pharmaceuticals, Inc.

NCT05234567 / RecruitingN/A

A Prospective Observational Sub-Study of the Global Hypophosphatasia Registry to Describe the Potential Risk of Immune-Mediated Loss of Pharmacological Effect of Asfotase Alfa in Participants With Hypophosphatasia

In this prospective observational sub-study, participants with pediatric-onset hypophosphatasia (HPP) (perinatal/infantile- or juvenile-onset) of any age will be followed for a minimum of 5 years at sites in the United States and potentially 1 or 2 other countries.

开始日期2022-08-25

申办/合作机构

Alexion Pharmaceuticals, Inc.

NCT04189315 / Withdrawn临床4期

A Phase 4, Randomized, Multicenter, Open-Label, 2-Dosage Regimen, Safety and Tolerability, Efficacy, Pharmacokinetic, and Pharmacodynamic Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia

The study will evaluate safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of 2 different doses (approved dose and lower dose) of asfotase alfa in adult participants with pediatric-onset hypophosphatasia (HPP).

开始日期2020-04-01

申办/合作机构

Alexion Pharmaceuticals, Inc.

100 项与阿司福酶α 相关的临床结果

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100 项与阿司福酶α 相关的转化医学

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100 项与阿司福酶α 相关的专利（医药）

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128

项与阿司福酶α 相关的文献（医药）

2024-03-01·AACE clinical case reports

A Case of Hypophosphatasia With Normal Alkaline Phosphatase Levels

作者: Dattagupta, Antara ; Petak, Steven

Background/Objective:

Hypophosphatasia (HPP) is a rare disease associated with low serum alkaline phosphatase (ALP) activity. Here, we present a case of a patient with normal serum ALP levels diagnosed with HPP.

Case Report:

A 36-year-old woman presented with progressive fatigue, weakness, and joint pain. She had been evaluated in the past for genetic disorders due to these symptoms and was found to have a history of several total ALP levels within normal limits but elevated vitamin B6 levels. She also reported having loose teeth and "gray gums" during her childhood. Bone-specific ALP was tested for suspicion of HPP and returned at 4.4 μ/L (reference range, 5.3-19.5 μg/L), which prompted genetic testing. Genetic testing confirmed a positive pathogenetic variant of the ALPL gene, the c.542C>T (p.Ser181Leu) variant. She started asfotase alfa treatment to improve her symptoms.

Discussion:

HPP was diagnosed based on clinical suspicion supported by laboratory findings, which can cause it to be underdiagnosed or misdiagnosed. Current literature reports that a low total ALP level is the main biochemical marker of HPP and the only level needed to diagnose the disease. However, bone-specific ALP, a common marker used for bone turnover, has not been required to be tested.

Conclusion:

This case highlights a patient with normal total ALP, but low bone-specific ALP diagnosed with HPP confirmed by genetic testing. This case warrants future investigation into the diagnostic approach to HPP and the diagnostic utility between ALP and bone-specific ALP.

2023-12-01·JBMR plus

The Effect of Asfotase Alfa on Plasma and Urine Pyrophosphate Levels and Pseudofractures in a Patient With Adult‐Onset Hypophosphatasia

作者: Kato, Hajime ; Hoshino, Yoshitomo ; Kinoshita, Yuka ; Ito, Nobuaki ; Makita, Noriko ; Tachikawa, Kanako ; Watanabe, So ; Murata, Hiroaki ; Osaki, Keiichi ; Hidaka, Naoko ; Michigami, Toshimi ; Sunouchi, Takashi ; Kimura, Soichiro ; Nangaku, Masaomi ; Sekiyama, Takashi

ABSTRACT:

Hypophosphatasia (HPP) is an inherited disease caused by variants of the ALPL gene encoding tissue‐nonspecific alkaline phosphatase. Adult‐onset HPP (adult HPP), known as a mild form of HPP, develops symptoms involving osteomalacia after the age of 18 years. Asfotase alfa (AA) is a modulated recombinant human alkaline phosphatase (ALP) that has been established as a first‐line therapy for severe forms of HPP, such as perinatal and infantile forms. We described a 64‐year‐old female who presented with pseudofractures in bilateral femur diaphyses and impaired mobility. Low serum ALP activity and a high concentration of urine phosphoethanolamine indicated the diagnosis of HPP, which was confirmed by the identification of a homozygous variant in the ALPL gene (c.319G > A; p.Val107Ile). An in vitro transfection experiment to measure the ALP activity of this novel variant protein was performed, resulting in 40% of the residual enzymatic activity compared with the wild type. AA was initiated to facilitate the union of pseudofracture and to improve mobility. After 6 months, radiographic images revealed the disappearance of fracture lines, and improvement of ambulatory ability was confirmed by the 6‐minute walk test (525 to 606 m). The EQ‐5D‐5L index was also improved (0.757 to 0.895). Within a follow‐up period, the levels of urine pyrophosphate corrected by urine creatinine (uPPi/Cre) declined in parallel with the level of plasma PPi (plasma PPi: 6.34 to 1.04 μM, uPPi/Cre: 226.8 to 75.4 nmol/mg). The beneficial effect of AA on pseudofracture healing in adult HPP was presented, although the application of AA should be restricted to patients exhibiting relatively severe manifestations. In addition, a novel pathogenic variant of the ALPL gene was identified with the supportive result of functional analysis. Furthermore, when monitoring patients with HPP treated with AA, uPPi/Cre might be a convenient substitute for plasma PPi, which requires immediate filtration after blood sampling. © 2023 The Authors. JBMR Plus published by Wiley Periodicals LLC. on behalf of American Society for Bone and Mineral Research.

2023-10-01·Bone

Effects of asfotase alfa in adults with pediatric-onset hypophosphatasia over 24 months of treatment

Article

作者: Genest, Franca ; Petryk, Anna ; Seefried, Lothar ; Veith, Marina

BACKGROUND:

Hypophosphatasia (HPP) is a rare, heritable metabolic disorder caused by deficient activity of tissue-nonspecific alkaline phosphatase (TNSALP). Asfotase alfa (AA) is a human recombinant TNSALP that promotes bone mineralization and is approved to treat eligible patients with HPP.

METHODS:

This prospective single-center observational study evaluated AA in adults with pediatric-onset HPP over 2 years of treatment (ClinicalTrials.govNCT03418389). Primary outcomes evaluated physical function; secondary outcomes assessed quality of life (QoL) and pain.

RESULTS:

The study included 17 females and 5 males (mean age: 48.7 years). Median distance walked in the 6-Minute Walk Test increased significantly from baseline to 12 months (P = 0.034) and results were sustained. Median Timed Up and Go test time significantly decreased from baseline at 12 (P = 0.003) and 24 months (P = 0.005), as did the median chair rise time test at 12 (P = 0.003) and 24 months (P < 0.002). The change from baseline in usual gait speed was significant at 12 (P = 0.003) and 24 months (P = 0.015). Mean dominant and nondominant hand grip strength improved at 24 months (P = 0.029 and P = 0.019, respectively). Median Short Form 36 Physical Component Summary scores significantly improved from baseline at 12 (P = 0.012) and 24 (P = 0.005) months, and median Lower Extremity Functional Scale scores improved from baseline at 12 (P = 0.001) and 24 (P = 0.002) months. No significant change was noted in pain level at these timepoints. While injection site reactions occurred in 86.4 % of the participants, there were no severe side effects or safety findings.

CONCLUSIONS:

Adults with pediatric-onset HPP treated with AA experienced marked improvement in functional and QoL outcomes that were observed as early as within 3 months of initial treatment and were sustained over 24 months.

项与阿司福酶α 相关的新闻（医药）

2024-09-12

·蒲公英Ouryao

【广东】第7批大湾区临床急需进口港澳药品目录发布

来源：广东药监局编辑：wangxinglai2004 2024年09月11日，广东药监发布了《第七批粤港澳大湾区内地临床急需进口港澳药品医疗器械目录通告》，涉及药品10种。根据《粤港澳大湾区药品医疗器械监管创新发展工作方案》（国市监药〔2020〕159号）和《广东省粤港澳大湾区内地临床急需进口港澳药品医疗器械管理暂行规定》（粤药监规许〔2021〕4号）要求，结合工作实际，广东省药品监督管理局、广东省卫生健康委员会决定发布第七批粤港澳大湾区内地临床急需进口港澳药品医疗器械目录（以下简称目录）。涉及药品名称：阿扎胞苷片、曲美木单抗、注射用芦比替定、罗培干扰素α-2b注射液、泊那替尼片、阿司福酶α注射液、阿伏利尤单抗、索米妥昔单抗、磷酸芦可替尼乳膏、特泽利尤单抗；一、药品目录二、医疗器械目录

申请上市

2024-08-21

·GBIHealth

优于泰瑞沙！强生埃万妥单抗联合兰泽替尼在美获批一线治疗NSCLC

药械追踪 No.1 / GSK/翰森制药B7-H3靶向ADC获FDA突破性疗法认定 2024年8月21日，葛兰素史克（GSK）与翰森制药（3692.HK）共同宣布，靶向B7-H3的抗体偶联药物（ADC）HS-20093（亦称GSK5764227）获得美国FDA突破性疗法认定，用于治疗复发或难治性广泛期小细胞肺癌（SCLC）。 B7-H3 又名 CD276，是一种免疫调节蛋白，在肿瘤组织中过度表达。HS-20093由全人源化的B7-H3单抗与拓扑异构酶抑制剂（TOPOi）有效载荷共价连接而成，可靶向杀伤表达B7-H3的恶性肿瘤。GSK与翰森制药曾在2023年12月达成授权合作，获得HS-20093的全球独占许可（不含中国大陆及港澳台）；交易预付款1.85 亿美元，里程碑费用15.25 亿美元。 HS-20093此次获FDA突破性疗法认定主要基于Ⅰ期开放标签、多中心试验ARTEMIS-001的数据支持。这项试验共有200多名患者参与，评估了HS-20093用于局部晚期或转移性实体瘤（包括复发或难治性ES-SCLC）的安全性、耐受性和初步抗肿瘤活性。该试验结果将于2024年世界肺癌大会（WCLC）上公布。 ->点击阅读原文，解锁完整双语新闻 No.2 / 优于泰瑞沙！强生埃万妥单抗联合兰泽替尼在美获批一线治疗NSCLC 强生（J&J；NYSE：JNJ）近日宣布，EGFR/c-Met双特异性抗体埃万妥单抗与EGFR TKI兰泽替尼（lazertinib）的联合方案获得美国FDA批准，用于一线治疗具有表皮生长因子受体（EGFR）外显子19缺失或外显子21 （L858R）置换突变的局部晚期或转移性非小细胞肺癌（NSCLC）成人患者。埃万妥单抗联合兰泽替尼是全球首个且目前唯一在NSCLC一线治疗中优于阿斯利康第三代EGFR TKI 泰瑞沙（奥希替尼）的多靶点、去化疗联合方案。埃万妥单抗是一种人源化双特异性单克隆抗体，能够同时与EGFR和MET结合，进而抑制相关的信号通路。兰泽替尼是一款第三代EGFR TKI，由强生于2018年从韩国公司Yuhan Corporation处获得许可权利。此次获批系兰泽替尼在全球范围内的首次批准。关键III期试验MARIPOSA支持了此次批准，该研究证明了埃万妥单抗与兰泽替尼联合一线治疗对EGFR阳性NSCLC的有效性。结果显示，与泰瑞沙相比，埃万妥单抗联合兰泽替尼可将患者的疾病进展或死亡风险降低30%，并显著延长中位无进展生存期（mPFS：23.7个月vs 16.6个月）和中位缓解持续时间（mDOR：25.8个月vs16.7个月）。 ->点击阅读原文，解锁完整双语新闻 No.3 / 阿斯利康罕见病药Strensiq引进大湾区 2024年8月21日，阿斯利康罕见病领域创新药Strensiq（通用名：阿司福酶α注射液/asfotase alfa）已获广东省药品监督管理局批准，正式引进大湾区药械通指定医疗机构，用于作为婴儿期发病的低磷酸酯酶症（hypophosphatasia，HPP）患者的长期酶替代疗法，以治疗该疾病的骨骼表现。低磷酸酯酶症是一种极其罕见的遗传性渐进式代谢紊乱疾病，患者因缺少碱性磷酸酶（alkaline phosphatase，ALP），骨矿化和骨质素生成受到影响，致使骨骼生长和其强度出现问题，并引发其他并发症，可能面临骨折、骨痛、行走和呼吸困难、癫痫等问题，甚至造终身负担，严重影响生存质量。阿司福酶α是全球首个且唯一获批用于治疗低磷酸酯酶症的酶替代疗法，最早于2015年7月在日本获批，随后陆续在欧盟和美国获批上市。酶替代治疗是国际公认的针对低磷酸酯酶症病因的有效治疗方法，尤其对于年幼和症状明显的患者，可被作为首选治疗方式。此次，阿司福酶α注射液得益于“港澳药械通”政策的支持先行获批引入大湾区，缩短了中国低磷酸酯酶症患者使用全球创新药物的用药时差。 ->点击阅读原文，解锁完整双语新闻企业动态 No.1 / 复宏汉霖获甫康药业奈拉替尼中国商业化权益近日，复宏汉霖（2696.HK）与甫康（上海）健康科技有限责任公司（简称“甫康药业”）宣布达成战略合作。复宏汉霖获得甫康药业马来酸奈拉替尼片（简称“奈拉替尼”，商品名：汉奈佳）在中国的商业化独占权利以及在约定海外国家和地区的独家谈判和附条件许可权。奈拉替尼是甫康药业自主开发的首个产品，属于新型口服、强效、不可逆的小分子泛HER激酶抑制剂（TKI）。2024年6月，奈拉替尼获得国家药品监督管理局（NMPA）批准上市，用于人类表皮生长因子受体2（HER2）阳性的早期乳腺癌成年患者，在接受含曲妥珠单抗辅助治疗之后的强化辅助治疗。基于此次合作，复宏汉霖将于近期全力推动汉奈佳步入商业化进程，与旗下曲妥珠单抗生物类似药汉曲优实现转换治疗，助力降低HER2阳性早期乳腺癌患者术后5年和10年复发风险。 ->点击阅读原文，解锁完整双语新闻全球医疗情报领导者解锁隐藏在数据中的商业潜力关于 G B I ” 自从2002年成立以来，GBI始终以技术为驱动，为药企、器械及行业相关服务商提供贯穿生命周期的全球药品市场竞争数据、全球行业资讯、HCPs洞察、全国医疗器械数据等商业信息与洞察，助力企业在进行战略布局和决策时，脱颖而出。历经20余年的深耕细作GBI已成为95%以上跨国药企、国内头部药企、咨询与投资机构等医疗圈灯塔用户值得信赖的长期合作伙伴。联系我们投稿 | 发稿 | 媒体合作 ▶ zhangxinyue13@baidu.com 数据库 | 咨询服务 | 资讯追踪 ▶ 点击左下“阅读原文”完成表单填写点击阅读原文，解锁完整双语新闻

突破性疗法引进/卖出临床3期抗体药物偶联物临床结果

2024-08-21

·美通社

低磷酸酯酶症药Strensiq依托"港澳药械通"政策先行引入大湾区

为罹患这一罕见病的患者群体实现新药可及上海 2024年8月21日 /美通社/ -- 阿斯利康中国宣布，依托 "港澳药械通"政策，其罕见病领域创新药Strensiq（通用名：阿司福酶α注射液 / asfotase alfa）已获广东省药品监督管理局批准，正式引进大湾区药械通指定医疗机构，用于作为婴儿期发病的低磷酸酯酶症(hypophosphatasia, HPP)患者的长期酶替代疗法，以治疗该疾病的骨骼表现。此次大湾区先行获批认可了该药的临床应用先进性，内地患儿将无需出境便可在大湾区药械通指定医疗机构使用该药，缓解了中国患者亟需用药的难题。低磷酸酯酶症是一种极其罕见的遗传性渐进式代谢紊乱疾病，患者因缺少碱性磷酸酶(alkaline phosphatase, ALP)，骨矿化和骨质素生成受到影响，致使骨骼生长和其强度出现问题，并引发其他并发症，可能面临骨折、骨痛、行走和呼吸困难、癫痫等问题，甚至造终身负担，严重影响生存质量 [1] 。作为全球首个且唯一*获批用于治疗低磷酸酯酶症的酶替代疗法 [2] ，阿司福酶α注射液中的活性物质可以替代患者体内缺少的碱性磷酸酶，并作为长期酶替代治疗控制患者症状 [1] 。针对围产期、婴儿期或幼儿期发病低磷酸酯酶症的患者的四项前瞻性的开放标签临床试验，评估并证实了阿司福酶α注射液的临床疗效和安全性。经该药治疗的围产期/婴儿期发病的低磷酸酯酶症患者（37名）与历史对照的低磷酸酯酶症患者（48名）的总生存率对比的Kaplan-Meier分析发现，阿司福酶α注射液可明显改善患者生存率。研究显示，经阿司福酶α注射液治疗的患者1岁时生存率为95%，历史对照组患者生存率为42%；经阿司福酶α注射液治疗患者3.5岁时生存率为86%，历史对照组患者生存率为27%；经阿司福酶α注射液患者5岁时生存率为82%，而历史对照组患者生存率为27% [3] 。对于婴幼儿，阿司福酶α注射液有助于逆转矿质过少的症状 [4],[5],[6] 。在儿童患者中，阿司福酶α注射液可帮助患者实现低磷酸酯酶症相关佝偻病的完全或实质性治愈 [7] 。酶替代治疗是国际公认的针对低磷酸酯酶症病因的有效治疗方法，尤其对于年幼和症状明显的患者，可被作为首选治疗方式 [8] ，然而，此前相关药物的可及性一直是困扰患者的一大难题。此次阿司福酶α注射液在大湾区的率先获批，是国内低磷酸酯酶症治疗的重大突破，为国内患有遗传性低磷酸酯酶症的儿童患者带来了新的治疗希望。阿斯利康中国生物制药业务总经理，香港及澳门地区负责人林骁表示："深耕中国逾30年，阿斯利康深刻洞察并理解中国患者未被满足的需求。此次，阿司福酶α注射液得益于‘港澳药械通'政策的支持先行获批引入大湾区，缩短了中国低磷酸酯酶症患者使用全球创新药物的用药时差。未来，我们将继续携手各方合作伙伴，让更多全球领先、患者亟需的创新药物通过大湾区这一桥梁加速引进国内，惠及更多中国患者。" 阿斯利康中国副总裁，罕见病事业部负责人胡轶清表示："‘以患者为中心'，阿斯利康不仅积极投身并持续探索罕见病领域，还致力于联合多方为中国罕见病患者破解无药可用的困境，并为提升创新药物的可及性而努力。今天，我们非常高兴地看到阿司福酶α注射液在大湾区先行获批引入，让中国低磷酸酯酶症患者加速用上全球领先的药物。未来，阿斯利康也将持续高度重视低磷酸酯酶症及其他罕见病患者的未尽之需，多措并举，加速创新药品引入、可及，并联合各方力量支持建设健全中国罕见病诊疗的生态体系，让我们携手同行，点亮生命之光。" *截至2024年8月，来源于 www.fda.gov , www.ema.europa.eu , https://strensiq-hcp.com/ 声明：本资料涉及的药品和适应症尚未在中国内地获批，阿斯利康不推荐任何未被批准的药品使用。关于低磷酸酯酶症低磷酸酯酶症是一种遗传性、慢性、进行性和危及生命的代谢性疾病，会对患者身体多个系统产生破坏性影响，导致衰弱或危及生命的并发症 [9] 。低磷酸酯酶症的特征是碱性磷酸酶（ALP）活性低和骨矿化缺陷，可导致骨骼破坏和畸形及其他骨骼异常，以及全身并发症，如肌无力和呼吸衰竭，导致婴儿早逝 [4] ,[ 8] ,[10] 。关于阿斯利康阿斯利康（LSE/STO/Nasdaq: AZN）是一家科学至上的全球生物制药企业，专注于研发、生产及营销处方类药品，重点关注肿瘤、罕见病以及包括心血管肾脏及代谢、呼吸及免疫在内的生物制药等领域。阿斯利康全球总部位于英国剑桥，业务遍布世界100多个国家，创新药物惠及全球数百万患者。更多信息，请访问 www.astrazeneca.com 。关于阿斯利康中国阿斯利康自1993年进入中国以来，专注中国患者需求最迫切的治疗领域，包括肿瘤、心血管、肾脏、代谢、呼吸、消化、罕见病、疫苗抗体及自体免疫等，已将40多款创新药物带到中国。阿斯利康中国总部及全球研发中国中心位于上海，并在无锡、泰州、青岛分别建立全球生产供应基地，已向近70个全球市场输送优质药品。近年来，公司分别在北京、广州、杭州、成都、青岛设立区域总部。阿斯利康还携手合作伙伴，通过打造包括中国智慧健康创新中心（CCiC）、国际生命科学创新园（iCampus）、阿斯利康中金医疗产业基金在内的创新"三驾马车"，构建多元化的国际创新健康生态圈，共同促进区域经济以及大健康行业的长足发展。今天，中国已经发展成为阿斯利康全球第二大市场。 [1] Strensiq Hong Kong Prescribing Information (40mg), version Apr 2023 (Chinese) [2] Scott LJ. Asfotase Alfa: A Review in Paediatric-Onset Hypophosphatasia. Drugs. 2016 Feb;76(2):255-62. doi: 10.1007/s40265-015-0535-2. [3] Kaplan-Meier analysis of survival of patients with perinatal and infantile hypophosphatasia. Asfotase alfa–treated patients (studies ENB-002-08/ENB-003-08 and ENB-010-10) had a significantly better survival rate, calculated using the Kaplan-Meier product-limit estimate, compared with historical controls (study ENB-011-10; P .0001, Kaplan-Meier log-rank test). [4] Whyte MP, Greenberg CR, Salman NJ, et al. Enzyme-replacement therapy in life-threatening hypophosphatasia. N Engl J Med. 2012;366(10):904-913. [5] Whyte MP, Simmons JH, Moseley S, et al. Asfotase alfa for infants and young children with hypophosphatasia: 7 year outcomes of a single-arm, open-label, phase 2 extension trial. Lancet Diabetes Endocrinol. 2019;7(2):93-105. [6] Hofmann CE, Harmatz P, Vockley J, et al. Efficacy and safety of asfotase alfa in infants and young children with hypophosphatasia: a phase 2 open-label study. J Clin Endocrinol Metab. [7] Whyte MP, Madson KL, Phillips D, Reeves AL, McAlister WH, Yakimoski A, Mack KE, Hamilton K, Kagan K, Fujita KP, Thompson DD, Moseley S, Odrljin T, Rockman-Greenberg C. Asfotase alfa therapy for children with hypophosphatasia. JCI Insight. 2016 Jun 16;1(9):e85971. doi: 10.1172/jci.insight.85971 [8] 刘敏,赵耘,巩纯秀. 低磷酸酶血症研究新进展[J]. 中华儿科杂志,2019,57(11):899-903 [9] Rockman-Greenberg C. Hypophosphatasia. Pediatr Endocrinol Rev. 2013;10(suppl 2):380-388. [10] Fraser D. Hypophosphatasia. Am J Med. 1957;22(5):730-746.

临床结果临床研究

100 项与阿司福酶α 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10595	阿司福酶α	A16AB13	DB09105

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
婴儿低磷酸酯脢症	美国	Alexion Pharmaceuticals, Inc.	2015-10-23
低碱性磷酸酶血症	日本	Alexion Pharmaceuticals, Inc.	2015-07-03

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
儿童低磷酸酯脢症	临床2期	美国	Alexion Pharmaceuticals, Inc.	2016-06-06
儿童低磷酸酯脢症	临床2期	德国	Alexion Pharmaceuticals, Inc.	2016-06-06
遗传性热变性异形红细胞增多症	临床2期	美国	Alexion Pharmaceuticals, Inc.	2008-09-01
遗传性热变性异形红细胞增多症	临床2期	加拿大	Alexion Pharmaceuticals, Inc.	2008-09-01
遗传性热变性异形红细胞增多症	临床2期	阿拉伯联合酋长国	Alexion Pharmaceuticals, Inc.	2008-09-01
遗传性热变性异形红细胞增多症	临床2期	英国	Alexion Pharmaceuticals, Inc.	2008-09-01

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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


ESPE2023	N/A	低碱性磷酸酶血症 alkaline phosphatase (ALP) \| PEA \| B6 vitamin	-	Asfotase alfa	憲壓簾選廠蓋鹹糧艱齋(餘鏇獵網繭鹹艱膚窪廠) = 襯窪簾齋鏇繭獵網衊醖選艱鑰廠構蓋襯醖餘顧 (顧鬱觸鏇窪築網襯構鹽 ) 更多	-	2023-09-21
NCT01176266 (Pubmed \| J Clin Endocrinol Metab) 人工标引	临床2期	低碱性磷酸酶血症	69	Asfotase Alfa	夢觸鏇餘簾遞繭醖築築(醖餘簾壓構遞選網網醖) = 衊選蓋襯積獵膚構艱獵積鏇淵膚鬱積衊鬱蓋襯 (遞鑰積蓋糧觸壓觸觸鹽 ) 更多	积极	2019-07-01
NCT01205152 \| EUCTR2009-009369-32-GB (Pubmed) 人工标引	临床2期	低碱性磷酸酶血症	11	Asfotase alfa	夢範顧簾鹹糧築襯網觸(願糧鹽廠壓簾獵鹹淵齋) = 遞構衊鑰艱願鹹範鹽積窪簾膚網積壓艱醖淵鑰 (願遞餘積膚顧觸獵鹹襯 ) 更多	积极	2019-02-01
ESPE2018	N/A	低碱性磷酸酶血症 ALPL	-	Asfotase Alfa	鬱繭範淵鏇餘遞網顧憲(鹽鬱製淵鹹獵築衊遞壓) = The patient needed a wheelchair even for in-home movement, suffered from continuous skeletal pain, had undetectable ALP serum levels and extremely high plasma PLP (997 mcg/l) 構鏇蓋鹽積淵構糧鏇積 (蓋簾憲築範餘鏇衊鹽廠 )	-	2018-09-27
NCT02797821 (CTgov)	临床2期	儿童低磷酸酯脢症	27	Asfotase alfa (Asfotase Alfa 0.5 mg/kg Dose)	範範蓋衊壓壓願廠範簾(鑰選鏇窪獵膚鹽繭淵選) = 餘衊醖顧壓範齋夢壓襯築鹹艱鬱鬱淵憲選餘築 (築鑰製淵鑰醖範夢構淵, 獵齋願艱觸網積構觸繭 ~ 鹹糧築齋壓積鹽廠顧蓋) 更多	-	2018-07-10
NCT02797821 (CTgov)	临床2期	儿童低磷酸酯脢症	27	Asfotase alfa (Asfotase Alfa 2.0 mg/kg Dose)	範範蓋衊壓壓願廠範簾(鑰選鏇窪獵膚鹽繭淵選) = 鹽醖膚積醖築繭獵醖範築鹹艱鬱鬱淵憲選餘築 (築鑰製淵鑰醖範夢構淵, 膚選廠觸蓋糧醖憲糧獵 ~ 獵壓衊鑰艱獵鏇齋選糧) 更多	-	2018-07-10
NCT01176266 (ENB-010-10, CTgov)	临床2/3期	低碱性磷酸酶血症	69	asfotase alfa	膚鹹廠鏇範範鹹繭襯衊(襯觸範鏇繭築夢簾壓鏇) = 齋憲獵艱觸構觸築襯壓獵選顧夢繭築齋獵鹽鬱 (築選鏇繭齋壓艱糧製顧, 廠夢憲膚範網觸鬱鹹醖 ~ 衊淵積壓築鑰鬱夢衊醖) 更多	-	2018-02-26
NCT01205152 (CTgov)	临床2期	低碱性磷酸酶血症	10	asfotase alfa	遞鏇糧襯憲遞簾鏇鏇顧(夢糧膚範獵齋鹽構襯簾) = 選窪網築網廠繭膚鹹構艱簾網繭鹽膚糧鏇鏇艱 (餘鬱衊鬱製製醖齋網遞, 鹽餘範襯襯憲廠構廠膚 ~ 艱遞衊遞齋獵願鹽築鏇) 更多	-	2017-11-17
NCT01163149 (CTgov)	临床2期	低碱性磷酸酶血症	19	asfotase alfa	獵廠淵膚蓋鏇構選淵夢(襯鏇繭襯範窪餘鏇觸憲) = 積餘齋餘鹹餘廠繭蓋顧餘網構築範觸蓋餘鏇願 (夢鑰築齋範蓋鏇衊顧願, 遞鏇餘窪膚艱壓廠襯夢 ~ 願鑰餘製淵網獵餘築遞) 更多	-	2017-09-18
NCT01203826 (CTgov)	临床2期	低碱性磷酸酶血症	12	Asfotase Alfa (Asfotase Alfa Combined)	淵遞壓憲顧繭鑰夢網選(願獵繭製廠獵範積顧鹹) = 範範鑰艱夢網鹽積範膚繭選膚齋壓廠製積餘範 (積範憲積鏇範範範廠鏇, 膚願範選鏇觸構獵選製 ~ 簾齋艱夢鏇網獵鏇襯積)	-	2017-07-26
NCT01203826 (CTgov)	临床2期	低碱性磷酸酶血症	12	Asfotase Alfa (2 mg/kg Asfotase Alfa)	獵網觸鑰選糧衊範艱範(糧衊廠壓襯鹽蓋夢淵築) = 窪簾糧鑰膚遞鹽齋繭鹽夢憲鏇夢淵廠觸鏇選糧 (夢餘遞窪繭鹹網獵膚鏇, 範憲壓鹹積網觸繭鑰築 ~ 觸餘鏇構憲蓋積鬱膚艱) 更多	-	2017-07-26
NCT02531867 (CTgov)	临床4期	低碱性磷酸酶血症	13	asfotase alfa	餘廠壓顧觸鏇選鏇鏇選(觸構製鑰鹽鏇齋積鹹願) = 鹹鹽鹽窪選選選齋糧艱積選鹹構鑰窪繭選憲鑰 (獵簾壓醖膚製鏇遞鏇遞, 糧膚衊艱範繭築構鹽蓋 ~ 夢艱製製願蓋壓衊淵鹽) 更多	-	2017-05-08