BAX-817(BAX-817) - 药物靶点：tissue factor_在研适应症：血友病_专利_临床

概要

基本信息

药物类型

重组凝血因子

别名

Recombinant factor VIIa、Recombinant Factor VIIa BI、rFVIIa

+ [2]

靶点

tissue factor

作用方式

抑制剂

作用机制

tissue factor抑制剂(组织因子抑制剂)

治疗领域

在研适应症

非在研适应症

原研机构

在研机构

Baxter BioScience Corp.

非在研机构

Baxalta, Inc.

权益机构-

最高研发阶段临床3期

首次获批日期-

最高研发阶段(中国)-

特殊审评-

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关联

9

项与 BAX-817 相关的临床试验

NCT04563520

/ Recruiting临床3期IIT

aPCC and Emicizumab Safety Study in Congenital Hemophilia A Patients With Inhibitors (SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis)

The purpose of the aPCC-emicizumab safety study is to investigate the hemostatic efficacy as measured by thrombin generation, of a low personalized dose of aPCC (FEIBA) in children and adults with hemophilia A and inhibitors on emicizumab prophylaxis.

开始日期2025-06-01

申办/合作机构

Emory University

[+1]

PACTR201901535105039

/ CompletedN/AIIT

Prospective study comparing between the effects of fibrinogen concentrate, and recombinant activated factor VII on reduction of blood products transfusion requirements in elective coronary artery bypass surgery

开始日期2017-11-01

申办/合作机构

Ain Shams University

NCT01757405

/ Completed临床3期

A PHASE 3, PROSPECTIVE, OPEN-LABEL, RANDOMIZED STUDY TO EVALUATE SAFETY AND EFFICACY OF RECOMBINANT ACTIVATED FVII BI (rFVIIa BI) IN THE TREATMENT OF ACUTE BLEEDING EPISODES PER AN ON-DEMAND REGIMEN IN PATIENTS WITH HEMOPHILIA A OR B WITH INHIBITORS

The purpose of the study is to determine the efficacy and safety of rFVIIa BI as part of a six-month on-demand treatment regimen in hemophilia A or B subjects with inhibitors.

开始日期2013-02-20

申办/合作机构

Baxalta, Inc.

100 项与 BAX-817 相关的临床结果

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100 项与 BAX-817 相关的转化医学

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100 项与 BAX-817 相关的专利（医药）

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304

项与 BAX-817 相关的文献（医药）

2024-12-29·Cureus Journal of Medical Science

Congenital Factor VII Deficiency: A Case Study of Four Family Members

Article

作者: Chakour, Mohamed ; Yahyaoui, Hicham ; Ghouzraf, Smail

Congenital factor VII (FVII) deficiency is a rare genetic disorder with autosomal recessive inheritance, characterized by molecular and clinical heterogeneity. This article reports four Moroccan cases of FVII deficiency within the same family, two of which were associated with Gilbert's syndrome. The index case was a 15-year-old girl with a history of menorrhagia and jaundice. Upon examination, she presented with indirect hyperbilirubinemia, iron deficiency anemia, and a prolonged prothrombin time (PT). FVII deficiency was confirmed with a factor VII level of 45%. Her mother, a 30-year-old woman with sarcoidosis and a history of postpartum hemorrhage, was found to have jaundice and an FVII deficiency at 42%. The brother of the index case, an eight-year-old boy who was asymptomatic, was discovered to have FVII deficiency during family screening, with a level of 41%. Similarly, the six-year-old sister, also asymptomatic, had an FVII level of 33%. The prevalence of homozygous FVII deficiency is rare, and the risk is increased in consanguineous marriages. Clinical presentations vary widely, ranging from asymptomatic cases to severe bleeding episodes. Diagnosis is based on a prolonged PT with a normal activated partial thromboplastin time (aPTT) and is confirmed through FVII assays. Severe cases may require prophylactic treatment, and recombinant activated FVII (rFVIIa) is the recommended therapy for bleeding episodes. In conclusion, FVII deficiency is the most common of the rare coagulation factor deficiencies. This study explores familial congenital factor VII deficiency, characterized by varied presentations from asymptomatic to mild hemorrhagic symptoms. Early diagnosis and family screening are essential. Management includes symptomatic treatment, tranexamic acid for menorrhagia, and fresh frozen plasma or recombinant factor VIIa for bleeding episodes.

2024-11-01·HAEMOPHILIA

Real‐world effectiveness of eptacog beta in patients with haemophilia and inhibitors: A multi‐institutional case series

Article

作者: Tran, Duc Q. ; Batsuli, Glaivy ; Young, Guy ; Acharya, Suchitra ; Sidonio, Robert ; Youkhana, Kimberley ; Abajas, Yasmina ; Khan, Osman ; Dvorak, Andrea ; Recht, Michael

Abstract:

Introduction:

The management of bleeding events (BEs) in haemophilia A (HA) and B (HB) patients with inhibitors necessitates the use of bypassing agents. The recombinant factor VIIa bypassing agent eptacog beta has demonstrated efficacy at treating BEs and managing perioperative bleeding in adults in phase three clinical studies.

Aim:

To provide real‐world descriptions of eptacog beta use for BE treatment in patients on emicizumab or eptacog beta prophylaxis.

Methods:

This is a retrospective case series of 14 patients who received eptacog beta at seven haemophilia treatment centres, with HA (n = 11) or HB (n = 3) and inhibitors or anaphylaxis to factor replacement.

Results:

Twenty‐four spontaneous and traumatic BEs are described (muscle hematomas, joint hemarthroses, port site, and epistaxis) involving 11 subjects. Eptacog beta was effective for acute bleed treatment as both first‐line therapy and for treatment of BEs refractory to eptacog alfa in 23/24 events. When eptacog beta was used for prophylaxis, 2/3 patients reported a decreased frequency of breakthrough BEs compared with prophylactic eptacog alfa and one patient experienced a similar frequency of breakthrough BEs compared with prophylactic activated prothrombin complex concentrate. Eptacog beta provided effective bleed control for three subjects who underwent minor surgical procedures. Treatment with eptacog beta was estimated to be 46%–72% more cost‐effective than eptacog alfa. No safety concerns or adverse events were reported.

Conclusions:

In this case series, eptacog beta was safe, effective, and economical as first‐line therapy, treatment of refractory BEs, management of perioperative bleeding, or prophylaxis in haemophilia patients with inhibitors.

2024-08-01·Neurocritical care

Sex Differences in Perihematomal Edema Volume and Outcome After Intracerebral Hemorrhage

Article

作者: Luo, Yunshi ; Witsch, Jens ; McCullough, Louise D ; Sloane, Kelly L ; Rothstein, Aaron ; Favilla, Christopher G ; Cao, Quy ; Kasner, Scott E ; Gusdon, Aaron M ; Choi, Huimahn A ; Song, Jae W ; Cucchiara, Brett L ; Messé, Steve R ; Mayer, Stephan A

BACKGROUND:

Although larger hematoma volume is associated with worse outcome after intracerebral hemorrhage (ICH), the association between perihematomal edema (PHE) volume and outcome remains uncertain, as does the impact of sex on PHE and outcome. Here we aimed to determine whether larger PHE volume is associated with worse outcome and whether PHE volume trajectories differ by sex.

METHODS:

We conducted a post hoc analysis of the Factor VIIa for Acute Hemorrhagic Stroke Treatment (FAST) trial, which randomized patients with ICH to receive recombinant activated factor VIIa or placebo. Computerized planimetry calculated PHE and ICH volumes on serial computed tomography (CT) scans (at baseline [within 3 h of onset], at 24 h, and at 72 h). Generalized estimating equations examined interactions between sex, CT time points, and FAST treatment arm on PHE and ICH volumes. Mixed and multivariable logistic models examined associations between sex, PHE, and outcomes.

RESULTS:

A total of 781 patients with supratentorial ICH (mean age 65 years) were included. Compared to women (n = 296), men (n = 485) had similar median ICH (14.9 vs. 13.6 mL, p = 0.053) and PHE volumes (11.1 vs. 10.5 mL, p = 0.56) at baseline but larger ICH and PHE volumes at 24 h (19.0 vs. 14.0 mL, p < 0.001; 22.2 vs. 15.7 mL, p < 0.001) and 72 h (16.0 vs. 11.8 mL, p < 0.001; 28.7 vs. 19.9 mL, p < 0.001). Men had higher absolute early PHE expansion (p < 0.001) and more hematoma expansion (growth ≥ 33% or 6 mL at 24 h, 33% vs. 22%, p < 0.001). An interaction between sex and CT time points on PHE volume (p < 0.001), but not on ICH volume, confirmed a steeper PHE trajectory in men. PHE expansion (per 5 mL, odds radio 1.19, 95% confidence interval 1.10-1.28), but not sex, was associated with poor outcome.

CONCLUSIONS:

Early PHE expansion and trajectory in men were significantly higher. PHE expansion was associated with poor outcomes independent of sex. Mechanisms leading to sex differences in PHE trajectories merit further investigation.

1

项与 BAX-817 相关的新闻（医药）

2025-04-24

·FierceBiotech

Bioxodes halts phase 2 stroke trial after successful interim review, plots registrational study

BIOX-101 is derived from a protein found in the salivary glands of a tick. \n Bioxodes has stopped enrolling people in a phase 2a stroke study after hitting the safety and efficacy endpoints, positioning the Belgian biotech to power into a potentially registrational trial.The interim phase 2a analysis included data on 16 intracerebral hemorrhagic stroke (ICH) patients. Some patients received a single intravenous dose of BIOX-101, also known as Ir-CPI, on top of standard of care. Other people only received standard of care. Bioxodes’ primary endpoints looked at safety outcomes. The biotech’s secondary endpoints assessed efficacy in the 10 days after randomization.At the interim analysis, the biotech saw positive changes in hematoma and edema volume, a secondary efficacy endpoint, and said inflammation biomarkers were encouraging. No patients died, and no serious adverse events were attributed to the drug. The treatment was well tolerated with no signs of increased bleeding.Hans Warrinnier, chief medical officer at Bioxodes, said in a statement that the results are preliminary but all point in the right direction. The biotech believes generating more phase 2a data won’t change the interim conclusions, leading it to stop enrollment in the study early. Bioxodes will now start raising a series B financing round and making BIOX-101 ready for a phase 2b trial. The process will take around one year, Bioxodes CEO Marc Dechamps said, putting the company on track to start enrolling patients in the potentially pivotal study in the first half of 2027. Bioxodes said the phase 2b could support filings for conditional marketing authorization in the U.S. and Europe before 2030.BIOX-101 is derived from a protein found in the salivary glands of a tick. The tick secretes the protein to stop the host’s blood from clotting and allow it to feed. Repurposed for treating ICH, the approach could tackle blood clots without causing bleeding. There are currently no approved treatments for ICH. Efforts to change that include a phase 3 study of recombinant factor VIIa (rFVIIa) that has an estimated complete date of January 2028. An earlier phase 3 trial found rFVIIa failed to improve survival or functional outcomes. That trial allowed patients to receive rFVIIa up to four hours after stroke onset. The ongoing rFVIIa trial has a treatment window of two hours.

临床结果临床2期临床3期

100 项与 BAX-817 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB00036

研发状态

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
血友病	临床3期	-	Baxter BioScience Corp.	2015-03-13
血友病A	临床3期	美国	Baxalta, Inc.	2013-02-20
血友病A	临床3期	日本	Baxalta, Inc.	2013-02-20
血友病A	临床3期	波兰	Baxalta, Inc.	2013-02-20
血友病A	临床3期	罗马尼亚	Baxalta, Inc.	2013-02-20
血友病A	临床3期	俄罗斯	Baxalta, Inc.	2013-02-20
血友病A	临床3期	塞尔维亚	Baxalta, Inc.	2013-02-20
血友病A	临床3期	西班牙	Baxalta, Inc.	2013-02-20
血友病A	临床3期	中国台湾	Baxalta, Inc.	2013-02-20
血友病A	临床3期	乌克兰	Baxalta, Inc.	2013-02-20

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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


ISTH2020	N/A	-	91	Reference rFVIIa product	餘窪蓋獵淵簾衊憲鬱衊(夢壓餘繭鑰膚築網糧鹹) = 鏇簾願遞糧範製憲網鬱憲遞觸憲遞艱鹹夢範遞 (襯襯觸範鹽壓簾醖構蓋 )	-	2020-07-12
				Biosimilar rFVIIa product	餘窪蓋獵淵簾衊憲鬱衊(夢壓餘繭鑰膚築網糧鹹) = 製選觸構鹽齋鑰築廠網憲遞觸憲遞艱鹹夢範遞 (襯襯觸範鹽壓簾醖構蓋 )
ISTH2020	N/A	血友病A	6	rFVIIa	觸築顧廠鹽簾醖蓋衊製(範廠窪膚衊鏇蓋醖範觸) = 網廠築淵獵觸夢積築餘襯淵醖繭齋壓齋壓繭築 (築鏇繭餘餘夢築獵遞遞 )	-	2020-07-12
NCT01757405 (CTgov)	临床3期	血友病B \| 血友病A	40	rFVIIa (Arm 1: up to 3 x 90 Micrograms/kg rFVIIa BI)	窪築鏇網選淵壓膚鹽築 = 膚壓遞築選構獵願醖構襯獵鹽網鑰鬱鹹鹹繭鏇 (製鬱構襯遞糧壓範網衊, 製構夢襯艱簾鏇齋襯遞 ~ 範顧觸製範鬱願餘遞鹹) 更多	-	2016-10-25
				rFVIIa (Arm 2: 1 x 270 Micrograms/kg rFVIIa BI)	窪築鏇網選淵壓膚鹽築 = 簾淵範繭壓淵網鏇鹽鹹襯獵鹽網鑰鬱鹹鹹繭鏇 (製鬱構襯遞糧壓範網衊, 繭製顧壓觸鏇鏇積鬱廠 ~ 廠糧觸鹽憲廠繭壓顧鑰) 更多
ESC2016	N/A	出血	281	Very low-dose recombinant factor VIIa	廠遞膚衊獵築觸淵壓顧(觸簾蓋鹽廠糧艱餘築醖) = 醖膚夢願鬱獵網鏇觸鬱鹹築簾襯遞網觸壓製糧 (鹹簾糧蓋鹹鏇齋鹽鏇鏇 ) 更多	-	2016-08-30
NCT01757405 (BusinessWire) 人工标引	临床3期	血友病B \| 血友病A	-	BAX-817 (dosing either 3x90µg/kg)	壓鹹範襯觸簾顧鏇簾構(蓋膚積淵餘簾製觸選繭) = 鬱選簾簾鏇淵構襯餘範齋築蓋廠鹹簾鹽觸淵積 (簾願齋獵齋壓遞憲壓觸 )	积极	2015-03-13
				BAX-817 (dosing either 1x270µg/kg)	壓鹹範襯觸簾顧鏇簾構(蓋膚積淵餘簾製觸選繭) = 壓鑰築鹽鏇憲衊廠鏇鏇齋築蓋廠鹹簾鹽觸淵積 (簾願齋獵齋壓遞憲壓觸 )