期刊
KIDNEY INTERNATIONAL
卷 97, 期 6, 页码 1109-1116出版社
ELSEVIER SCIENCE INC
DOI: 10.1016/j.kint.2020.02.029
关键词
Alport syndrome; chronic kidney disease; CKD; clinical trial design; end-stage kidney disease; ESKD; end-stage renal disease; ESRD; genetic disease; rare disease
资金
- Reata Pharmaceuticals, Inc.
- Regulus Therapeutics Inc.
- ASF
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 criticallypatients 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.
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