Alport Syndrome Foundation, Phoenix, Arizona, USA.
National Kidney Foundation, New York, New York, USA.
Kidney Int. 2020 Jun;97(6):1109-1116. doi: 10.1016/j.kint.2020.02.029. Epub 2020 Apr 6.
Alport syndrome is experiencing a remarkable increase in preclinical investigations. To proactively address the needs of the Alport syndrome community, as well as offer clarity for future clinical research sponsors, the Alport Syndrome Foundation hosted a workshop to generate consensus recommendations for prospective trials for conventional drugs. Opinions of key stakeholders were carefully considered, including those of the biopharmaceutical industry representatives, academic researchers, clinicians, regulatory agency representatives, and-most critically-patients with Alport syndrome. Recommendations were established for preclinical researchers, the use and selection of biomarkers, standards of care, clinical trial designs, trial eligibility criteria and outcomes, pediatric trial considerations, and considerations for patient engagement, recruitment, and treatment. This paper outlines their recommendations.
Alport 综合征的临床前研究显著增加。为积极满足 Alport 综合征患者群体的需求，并为未来的临床研究赞助商提供明确指导，Alport 综合征基金会组织了一次研讨会，旨在为常规药物的前瞻性试验制定共识建议。认真考虑了主要利益相关者的意见，包括生物制药行业代表、学术研究人员、临床医生、监管机构代表以及最重要的 Alport 综合征患者的意见。为临床前研究人员制定了建议，包括生物标志物的使用和选择、护理标准、临床试验设计、试验资格标准和结果、儿科试验考虑因素以及患者参与、招募和治疗的考虑因素。本文概述了这些建议。
