A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 12 Months of Age and Older

The purpose of the study is to evaluate the long-term safety, tolerability, efficacy, and pharmacodynamics (PD) of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA).

开始日期2024-10-01

申办/合作机构

Vertex Pharmaceuticals, Inc.

ACTRN12624000146594 / Not yet recruitingN/AIIT

Assessment of regional lung ventilation via X-ray velocimetry (XV) imaging following thecommencement of ETI (elexacaftor/tezacaftor/ivacaftor) treatment in children with cystic fibrosis

开始日期2024-03-01

申办/合作机构

WomenS & ChildrenS Hospital

[+1]

NCT06331000 / Not yet recruitingN/A

DIATRIM : Effect of One Year Elexacaftor-tezacaftor-ivacaftor Treatment on Glucose Tolerance Abnormalities in Adult Patients With Cystic Fibrosis.

The development of CFTR (cystic fibrosis conductance transmembrane regulator) modulators for people with cystic fibrosis (pwCF) and eligible for these treatments is a true therapeutic revolution. The major beneficial effect of CFTR modulators (CFTRm) on pulmonary function and the reduction of pulmonary exacerbations should have a considerable impact on the quality of life and patient's life expectancy. Data on the impact of CFTRm on glucose tolerance abnormalities are still very fragmentary. The investigators can think that their use, earlier and earlier in the history of the disease, will transform the evolutionary trajectories of patients on the respiratory, nutritional and metabolic levels.
Diabetes represents a major challenge in the management of pwCF because it is a factor in morbidity and mortality at all stages of the disease, from children to patients with terminal respiratory failure requiring lung transplantation. Early abnormalities in glucose tolerance observed in childhood, before the stage of diabetes, are also associated with poor pulmonary and nutritional outcomes. Experimental data suggest a positive effect of CFTRm on insulin secretion. However, investigators do not currently know the impact of CFTRm in patients with very early glucose disorders or at the stage of diabetes treated with insulin. Recently continuous glucose measurement (CGM) devices represent very effective tools for assessing abnormalities in glucose tolerance before the stage of diabetes and for monitoring patients treated with insulin.

开始日期2024-03-01

申办/合作机构

Les Hopitaux Universitaires de Strasbourg

100 项与 Elexacaftor/Ivacaftor/Tezacaftor 相关的临床结果

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100 项与 Elexacaftor/Ivacaftor/Tezacaftor 相关的转化医学

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100 项与 Elexacaftor/Ivacaftor/Tezacaftor 相关的专利（医药）

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项与 Elexacaftor/Ivacaftor/Tezacaftor 相关的文献（医药）

2024-02-01·American journal of respiratory and critical care medicine

Elexacaftor/Tezacaftor/Ivacaftor Treatment and Depression-related Events

Review

作者: Tian, Simon ; Volkova, Nataliya ; Ramsey, Bonnie ; Tullis, Elizabeth ; Correll, Christoph U ; Ahluwalia, Neil ; Mall, Marcus A ; McKone, Edward ; Waltz, David ; Chu, Chenghao ; Taylor-Cousar, Jennifer L ; DeMaso, David R

Rationale: Elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) has been shown to be safe and efficacious in people with cystic fibrosis (pwCF) aged 2 years and older with at least one F508del-CFTR allele or more. After U.S. approval in 2019, reports emerged of depression-related adverse events in pwCF treated with ELX/TEZ/IVA. Objectives: To review available evidence on depression-related events in pwCF treated with ELX/TEZ/IVA in the context of background epidemiology in pwCF. Methods: Safety data from 14 ELX/TEZ/IVA clinical trials and 10 trials of CF transmembrane conductance regulator (CFTR) modulators in which placebo was administered, along with data from CF registries in the United States and Germany and cumulative postmarketing adverse event data from 61,499 pwCF who initiated ELX/TEZ/IVA after initial approval in the United States (October 2019) through October 2022, were reviewed and used to calculate exposure-adjusted rates of depression-related adverse events and prevalence of depression. In addition, a scientific literature review was conducted to identify ELX/TEZ/IVA publications reporting depression-related events or changes in depressive symptoms after treatment initiation. Measurements and Main Results: In clinical trials, the exposure-adjusted rate of any depression-related adverse event was 3.32/100 person years (PY) in the pooled ELX/TEZ/IVA group (n = 1,711) and 3.24/100 PY in the pooled placebo group (n = 1,369). The exposure-adjusted rates of suicidal ideation and suicide attempt were also similar between the pooled ELX/TEZ/IVA group and pooled placebo group (ideation: 0.23/100 PY vs. 0.28/100 PY; attempt: 0.08/100 PY vs. 0.14/100 PY). In the postmarketing setting, the exposure-adjusted reporting rates of depression-related events were low in context of the background prevalence in pwCF (all depression-related events: 1.29/PY; suicidal ideation: 0.12/100 PY; and suicide attempt: 0.05/100 PY). Assessments of individual case reports were confounded by preexisting mental health conditions, intercurrent psychosocial stressors (including coronavirus disease [COVID-19] lockdowns), and the heterogeneous and fluctuating nature of depression. Data from CF registries in the United States and Germany showed that patterns of depression prevalence in pwCF exposed to ELX/TEZ/IVA did not change after treatment initiation. Published studies utilizing the nine-item Patient Health Questionnaire did not show evidence of worsening depression symptoms in pwCF treated with ELX/TEZ/IVA. Conclusions: Our review of data from clinical trials, postmarketing reports, an ongoing registry-based ELX/TEZ/IVA postauthorization safety study, and peer-reviewed literature suggests that depression symptoms and depression-related events reported in pwCF treated with ELX/TEZ/IVA are generally consistent with background epidemiology of these events in the CF population and do not suggest a causal relationship with ELX/TEZ/IVA treatment.

2024-02-01·Molecular biology of the cell

Small-molecule correctors divert CFTR-F508del from ERAD by stabilizing sequential folding states

Article

作者: Wąchalska, Magda ; Deol, Kirandeep K ; Riepe, Celeste ; Kopito, Ron R ; Olzmann, James A ; Porteus, Matthew H ; Amaya, Anais K

Over 80% of people with cystic fibrosis (CF) carry the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR), a chloride ion channel at the apical plasma membrane (PM) of epithelial cells. F508del impairs CFTR folding causing it to be destroyed by endoplasmic reticulum associated degradation (ERAD). Small-molecule correctors, which act as pharmacological chaperones to divert CFTR-F508del from ERAD, are the primary strategy for treating CF, yet corrector development continues with only a rudimentary understanding of how ERAD targets CFTR-F508del. We conducted genome-wide CRISPR/Cas9 knockout screens to systematically identify the molecular machinery that underlies CFTR-F508del ERAD. Although the ER-resident ubiquitin ligase, RNF5 was the top E3 hit, knocking out RNF5 only modestly reduced CFTR-F508del degradation. Sublibrary screens in an RNF5 knockout background identified RNF185 as a redundant ligase and demonstrated that CFTR-F508del ERAD is robust. Gene-drug interaction experiments illustrated that correctors tezacaftor (VX-661) and elexacaftor (VX-445) stabilize sequential, RNF5-resistant folding states. We propose that binding of correctors to nascent CFTR-F508del alters its folding landscape by stabilizing folding states that are not substrates for RNF5-mediated ubiquitylation.

2024-01-01·SAGE open medicine

Elexacaftor–tezacaftor–ivacaftor for cystic fibrosis with Phe508del mutation: Evidence from randomized controlled trials

Article

作者: He, Rong ; Deng, Zehui ; Yu, Bin ; Lin, Fei

Objective::

This study aimed to conduct a systematic review and meta-analysis of randomized controlled trials to evaluate the effects of elexacaftor–tezacaftor–ivacaftor (ELX-TEZ-IVA) on patients with cystic fibrosis (CF).

Methods::

A systematic search was performed in PubMed, Embase, and the Cochrane Library from inception to August 1, 2022. Meta-analysis was conducted using Review Manager 5.3 software.

Results::

Six studies comprising seven reports involving a total of 1125 CF patients were included. The meta-analyses indicated that ELX-TEZ-IVA significantly improved the percentage predicted forced expiratory volume in 1 s (ppFEV1) by 10.29% (95% confidence interval (CI) (6.44, 14.14), p < 0.00001) and the CF questionnaire-revised respiratory domain (CFQ-R RD) by 14.59 points (95% CI (9.25, 19.94), p < 0.00001) compared to placebo, ivacaftor (IVA), or tezacaftor–ivacaftor (TEZ-IVA). In addition, the ELX-TEZ-IVA group showed significantly lower sweat chloride concentrations by 40.30 mmol/L (95% CI (−49.85, −30.74), p < 0.00001). However, the incidence of adverse events in the ELX-TEZ-IVA group was slightly higher than that in the placebo, IVA, or TEZ-IVA groups.

Conclusion::

ELX-TEZ-IVA demonstrated efficacy in improving ppFEV1, CFQ-R RD, and sweat chloride concentrations in patients with CF. However, caution should be exercised regarding the incidence of AEs, particularly mild and moderate ones.

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项与 Elexacaftor/Ivacaftor/Tezacaftor 相关的新闻（医药）

2024-06-18

·生物探索

Science丨孙晔僡等开发肺部靶向基因编辑

引言尽管基因编辑作为某些遗传疾病的干预措施开始在临床上获得接受，但仍存在一些困难。挑战包括直接在体内而不是在体外有效地进行编辑细胞，以及编辑干细胞中的致病基因的能力对于确保分化细胞中的修正 DNA 库，从而提供持久的治疗效果至关重要。 2020年，Siegwart团队开发了一种名为器官选择性递送mRNA和CRISPR-Cas基因标记的方法Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing，发表于Nature Nanotechnology（共同一作为程强和魏妥博士）。这种方法在传统的四组分脂质纳米颗粒（LNPs）中添加了第五组分，使其从肝脏重新靶向到肝外器官（包括肺部）以传递mRNA和基因编辑分子。尽管此项研究证明了SORT可以将基因编辑工具递送到肺部，但是否能成功编辑肺干细胞仍然未知。 2024年6月13日，在Daniel Siegwart教授带领下，德克萨斯大学西南医学中心团队联合凯斯西储大学团队和ReCode Therapeutic合作在Science杂志上发表了题为In vivo editing of lung stem cells for durable gene correction in mice的研究论文。报道了肺部选择性脂质纳米颗粒（Lung SORT LNPs）成功在囊性纤维化小鼠模型的肺部实现对致病基因的修正。此项研究可能为囊性纤维化和其他遗传性肺病患者带来希望。与常见的针对肺部递送的吸入法（由于给药方便，适合蛋白质替代疗法）相比，静脉注射的 SORT LNPs 通过避免疾病相关的粘膜屏障，并且由于肺内皮床与 LNPs 的紧密接触，可能更有机会进入肺基底干细胞。 Siegwart团队最初使用经过基因改造的健康小鼠进行实验，使得经过基因编辑的细胞会发出红光。然后，他们通过静脉注射递送包含靶向肺部的基因编辑工具的 SORT LNPs。肺部持续的红光表明带有编辑基因的细胞至少存在了22个月。进一步的研究表明，超过70%的小鼠肺干细胞被基因编辑了。这种治疗方法对大约10%的囊性纤维化患者尤其有益，这些患者的疾病是由CFTR基因罕见突变或一种称为无义突变（例如 R553X）的特定突变类型引起的。Trikafta 无法治疗他们的疾病，而 Trikafta (Vertex)是目前囊性纤维化的黄金标准疗法。在另一项实验中，研究人员使用 SORT LNP对携带 R553X/F508del 突变的囊性纤维化患者分离出的肺细胞进行了碱基编辑，这些细胞在气液界面上培养，可以模拟肺部上皮细胞结构和功能，被认为是预测临床治疗效果的有力指标。实验表明，针对R553X突变基因的碱基编辑成功恢复了约53%的囊状纤维化跨膜转导调节子(CFTR) 的功能，这一结果与接受正对F508del突变的 Trikafta 治疗组在该实验中的结果相当。接下来，研究人员使用携带 R553X 突变的小鼠模型进行实验。尽管囊性纤维化的小鼠模型不会表现出人类囊性纤维化的呼吸系统症状，但它们确实与健康小鼠相比具有明显的生理差异。实验表明，碱基编辑在这种疾病模型中也取得了成功。这些结果表明，基于器官选择性的脂质纳米颗粒（SORT）的基因编辑疗法有望长期治疗囊性纤维化和其他遗传性肺病。后续更多的研究需要在具有囊性纤维化症状的动物模型中验证这种方法，并确保这种潜在疗法的安全性。 Science同期发表了博士生Mattijs Bulcaen与Marianne S. Carlon教授 (KU Leuven) 题为Gene editing flows to the lungs的专评，认为该研究为静脉注射靶向肺部干细胞有克服肺部屏障的潜力用于囊性纤维化基因疗法。参考文献 science.org/doi/10.1126/science.adk9428 责编|探索君排版|探索君文章来源|“BioArt” End 往期精选围观一文读透细胞死亡(Cell Death) | 24年Cell重磅综述（长文收藏版）热文 Cell | 是什么决定了细胞的大小？热文 Nature | 2024年值得关注的七项技术热文 Nature | 自身免疫性疾病能被治愈吗？科学家们终于看到了希望热文 CRISPR技术进化史 | 24年Cell综述

基因疗法临床结果信使RNA

2024-06-18

·BioSpace

Enlaza Therapeutics Appoints Jeffrey Leiden, M.D., Ph.D., as a Key Advisor

Executive Chairperson and former CEO of Vertex Pharmaceuticals to help guide the development of a high-value pipeline of first-in-class covalent protein drugs from Enlaza’s War-Lock™ platform LA JOLLA, Calif.--(BUSINESS WIRE)-- Enlaza Therapeutics, the first covalent biologic platform company, today announced the appointment of Dr. Jeffrey Leiden, Executive Chairperson of Vertex Pharmaceuticals, as a key advisor to the company. Dr. Leiden has more than 40 years of experience in the biopharma industry developing life-improving medicines and building companies that transform the industry. “Jeff is one of the most accomplished and admired leaders in our industry. He has dedicated his career to developing important first-in-class medicines from novel breakthrough technologies that have helped millions of people with serious diseases,” said Sergio Duron, Ph.D., CEO of Enlaza Therapeutics. “With our War-Lock platform, we can create covalent protein drugs with the potential to improve treatments for many diseases. Jeff’s guidance will be invaluable as we establish a diverse pipeline and advance our lead assets toward clinical development.” "While covalency has been a long-standing strategy in small molecule drug development, Enlaza is the first to incorporate covalency into biologics, unlocking a new class of protein therapeutics," said Dr. Leiden. "I look forward to working closely with the Enlaza team to realize the full potential of this exciting new drug discovery and development platform and help create meaningful new medicines for patients.” As CEO and President of Vertex from 2012 through 2020, Dr. Leiden built a robust pipeline of drug candidates, with a particular focus on therapies that cure or modify the course of disease. Under his leadership, Vertex delivered the first and only medicines to treat the underlying cause of cystic fibrosis for patients with certain forms of the disease, including KALYDECO®, ORKAMBI®, SYMDEKO® and TRIKAFTA®. In 2015, he established a collaboration with CRISPR Therapeutics to develop transformative therapies for sickle cell disease (SCD), beta thalassemia (TDT) and other diseases; and in 2023, CASGEVY™ was approved as the first gene-editing therapy in the world for SCD and TDT. In 2019, he led the Vertex acquisition of Semma Therapeutics to develop a clinical program to investigate what may be the first potentially curative stem cell-based therapy for type 1 diabetes. Prior to joining Vertex, Dr. Leiden was a managing director at Clarus Ventures, a life sciences venture capital firm, from 2006 through 2012. Dr. Leiden was President, Chief Operating Officer and Chief Scientific Officer of Abbott Laboratories, Pharmaceuticals Products Group, and a member of the board of directors of Abbott Laboratories from 2000 to 2006. At Abbott he led the development and commercialization of HUMIRA®. From 1987 to 2000, Dr. Leiden held several academic appointments, including the Rawson Professor of Medicine and Pathology and Chief of Cardiology and Director of the Cardiovascular Research Institute at the University of Chicago, the Elkan R. Blout Professor of Biological Sciences at the Harvard School of Public Health, and Professor of Medicine at Harvard Medical School. He is an elected member of both the American Academy of Arts and Sciences and the National Academy of Medicine. About the War-Lock™ Platform Enlaza’s covalent biologic platform, called War-Lock, creates highly specific therapeutic warheads that covalently bind to drug targets of interest. This white-space technology enables, for the first time, a covalent-acting protein drug that retains the selectivity of small-format biologics. Protein drugs produced by the platform can be modified to incorporate various payloads and achieve specific delivery to target tissues with high fidelity. About Enlaza Therapeutics Enlaza Therapeutics is pioneering the field of covalent biologics. This novel class of protein therapeutics has the potential to delivers more efficacious and safer treatments for patients. The company's proprietary War-Lock™ platform leverages leading synthetic biology technology for site-specific covalent coupling driven by drug binding. Enlaza is building a pipeline of first-in-class covalent biologics. For more information, please visit .

高管变更

2024-06-10

·赛柏蓝

百亿畅销药Top10（附名单）

编者按：本文来自药智网，作者粽哥2025；赛柏蓝授权转载，编辑凯西 2023年，全球畅销药的竞争日趋白热化，催生了10款销售额突破百亿美元的“超级重磅炸弹”药物。 01 神仙打架全球10款百亿药物 2023年全球畅销药榜单的竞争，可谓是“神仙打架”，排名前10畅销药的销售额均超过了100亿美元。具体来看，跻身百亿美元俱乐部的“超级重磅炸弹”，分别为Keytruda（K药）、司美格鲁肽、Humira（修美乐）、Eliquis（阿哌沙班）、Biktarvy、Dupixent(度普利尤单抗)、新冠疫苗Comirnaty、Stelara(乌司奴单抗)、Jardiance(恩格列净)、Opdivo(O药)。此外，2023年还是医药史上诞生“超级重磅炸弹”药物最多的年份，极具里程碑意义。有市场人士认为，药品市场已经迈入了“百亿美元时代”。数据来源：根据各家企业年报整理（单位：亿美元）；注：数据皆为作者手动整理，如有疏漏，欢迎留言指正从整体上看，这10款百亿药物主要集中在肿瘤、自免和代谢等疾病领域。肿瘤领域，包括K药、BMS/小野制药的Opdivo（O药），其中O药销售额达到100.31亿美元，首次跻身百亿美元俱乐部；自免领域，包括修美乐、赛诺菲/再生元的度普利尤单抗、强生的乌司奴单抗，后两者均实现两位数同比增长；代谢领域，包括诺和诺德的司美格鲁肽、BI/礼来的Jardiance（恩格列净），也实现两位数增长。由此可见，唯有具有广泛市场需求的疾病领域，方能成为孕育“大药”的肥沃土壤。例如，司美格鲁肽不仅获批治疗糖尿病，还拓展至肥胖、心血管风险等适应症；恩格列净除针对糖尿病外，还拓展至心衰、慢性肾病。而且，两款药物都有进军患者人数庞大的心血管疾病领域。类似地，在心血管治疗领域内，老牌抗凝血药物Eliquis（阿哌沙班）同样以超百亿美元的销售额跻身“超级重磅炸弹”之列。全球首个“药王”Lipitor（立普妥），更是心血管领域的超级“销冠”，自上市以来累计销量超过1000亿美元。抗病毒领域的Biktarvy，是吉利德研发的HIV治疗药物，2023年也以118.5亿美元销售额跻身百亿美元俱乐部。值得一提的是，默沙东K药以250亿美元摘下了2023年全球“药王”宝座，击败了蝉联10年“药王”的Humira（修美乐）。 K药之所以能登顶全球“药王”，一方面在于获批了众多适应症，以及默沙东打造了强大的全球销售网络；二是由于竞品修美乐受到专利悬崖的冲击，导致销售额大幅下滑32%至144亿美元。然而，值得注意的是，K药的专利保护将在2028年到期。为延长其生命周期，默沙东豪掷重金引进了第一三共与科伦博泰的多款ADC资产，以研发“PD-1+ADC”的联合治疗方案，并同步进行其他联合疗法的临床试验探索。有分析师预测，K药的销售额有望于2027年突破300亿美元大关。 02 水大鱼大潜在爆款在路上若将“水大鱼大”的市场环境视作孕育百亿级药物的坚实基础，那么获得广泛适应症的批准，则无疑是延长药物生命周期的关键所在。销售额超过百亿美元的“超级重磅炸弹”，多数都获批5种以上适应症，有些甚至超过10种。例如，上一任“药王”修美乐可用于治疗超过10种自免疾病，使其连续11年营收超过百亿美元；现任“药王”K药和O药更是覆盖了20种左右不同癌种，包括度普利尤单抗、乌司奴单抗目前也已经获批治疗5种左右自免疾病。诺和诺德的司美格鲁肽，能成为下一任全球“药王”候选药物，也与广谱适应症密切相关。司美格鲁肽不仅在降糖这一存量市场遥遥领先，还开辟了减重、降低心血管风险等增量市场，而且还在探索治疗非酒精性脂肪肝（NASH/MASH）、慢性肾病（CKD）、外周动脉疾病、阿尔兹海默症和心衰等各种疾病。 2023年，司美格鲁肽实现销售额1458.11亿丹麦克朗（约合212亿美元），同比增长约89%，其中Ozempic（注射用降糖药）、Rybelsus（口服降糖药）、Wegovy（注射用减重药）分别实现138.4亿美元、27.1亿美元、45.3亿美元，同比增长60%、66%、407%。快速增长的销售增速叠加后续拓展新适应症，司美格鲁肽大概率将超越K药成为新一代全球“药王”。图片来源：国泰君安证券研报值得一提的是，还有不少药物有潜力跻身百亿美元俱乐部。 2023年，强生的Darzalex（达雷妥尤单抗）实现销售额97.44亿美元，同比增长22.2%；Vertex（福泰制药）的囊性纤维化（CF）三联疗法Trikafta/Kaftrio销售额为89.45亿美元，同比增长16.4%；默沙东的两款HPV疫苗Gardasil/Gardasil9销售额达88.86亿美元，同比增长29%。作为盛产“大药”的自免领域，艾伯维的Skyrizi（瑞莎珠单抗）销售相当强劲，2023年实现销售额77.63%，同比增长达50.3%，被市场称为修美乐的“继任者”，目前已获批13项适应症，还有10多项在研。2024年一季度，Skyrizi销售额同比增长48%至20.08亿美元，艾伯维预期今年销售额将达到107亿美元。另外，再生元/拜耳合作开发的Eylea（阿柏西普眼内注射溶液），2023年实现销售额93.81亿美元，不过同比下滑3%，今年一季度也下滑了1%，最终能否跻身百亿美元俱乐部，还有待观察。 03 结语总体来看，“超级重磅炸弹”药物不仅能为药企带来巨大的现金流，还是其开疆扩土和制定战略规划的基石。例如，艾伯维通过修美乐和自免“新星”Skyrizi捍卫了自免领域王者地位；默沙东以K药为基石，进军ADC领域，从而牢固肿瘤业务管线；深耕糖尿病领域的诺和诺德，也借助司美格鲁肽拓展至糖尿病以外的更多疾病领域。值得一提的是，原本只是“小而美”的Vertex（福泰制药），如今正凭借潜在百亿美元药物Trikafta/Kaftrio，从罕见病囊性纤维化领域，将管线拓展至糖尿病、镰状细胞病、β地中海贫血、疼痛和肾脏自免等疾病领域，俨然下一个跨国制药巨头的架势，未来可期。参考资料：1.各家公司的财报、公告、官微；2.国泰君安证券研报 END 内容沟通：郑瑶（13810174402）医药代表交流群扫描下方二维码加入银发经济市场机遇交流群扫描下方二维码加入左下角「关注账号」，右下角「在看」，防止失联

抗体药物偶联物疫苗

100 项与 Elexacaftor/Ivacaftor/Tezacaftor 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	R07AX	-

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
囊性纤维化	美国	Vertex Pharmaceuticals, Inc.	2019-10-21

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
咳嗽	临床3期	澳大利亚	Vertex Pharmaceuticals, Inc.	2021-10-12
咳嗽	临床3期	比利时	Vertex Pharmaceuticals, Inc.	2021-10-12
咳嗽	临床3期	加拿大	Vertex Pharmaceuticals, Inc.	2021-10-12
咳嗽	临床3期	西班牙	Vertex Pharmaceuticals, Inc.	2021-10-12
葡萄糖代谢紊乱	临床3期	澳大利亚	Vertex Pharmaceuticals, Inc.	2021-01-15
葡萄糖代谢紊乱	临床3期	比利时	Vertex Pharmaceuticals, Inc.	2021-01-15
葡萄糖代谢紊乱	临床3期	捷克	Vertex Pharmaceuticals, Inc.	2021-01-15
葡萄糖代谢紊乱	临床3期	法国	Vertex Pharmaceuticals, Inc.	2021-01-15
葡萄糖代谢紊乱	临床3期	意大利	Vertex Pharmaceuticals, Inc.	2021-01-15
葡萄糖代谢紊乱	临床3期	荷兰	Vertex Pharmaceuticals, Inc.	2021-01-15

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临床结果

适应症

分期

评价

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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


NCT03525574 (CTgov)	临床3期	囊性纤维化	507	IVA+ELX/TEZ/IVA	鹹網製築夢齋餘淵簾壓(膚餘糧糧簾淵繭獵醖鑰) = 憲遞積膚衊繭淵蓋齋糧構艱夢簾壓齋簾衊範築 (壓遞願顧餘淵醖繭遞願, 遞鹹衊鏇醖廠鑰糧齋觸 ~ 衊繭膚衊醖齋鏇遞網鬱) 更多	-	2024-05-08
NCT04545515 (CTgov)	临床3期	囊性纤维化	120	IVA+ELX/TEZ/IVA	蓋艱構繭餘繭衊鏇範醖(願鏇衊壓憲鏇獵遞製壓) = 顧觸窪獵廠蓋範範蓋壓糧淵襯遞糧鹽選憲鹹積 (鏇憲選網獵網衊構繭選, 繭願遞糧艱壓壓顧餘遞 ~ 築壓鬱願糧醖製衊窪衊) 更多	-	2024-05-08
NCT04058366 (CTgov)	临床3期	囊性纤维化	251	IVA+ELX/TEZ/IVA	獵繭選醖願淵顧鬱憲遞(鑰積憲鏇憲範鑰鑰選餘) = 襯壓餘夢觸積範構壓簾繭鹽鑰襯願繭鹽選顧壓 (蓋顧鹹膚製築網襯醖製, 淵鹽壓繭鏇襯窪願膚壓 ~ 襯廠獵鏇淵構夢鑰窪選) 更多	-	2024-01-16
AASLD2023	N/A	囊性纤维化	154	Elexacaftor/tezacaftor/ivacaftor	夢憲襯築網構選鹽蓋選(遞廠醖鬱獵餘襯膚範齋) = 鏇齋鑰觸顧選願繭壓選願鑰繭鹹築獵築顧齋餘 (窪蓋蓋繭衊壓齋顧膚醖 ) 更多	-	2023-11-10
NCT04969224 (CTgov)	临床3期	囊性纤维化 \| 咳嗽	82	IVA+ELX/TEZ/IVA	網蓋鬱夢鬱鹽鑰積艱淵(衊淵構衊壓繭蓋築蓋艱) = 遞願選壓鏇糧鏇願衊願鹽網選餘範簾衊簾醖憲 (構選顧網壓糧顧鹽鬱醖, 鹹構壓淵窪鑰鏇廠遞餘 ~ 簾繭積積積顧製選鏇選) 更多	-	2023-10-03
NCT04599465 (CTgov)	临床3期	囊性纤维化 \| 葡萄糖代谢紊乱	69	IVA+ELX/TEZ/IVA	簾觸艱鹹鬱壓蓋願積簾(蓋淵廠遞觸膚遞構齋糧) = 鏇簾壓鏇餘積範築願蓋膚繭窪壓廠糧選廠淵鏇 (齋醖餘艱淵醖積糧廠壓, 齋襯觸蓋範鏇餘餘艱鹹 ~ 夢衊廠觸獵網齋觸窪窪) 更多	-	2023-08-03
NCT04043806 (CTgov)	临床3期	囊性纤维化	458	IVA+ELX/TEZ/IVA	鑰觸鹽構膚鹹齋選製獵(遞鹹構糧鏇壓膚廠鹽齋) = 願製鑰淵網簾蓋襯遞衊廠窪顧淵鏇鹽廠糧網構 (鑰鑰製鏇鏇廠顧範遞繭, 廠窪範鑰網觸觸糧積觸 ~ 艱鑰憲選構鑰積艱願餘) 更多	-	2023-07-06
ADA2023	N/A	囊性纤维化相关糖尿病	24	Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy	鏇積夢蓋鹽遞觸糧選壓(獵網窪衊鹹鑰廠簾範網) = 壓遞範齋糧鹽醖襯範製獵築鬱齋窪淵積廠廠襯 (壓觸膚餘蓋壓獵積襯鬱 ) 更多	-	2023-06-20
FDA 人工标引	N/A	囊性纤维化 F508del Mutation	107	TRIKAFTA	壓襯顧壓餘醖衊範繭繭(鹽構鏇積構廠襯餘鏇蓋): Difference = 10.0 (95% CI, 7.4 ~ 12.6), P-Value = <0.0001 更多	积极	2023-04-26
FDA 人工标引	N/A	囊性纤维化 F508del Mutation	107	Tezacaftor/Ivacaftor		积极	2023-04-26
FDA 人工标引	N/A	囊性纤维化 F508del Mutation	403	TRIKAFTA	鹽繭夢顧醖襯網淵鹽窪(遞蓋鏇壓築網餘餘壓鹹): Difference = 13.8 (95% CI, 12.1 ~ 15.4), P-Value = <0.0001 更多	积极	2023-04-26
FDA 人工标引	N/A	囊性纤维化 F508del Mutation	403	Placebo		积极	2023-04-26