[Translation] A single-arm, open-label, multicenter clinical trial to evaluate the safety, efficacy, and pharmacokinetic characteristics of recombinant human coagulation factor VIII in the prevention and treatment of children with severe hemophilia A (<12 years old) who have been treated

主要目的：评价12岁以下经治疗的重型血友病A患儿给予注射用重组人凝血因子Ⅷ制剂进行预防治疗的有效性和安全性。次要目的：观察注射用重组人凝血因子Ⅷ在12岁以下经治疗的重型血友病A患儿中单次给药后的药代动力学特征，并计算药代动力学参数。

[Translation]

Primary objective: To evaluate the efficacy and safety of recombinant human coagulation factor VIII preparations for prophylaxis in children under 12 years of age with severe hemophilia A who have been treated. Secondary objective: To observe the pharmacokinetic characteristics of recombinant human coagulation factor VIII for injection in children under 12 years of age with severe hemophilia A who have been treated, and to calculate the pharmacokinetic parameters.

Start Date-

Sponsor / Collaborator

Beijing Neoletix Biological Technology

CTR20231408 / Active, not recruitingPhase 3

评价重组人凝血因子Ⅷ在经治疗重型血友病A患者（≥12岁）中按需治疗序贯预防治疗的安全性、有效性和药代动力学特征的单臂、开放、多中心临床试验

[Translation] A single-arm, open-label, multicenter clinical trial to evaluate the safety, efficacy, and pharmacokinetic characteristics of recombinant human coagulation factor VIII as an on-demand treatment for sequential prophylaxis in patients with severe hemophilia A (≥12 years old) who have been treated

主要目的：评价重型血友病A患者给予注射用重组人凝血因子Ⅷ制剂进行按需治疗和预防治疗的安全性和有效性；次要目的：1）观察注射用重组人凝血因子Ⅷ在血友病A患者中单次给药和持续给药后的药代动力学特征，并计算药代动力学参数；2）探索性观察围手术期的重型血友病A患者使用注射用重组人凝血因子Ⅷ的疗效。

[Translation]

Primary purpose: To evaluate the safety and efficacy of recombinant human coagulation factor VIII preparations for injection for on-demand treatment and preventive treatment in patients with severe hemophilia A; Secondary purpose: 1) To observe the pharmacokinetic characteristics of recombinant human coagulation factor VIII for injection in patients with hemophilia A after single administration and continuous administration, and to calculate the pharmacokinetic parameters; 2) To explore the efficacy of recombinant human coagulation factor VIII for injection in patients with severe hemophilia A during the perioperative period.

Start Date25 Jul 2023

Sponsor / Collaborator

Beijing Neoletix Biological Technology

100 Clinical Results associated with Recombinant human coagulation factor VIII ( Beijing Neoletix)

100 Translational Medicine associated with Recombinant human coagulation factor VIII ( Beijing Neoletix)

100 Patents (Medical) associated with Recombinant human coagulation factor VIII ( Beijing Neoletix)

Literatures (Medical) associated with Recombinant human coagulation factor VIII ( Beijing Neoletix)

01 Dec 2023·Zhongguo shi yan xue ye xue za zhi

[Pharmacokinetics of Recombinant Human Coagulation Factor Ⅷ Preparations in Patients with Severe Hemophilia A].

Article

Author: Chen, Yi-Jian ; Xin, Liu-Yan ; Yang, Xiang ; Lin, Chuan-Ming ; Huang, Zou-Fang ; Li, Ting ; Li, Lin

OBJECTIVETo calculate the pharmacokinetic parameters of recombinant human coagulation factor Ⅷ using myPKFiT in patients with severe hemophilia A, and provide an individualized treatment plan for patients.METHODSA total of 42 patients with severe hemophilia A who were treated with recombinant human coagulation factor Ⅷ were included from January 2021 to December 2021. myPKFiT was used to calculate the pharmacokinetic parameters of FⅧ, and the individualized treatment plan for hemophilia A patients was formulated.RESULTSThe median age of 42 patients with severe hemophilia A was 31（16-50） years old, the average weight was 54.0±9.9 kg, the half-life of FⅧ was 12.05±1.6 h, the time to more than 1% of the baseline was 62.3±15.3 h, and the 0 bleeding rate after the guidance of myPKFiT was significantly increased from 39% to 49%, the Annual bleeding rate was reduced from 3.6±2.5 to 2.1±2.0, and the Annual joint bleeding rate was reduced from 3.2±2.2 to 1.9±0.9, all of which were statistically different (P<0.05).CONCLUSIONIndividualized therapy in patients with severe hemophilia A who were guided by myPKFiT assay of pharmacokinetics parameters can significantly reduce the annual bleeding rate and annual joint bleeding rate of patients.

28 May 2008·FEBS LettersQ3 · BIOLOGY

Binding of recombinant human coagulation factor VIII to lipid nanotubes

Q3 · BIOLOGY

Article

Author: Christopher D.J. Parmenter ; Svetla Stoilova-McPhie

Cryo‐electron microscopy has the power to visualise lipid membranes at the closest to in vivo conditions. The structure of the lipid bilayer can be well resolved and the interactions between lipid–protein and protein–protein molecules followed at the molecular level. We undertook an extended Cryo‐electron microscopy study to follow the factor VIII binding to phosphatidylserine containing lipid nanotubes at different lipid composition. Obtaining well ordered tubes is required to define the factor VIII membrane‐bound structure. The observed alterations in the arrangement of the protein molecules are indicative for the flexibility of the membrane‐bound factor VIII. Understanding the significance of these conformational changes is essential to comprehend the function of factor VIII in coagulation and as a drug for Hemophilia A.

01 Jan 2008·DrugsQ1 · MEDICINE

Sucrose-Formulated Octocog Alfa

Q1 · MEDICINE

Review

Author: James E Frampton ; Antona J Wagstaff

Sucrose-formulated octocog alfa (Kogenate Bayer, Kogenate FS, Helixate FS, Helixate nexgen) is a full-length recombinant human coagulation factor VIII (FVIII) product that is purified and formulated without the addition of human serum albumin and is stabilized with sucrose. The purification process of this formulation includes a solvent/detergent viral inactivation step. Sucrose-formulated octocog alfa is approved in the EU and US for the treatment of bleeding in patients with haemophilia A (congenital FVIII deficiency). Additionally, it is approved in the EU for the prophylaxis of bleeding in patients with haemophilia A and as a continuous infusion treatment in patients undergoing major surgery. Sucrose-formulated octocog alfa is effective and well tolerated as a FVIII replacement therapy in patients with haemophilia A, including those with severe disease undergoing major surgery. The therapeutic profile of this sucrose-formulated product cannot be compared with that of other octocog alfa or moroctocog alfa products because of a lack of head-to-head comparative studies. Pathogen transmission has not been reported with use of sucrose-formulated octocog alfa. Available data indicate that sucrose-formulated octocog alfa is an appropriate alternative to other recombinant FVIII products for the treatment and prophylaxis of bleeding episodes in adults and children with haemophilia A.

100 Deals associated with Recombinant human coagulation factor VIII ( Beijing Neoletix)

R&D Status

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Indication	Highest Phase	Country/Location	Organization	Date
Hemophilia A	Phase 3	CN	Beijing Neoletix Biological Technology	25 Jul 2023
Hemorrhage	IND Application	CN	Beijing Neoletix Biological Technology	30 Jan 2022

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