Ohga Shouichi, Takeyama Masahiro, Ishimura Masataka, Inoue Hirosuke, Nosaka Daisuke, Iwasaki Keisuke, Mitsui Chika, Nogami Keiji
Department of Paediatrics, Graduate School of Medical Sciences, Kyushu University, Fukuoka, Japan
Department of Paediatrics, Nara Medical University, Kashihara, Japan.
BMJ Open. 2024 Dec 26;14(12):e087556. doi: 10.1136/bmjopen-2024-087556.
Emicizumab prophylaxis is approved for people of all ages with haemophilia A (HA) including infants and children. Although previous studies have demonstrated the efficacy and tolerability of emicizumab in infants with HA, real-world data on emicizumab use in infants are limited. The Haemophilia A in Infancy and NewbOrns: multi-instituional prospective observational study to assess the efficacy anD safety of Emicizumab (HINODE) study aims to evaluate the coagulation potential and safety of emicizumab prophylaxis in infants with congenital HA from birth to <12 months of age.
This is a multicentre, observational study conducted in Japan in infants with congenital HA aged <12 months who are receiving or are scheduled to receive prophylactic emicizumab at an approved dosing regimen: 1.5 mg/kg weekly, 3 mg/kg every 2 weeks or 6 mg/kg every 4 weeks. The target inclusion is 50 infants. The primary endpoint is to evaluate the relationship between global coagulation test parameters (using clot waveform analysis and thrombin generation assay) and plasma emicizumab concentrations in infants aged from 6 to <12 months. Secondary endpoints include evaluating coagulation profiles in infants aged <6 months and changes between the age of <6 and 6 to <12 months. Additionally, coagulation parameters will be evaluated with the in vitro addition of anti-idiotype antibodies against emicizumab or the addition of a factor VIII product in infants aged from 6 to <12 months. The study will also evaluate adverse events and bleeds.
The study was approved by the MINS Clinical Trial Review Committee (no. 230214) and will be conducted in compliance with the Declaration of Helsinki, the Act on the Protection of Personal Information and the Guidance of Ethical Guidelines for Medical and Biological Research Involving Human Subjects. Written informed consent for participation in the study will be obtained from a legally acceptable representative. Results will be published in scientific/medical journals and presented at international congresses.
Japan Registry of Clinical Trials; jRCT1031230264.
艾美赛珠单抗预防治疗已被批准用于所有年龄段的甲型血友病（HA）患者，包括婴儿和儿童。尽管先前的研究已证明艾美赛珠单抗在HA婴儿中的疗效和耐受性，但关于婴儿使用艾美赛珠单抗的真实世界数据有限。婴儿和新生儿甲型血友病：评估艾美赛珠单抗疗效和安全性的多机构前瞻性观察性研究（HINODE）旨在评估从出生至＜12月龄的先天性HA婴儿使用艾美赛珠单抗预防治疗的凝血潜能和安全性。
这是一项在日本进行的多中心观察性研究，研究对象为年龄＜12月龄、正在接受或计划接受按批准给药方案（每周1.5mg/kg、每2周3mg/kg或每4周6mg/kg）预防性使用艾美赛珠单抗的先天性HA婴儿。目标纳入50名婴儿。主要终点是评估6至＜12月龄婴儿的整体凝血试验参数（采用凝血块波形分析和凝血酶生成测定）与血浆艾美赛珠单抗浓度之间的关系。次要终点包括评估＜6月龄婴儿的凝血谱以及＜6月龄与6至＜12月龄之间的变化。此外，将在6至＜12月龄婴儿中通过体外添加抗艾美赛珠单抗独特型抗体或添加凝血因子VIII产品来评估凝血参数。该研究还将评估不良事件和出血情况。
该研究已获得MINS临床试验审查委员会批准（编号230214），并将按照《赫尔辛基宣言》、《个人信息保护法》以及涉及人类受试者的医学和生物学研究伦理准则指南进行。将从具有法律认可资格的代表处获得参与研究的书面知情同意书。研究结果将发表在科学/医学期刊上，并在国际大会上展示。
日本临床试验注册中心；jRCT1031230264。
