Antihemophilic Factor (Human) (CSL Behring)(Antihemophilic Factor (Human) (CSL Behring)) - 药物靶点：F10_在研适应症：血友病A_专利_临床

概要

基本信息

药物类型

非重组凝血因子

别名

Monoclate、Monoclate-P

靶点

F10

作用机制

F10刺激剂(凝血因子X刺激剂)

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A

非在研适应症-

原研机构

CSL Behring LLC

在研机构

CSL Behring LLC

非在研机构-

最高研发阶段批准上市

首次获批日期

美国 (2003-05-14),

血友病A

最高研发阶段(中国)-

特殊审评-

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关联

1

项与 Antihemophilic Factor (Human) (CSL Behring) 相关的临床试验

NCT01051544

/ WithdrawnN/AIIT

Randomised Study of First TIME Immunotolerance Induction in Patients With Severe Type A Haemophilia With Inhibitor at High Risk of Failure: Comparison of Induction of Immune Tolerance With FVIII Concentrates With or Without Von Willebrand Factor Acronym: RES.I.S.T.- Naive

This is a prospective, controlled, randomized, open label study, aimed at comparing FVIII/VWF concentrates with FVIII concentrates at 200 IU/kg daily in their ability to induce immune tolerance in Haemophilia A patients with high responding inhibitors and poor prognosis for success.

开始日期2009-09-25

申办/合作机构

City of Hope National Medical Center

[+4]

100 项与 Antihemophilic Factor (Human) (CSL Behring) 相关的临床结果

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100 项与 Antihemophilic Factor (Human) (CSL Behring) 相关的转化医学

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100 项与 Antihemophilic Factor (Human) (CSL Behring) 相关的专利（医药）

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20

项与 Antihemophilic Factor (Human) (CSL Behring) 相关的文献（医药）

2003-09-01·Haemophilia2区 · 医学

Impact of inhibitor epitope profile on the neutralizing effect against plasma‐derived and recombinant factor VIII concentrates in vitro

2区 · 医学

Article

作者: Erik Berntorp ; A. Shapiro ; Jan Astermark ; G. Tjönnfjord ; D. DiMichele ; J. Voorberg ; H. Lenk

Summary. The inhibitory capacity of plasma samples from 24 patients with severe haemophilia A and high‐responding inhibitors were evaluated in a concentrate‐based assay using two plasma‐derived (Haemate and Monoclate‐P) and three recombinant (Helixate, Recombinate and ReFacto) factor VIII concentrates and correlated with the corresponding epitope profile. In most, but not all, inhibitor plasmas with a relatively low reactivity against the von Willebrand‐containing product Haemate, the main epitopes were located in the FVIII light chain. The reactivities within the group of recombinant products varied in that the reactivity against the B‐domain deleted ReFacto was in general higher than that against Recombinate and Helixate. This difference did not correlate with any particular epitope profile and indicates that the B‐domain, type of formulation and/or purification procedures may have an impact on the inhibitor reactivity in vitro. The ratio between the inhibitor titres in the concentrate‐based assay and the Bethesda assay was dependent on the inhibitor plasma and concentrate used. Taken together, our results show that the reactivity of inhibitor plasmas varies considerably between different FVIII concentrates and that it does not fully correlate with the epitope profile. Potential clinical implications of the observed differences in inhibitor reactivity are discussed.

2001-03-14·Haemophilia2区 · 医学

Viral safety of a pasteurized, monoclonal antibody‐purified factor VIII concentrate in previously untreated haemophilia A patients

2区 · 医学

Article

作者: Philipp, C S

The efficacy and viral safety of a pasteurized, immunoaffinity‐purified procoagulant factor VIII protein (FVIII:C; Monoclate‐P) was studied in two multicentre, prospective, open‐label trials in 30 previously untreated patients, 18 with severe (< 1% FVIII:C activity), and 12 with moderate (1% to 5% FVIII:C activity) haemophilia A. Clinical assessments, performed at screening and regularly thereafter for 6 to > 24 months (maximum 34 months), showed that none of 24 assessable patients acquired illnesses consistent with monitored transfusion‐transmissible diseases. No patients acquired hepatitis B surface antigen, or antibodies against hepatitis B core antigen, hepatitis C, or human immunodeficiency virus. Likewise, no patients acquired treatment‐related hepatitis A antibodies or sustained elevations of alanine aminotransferase levels. The safety profile for Monoclate‐P is brought about by a multi‐step safety system that incorporates viral inactivation (through a combination of immunoaffinity chromatography and pasteurization) plus donor screening, plasma testing, and quality assurance. The inhibitor development rate (13% low titre, 10% high titre) was similar to that reported in the literature for other FVIII concentrates (24% to 52%). The most frequently reported adverse events were related to typical infant and childhood diseases. Monoclate‐P was effective in all patients treated according to protocol, except in two, who developed inhibitors.

2000-03-01·Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis

Pasteurized, monoclonal antibody factor VIII concentrate: establishing a new standard for purity and viral safety of plasma-derived concentrates.

Review

作者: Goldsmith, J C

A factor VIII concentrate (Monoclate-P) manufactured using a combination of pasteurization and immunoaffinity chromatography has been chosen to compare and contrast manufacturing aspects of plasma-derived factor VIII concentrates. Pasteurization is a virucidal method with a long safety record in clinical practice, while immuno-affinity chromatography selectively isolates and purifies the procoagulant protein of factor VIII, and partitions potential viral contaminants and nonessential proteins to the unbound fraction. The complete Monoclate-P production process reduces human immunodeficiency virus by > or = 10.5 log10, Sindbis (a model for hepatitis C virus) by > or = 6.5 log10, and murine encephalomyocarditis virus (a non-enveloped model virus) by 7.1 log10. The viral safety of Monoclate-P has been further demonstrated in clinical studies in patients not previously treated with blood or plasma-derived products. Additionally, the manufacture of Monoclate-P includes careful donor screening and plasma testing for antibodies to syphilis and human immunodeficiency, hepatitis B, and hepatitis C viruses to enhance source plasma safety. Combined with donor selection and plasma testing, multiple viral reduction steps effectively eliminate both lipid-enveloped viruses (e.g. human immunodeficiency, hepatitis B and C) and non-lipid-enveloped viruses (e.g. hepatitis A). In addition, polymerase chain reaction-based nucleic acid detection tests for hepatitis B and C viruses and for human immunodeficiency virus-1 have been introduced as part of an investigational new drug mechanism.

100 项与 Antihemophilic Factor (Human) (CSL Behring) 相关的药物交易

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