Factor IX(Bio Products Laboratory)(Factor IX(Bio Products Laboratory)) - 药物靶点：factor IX_在研适应症：血友病B_专利_临床

概要

基本信息

药物类型

非重组凝血因子

别名

Factor IX、High purity Factor IX concentrate、Replenine-VF

靶点

factor IX

作用方式

刺激剂

作用机制

factor IX刺激剂(凝血因子IX刺激剂)、凝血途径激活剂

治疗领域

遗传病与畸形血液及淋巴系统疾病其他疾病

在研适应症

血友病B

非在研适应症-

原研机构

Bio Products Laboratory USA, Inc.

在研机构

Bio Products Laboratory USA, Inc.

非在研机构-

权益机构-

最高研发阶段批准上市

首次获批日期

英国 (1999-08-09),

血友病B

最高研发阶段(中国)-

特殊审评-

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关联

6

项与 Factor IX(Bio Products Laboratory) 相关的临床试验

JPRN-UMIN000049908

/ SuspendedN/AIIT

Safety evaluation in an open-label study of an overdose of the capsules containing Valeriana fauriei root (VF) extract. - Safety evaluation of the capsules containing VF extract.

开始日期2023-01-17

申办/合作机构-

NCT02250573

/ Completed临床3期

An Open-study to Investigate the Safety and Efficacy of Replenine®-VF in Haemophilia B Subjects Undergoing Surgery.

The main objectives of this study were to investigate the safety and efficacy of Replenine®-VF administered in appropriate dosage by bolus infusion to prevent bleeding and achieve haemostasis in subjects with haemophilia B undergoing surgery.

开始日期2005-12-01

申办/合作机构

Bio Products Laboratory USA, Inc.

NCT02263469

/ Completed临床3期

An Open Multi-centre Phase III Study to Investigate the Safety and Efficacy of Replenine®-VF in Severe Haemophilia B Patients Under the Age of 6 Years

The objective of this study was to assess the safety and efficacy of Replenine®-VF in children enrolled in the study, under the age of six years, with severe haemophilia B.

开始日期2005-08-01

申办/合作机构

Bio Products Laboratory USA, Inc.

100 项与 Factor IX(Bio Products Laboratory) 相关的临床结果

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100 项与 Factor IX(Bio Products Laboratory) 相关的转化医学

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100 项与 Factor IX(Bio Products Laboratory) 相关的专利（医药）

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88

项与 Factor IX(Bio Products Laboratory) 相关的文献（医药）

2025-03-01·Haemophilia

Bleeding Episodes in Patients With Haemophilia B Receiving Prophylactic Factor IX Treatment: A Systematic Review and Meta‐Analysis

Review

作者: Mannucci, Pier Mannuccio ; Pasca, Samantha ; Mengoli, Carlo ; Focosi, Daniele ; Franchini, Massimo

ABSTRACTBackgroundProphylaxis with coagulation factor concentrates is the mainstay of treatment in severe hemophilia A and B. Data on bleeding rates in persons with congenital haemophilia B (PwcHB) receiving prophylaxis are inconsistent.AimThis systematic review and meta‐analysis were aimed at assessing bleeding outcomes, including annualised bleeding rates (ABR) and the proportion of patients with zero bleeding events, in PwcHB receiving prophylaxis with plasma‐derived or recombinant FIX products with standard (rSHL) or extended half‐life (rEHL).MethodsA systematic search was conducted using the bibliographic database Medline, Embase and Cochrane Central Register. The protocol was registered on PROSPERO (registration number: CRD42024592785).ResultsThe search yielded 2440 citations and a total of 42 studies (2 randomised and 40 nonrandomised) were included in the final analysis. The pooled estimated mean (95% confidence interval [CI]) ABR was significantly lower in PwcHB treated prophylactically with rEHL FIX than in those receiving rSHL FIX products (1.29 [95% CI: 0.91, 1.66] vs. 3.12 [95% CI: 2.63, 3.62], p < 0.01). The proportion of participants with zero bleeding events was significantly higher in PwcHB treated prophylactically with rEHL FIX than in those receiving rSHL FIX (0.53 [95% CI: 0.37, 0.69] vs. 0.24 [95% CI: 0.14, 0.39], p = 0.01). The ABR did not differ according to age groups (more or less than 12 years).ConclusionThe results of this meta‐analysis suggest that compared to standard half‐life FIX concentrates, prophylaxis with rEHL FIX products is associated with a reduction in ABR and a higher proportion of patients with no bleeding episodes.

2024-11-01·Special Care in Dentistry

Spontaneous resolution and bone regeneration in hemophilic pseudotumor: A rare case report and literature review

Review

作者: Gupta, Sunita ; Singh, Khushboo ; Aggarwal, Sunita

AbstractAimHemophilic pseudotumor (HP) is a very rare complication of hemophilia seen in only 1–2% of the cases. Although it is much more common in long bones, pelvis and small bones of hands and feet and very rarely involving jaw bones.Method and resultIn the present case, the presence of a rare hemophilic pseudotumor of the mandible with the positive history of Hemophilia B justifies that the history, clinical and radiological examinations were sufficient to arrive at conclusive diagnosis precluding invasive diagnostic procedures such as biopsy hence avoiding the risk of hemorrhage, infection, or fistula. This case also highlights that patient was conservatively managed with Factor IX replacement alone with a very good clinical outcome.ConclusionHP should be considered as a differential diagnosis of any progressive swelling of hard and soft tissues occurring in a patient with severe haemophilia.

2021-12-01·Trials4区 · 医学

Four-factor prothrombin complex concentrate to reduce allogenic blood product transfusion in patients with major trauma, the PROCOAG trial: study protocol for a randomized multicenter double-blind superiority study

4区 · 医学

ArticleOA

作者: Albrice Levrat ; Jacques Duranteau ; Jean-Stéphane David ; Jean-Luc Bosson ; Delphine Garrigue ; Julien Pottecher ; Bruno Riou ; Pierre Bouzat ; Jonathan Charbit ; Guillaume Marcotte ; Karim Asehnoune ; Jean-François Payen ; Paër-Selim Abback ; Marc Leone

AbstractBackgroundOptimal management of severe trauma patients with active hemorrhage relies on adequate initial resuscitation. Early administration of coagulation factors improves post-traumatic coagulation disorders, and four-factor prothrombin complex concentrate (PCC) might be useful in this context. Our main hypothesis is that four-factor PCC in addition to a massive transfusion protocol decreases blood product consumption at day 1 in severe trauma patients with major bleeding.MethodsThis is a prospective, randomized, multicenter, double-blind, parallel, controlled superiority trial. Eligible patients are trauma patients with major bleeding admitted to a French level-I trauma center. Patients randomized in the treatment arm receive 1 mL/kg (25 IU/ml of Factor IX/Kg) four-factor PCC within 1-h post-admission while patients randomized in the controlled group receive 1 mL/kg of saline solution 0.9% as a placebo. Treatments are given as soon as possible using syringe pumps (120 mL/h). The primary endpoint is the amount of blood products transfused in the first 24 h post-admission (including red blood cells, frozen fresh plasma, and platelets). The secondary endpoints are the amount of each blood product transfused in the first 24 h, time to achieve prothrombin time ratio < 1.5, time to hemostasis, number of thrombo-embolic events at 28 days, mortality at 24 h and 28 days, number of intensive care unit-free days, number of ventilator-free days, number of hospital-free days within the first 28 days, hospitalization status at day 28, Glasgow outcome scale extended for patients with brain lesions on initial cerebral imaging, and cost of each strategy at days 8 and 28. Inclusions have started in December 2017 and are expected to be complete by June 2021.DiscussionIf PCC reduces total blood consumption at day 1 after severe trauma, this therapy, in adjunction to a classic massive transfusion protocol, may be used empirically on admission in patients at risk of massive transfusion to enhance coagulation. Moreover, this treatment may decrease blood product-related complications and may improve clinical outcomes after post-traumatic hemorrhage.Trial registrationClinicalTrials.govNCT03218722. Registered on July 14, 2017

100 项与 Factor IX(Bio Products Laboratory) 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D04123	Factor IX(Bio Products Laboratory)	B02BD04	-

研发状态

批准上市

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适应症	国家/地区	公司	日期
血友病B	英国	Bio Products Laboratory USA, Inc.	1999-08-09

未上市

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适应症	最高研发状态	国家/地区	公司	日期
血友病B	临床前	波兰	Bio Products Laboratory USA, Inc.	1997-07-01

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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


ASGCT2016	N/A	血友病B	-	Lentiviral Vectors Expressing Hyper-Functional Factor IX (Portal vein administration of LVs expressing canine FIX)	艱壓選憲蓋夢齋鏇製憲(網艱顧願願觸鏇選膚窪) = Intravenous LV administration was well tolerated with mild and self-limiting elevation of aminotransferases in one dog 憲衊夢鬱淵築餘襯築繭 (積製製獵壓遞製鑰範餘 ) 更多	-	2016-05-01
				Lentiviral Vectors Expressing Hyper-Functional Factor IX (Peripheral vein administration of LVs expressing hyperfunctional canine FIX)
ASGCT2010	N/A	血友病B	-	Factor IX-R338L (FIX Padua) (AAV6-cFIX-wt)	餘網膚範繭醖餘鏇鹽夢(觸繭鏇製膚簾襯鏇築網) = 衊壓鑰窪糧簾選壓構製繭顧鏇壓衊廠鏇積範鬱 (顧衊窪選窪獵窪築憲鏇 ) 更多	-	2010-05-01
				Factor IX-R338L (FIX Padua) (AAV6-cFIX-Padua)	餘網膚範繭醖餘鏇鹽夢(觸繭鏇製膚簾襯鏇築網) = 顧願觸網夢艱繭夢糧憲繭顧鏇壓衊廠鏇積範鬱 (顧衊窪選窪獵窪築憲鏇 ) 更多