Institute for Applied Research and Clinical Trials GmbH, Georg August University Göttingen, Göttingen, Germany; Institute for Applied Research and Clinical Trials GmbH, Georg August University Göttingen, Göttingen, Germany.
Institute for Applied Research and Clinical Trials GmbH, Georg August University Göttingen, Göttingen, Germany.
Clin Ther. 2014 Feb 1;36(2):184-90. doi: 10.1016/j.clinthera.2014.01.013.
Clinical trials require great effort, time, expertise, and money. For clinicians at university hospitals with their full work load of teaching and medical care, the planning of an investigator-initiated clinical trial seems almost unthinkable. Despite their expertise in distinct diseases, university clinicians lack the time necessary to organize the funding and to initiate and conduct Phase III clinical trials in adults or in children.
We sought to determine whether the difficulties faced by a clinician conducting a pediatric clinical trial can be overcome by passionate motivation and external support.
Critical aspects of the application process of the world's first clinical trial in children with the rare hereditary kidney disease Alport syndrome treated with an angiotensin-converting enzyme inhibitor (Early Prospective Therapy Trial to Delay Renal Failure in Children With Alport Syndrome [EARLY PRO-TECT Alport]; http://www.clinicaltrials.gov NCT01485978; EudraCT 2010-024300-10) are described.
The following crucial factors enabled the investigator to complete this trial: (1) support through clinical trial, biometrician, and regulatory experts (Institute for Applied Research and Clinical Studies [IFS], Göttingen, Germany); (2) advice from the university's ethics committee (University Medicine Göttingen, Göttingen, Germany); (3) public funding (€1 million from the German Federal Ministry of Education and Research); (4) support from the respective medical society, aiming at the resolution of an important clinical problem (German Society of Pediatric Nephrology); and (5) support from the investigator's university as the official sponsor of the trial, providing long-term commitment and covering financial risks (University Medical Center Göttingen, Göttingen, Germany).
The study could pave the way for approval of ramipril as a drug to treat children with Alport syndrome. Even though the study might not result in label changes, the EARLY PRO-TECT Alport trial provides the basis of an educational campaign to sensitize physicians, especially pediatricians, general practitioners, and nephrologists, to pay special attention to the early detection of kidney diseases in children, which could improve medical care for all children with kidney diseases.
临床试验需要付出巨大的努力、时间、专业知识和资金。对于大学附属医院的临床医生来说，他们的教学和医疗工作负担已经很重，因此规划一项由研究者发起的临床试验似乎几乎是不可能的。尽管他们在特定疾病方面具有专业知识，但大学临床医生缺乏必要的时间来组织资金，并启动和开展成人或儿童的 III 期临床试验。
我们试图确定，对于开展儿科临床试验的临床医生来说，通过满腔的热情和外部支持，是否可以克服所面临的困难。
描述了世界上首例针对遗传性肾脏疾病 Alport 综合征患儿进行的临床试验（用血管紧张素转换酶抑制剂治疗的早期前瞻性治疗试验以延迟 Alport 综合征患儿的肾脏衰竭[EARLY PRO-TECT Alport]；http://www.clinicaltrials.gov NCT01485978；EudraCT 2010-024300-10）的申请流程的关键方面。
以下关键因素使研究者得以完成该试验：（1）通过临床试验、生物统计学家和监管专家（德国哥廷根应用研究与临床研究学院[IFS]）提供的支持；（2）来自大学伦理委员会（德国哥廷根大学医学中心，哥廷根）的建议；（3）公共资金（德国联邦教育与研究部提供 100 万欧元）；（4）来自相关医学协会的支持，旨在解决一个重要的临床问题（德国儿科肾脏病学会）；（5）研究者所在大学作为试验的官方赞助商提供的支持，提供长期承诺并承担财务风险（德国哥廷根大学医学中心）。
该研究可能为批准雷米普利作为治疗 Alport 综合征患儿的药物铺平道路。尽管该研究可能不会导致标签变化，但 EARLY PRO-TECT Alport 试验为教育运动提供了基础，旨在使医生，特别是儿科医生、全科医生和肾病学家提高对儿童肾脏疾病早期发现的认识，从而改善所有患有肾脏疾病的儿童的医疗服务。
