BOND 研究：一项为期 12 个月的随机、双盲、安慰剂对照试验，旨在评估苯磷硫胺对伴有症状性多发性神经病的 2 型糖尿病患者形态计量、神经生理学和临床指标的影响。
BOND study: a randomised double-blind, placebo-controlled trial over 12 months to assess the effects of benfotiamine on morphometric, neurophysiological and clinical measures in patients with type 2 diabetes with symptomatic polyneuropathy.
机构信息
Institute for Clinical Diabetology, German Diabetes Center, Leibniz Center for Diabetes Research at Heinrich Heine University, Düsseldorf, Germany.
Department of Endocrinology and Diabetology, Medical Faculty and University Hospital Düsseldorf, Heinrich Heine University, Düsseldorf, Germany.
出版信息
BMJ Open. 2022 Feb 3;12(2):e057142. doi: 10.1136/bmjopen-2021-057142.
INTRODUCTION
Diabetic sensorimotor polyneuropathy (DSPN) affects approximately 30% of people with diabetes, while around half of cases are symptomatic. Currently, there are only few pathogenetically oriented pharmacotherapies for DSPN, one of which is benfotiamine, a prodrug of thiamine with a high bioavailability and favourable safety profile. While benfotiamine has shown positive effects in preclinical and short-term clinical studies, no long-term clinical trials are available to demonstrate disease-modifying effects on DSPN using a comprehensive set of disease-related endpoints.
METHODS AND ANALYSIS
The benfotiamine on morphometric, neurophysiological and clinical measures in patients with type 2 diabetes trial is a randomised double-blind, placebo-controlled parallel group monocentric phase II clinical trial to assess the effects of treatment with benfotiamine compared with placebo in participants with type 2 diabetes and mild to moderate symptomatic DSPN. Sixty participants will be 1:1 randomised to treatment with benfotiamine 300 mg or placebo two times a day over 12 months. The primary endpoint will be the change in corneal nerve fibre length assessed by corneal confocal microscopy (CCM) after 12 months of benfotiamine treatment compared with placebo. Secondary endpoints will include other CCM measures, skin biopsy and function indices, variables from somatic and autonomic nerve function tests, clinical examination and questionnaires, general health, health-related quality of life, cost, safety and blood tests.
ETHICS AND DISSEMINATION
The trial was approved by the competent authority and the local independent ethics committee. Trial results will be published in peer-reviewed journals, conference abstracts, and via online and print media.
TRIAL REGISTRATION NUMBER
DRKS00014832.
简介
糖尿病周围感觉运动神经病变（DSPN）影响大约 30%的糖尿病患者，而大约一半的病例有症状。目前，针对 DSPN 仅有少数几种基于发病机制的药物治疗方法，其中之一是苯磷硫胺，一种硫胺素前体药物，生物利用度高，安全性良好。虽然苯磷硫胺在临床前和短期临床试验中显示出积极的效果，但尚无长期临床试验可用，以使用一套全面的与疾病相关的终点来证明其对 DSPN 的疾病修饰作用。
方法和分析
在 2 型糖尿病患者中评估苯磷硫胺对形态计量学、神经生理学和临床指标的影响的试验是一项随机、双盲、安慰剂对照的平行分组单中心 2 期临床试验，旨在评估与安慰剂相比，苯磷硫胺治疗对 2 型糖尿病和轻度至中度有症状 DSPN 患者的治疗效果。60 名参与者将按照 1:1 的比例随机分为苯磷硫胺 300mg 组或安慰剂组，每天两次，治疗 12 个月。主要终点是 12 个月苯磷硫胺治疗后角膜神经纤维长度的变化，通过角膜共聚焦显微镜（CCM）评估。次要终点包括其他 CCM 测量值、皮肤活检和功能指标、躯体和自主神经功能测试的变量、临床检查和问卷调查、一般健康状况、健康相关生活质量、成本、安全性和血液检查。
伦理和传播
该试验已获得主管部门和当地独立伦理委员会的批准。试验结果将发表在同行评议的期刊、会议摘要以及在线和印刷媒体上。
试验注册号
DRKS00014832。
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