2026-06-14T03:46:26Zhttps://repisalud.isciii.es/rest/oai/request

oai:repisalud.isciii.es:20.500.12105/235372024-11-28T21:00:51Zcom_20.500.12105_15322com_20.500.12105_2051col_20.500.12105_16967

00925njm 22002777a 4500 dc Sinha, Smeeta author Gould, Lisa J author Nigwekar, Sagar U author Serena, Thomas E author Brandenburg, Vincent author Moe, Sharon M author Aronoff, George author Chatoth, Dinesh K author Hymes, Jeffrey L author Miller, Stephan author Padgett, Claire author Carroll, Kevin J author Perello, Joan author Gold, Alex author Chertow, Glenn M author 2022-01 Background: Calcific uraemic arteriolopathy (CUA; calciphylaxis) is a rare disease seen predominantly in patients receiving dialysis. Calciphylaxis is characterized by poorly healing or non-healing wounds, and is associated with mortality, substantial morbidity related to infection and typically severe pain. In an open-label Phase 2 clinical trial, SNF472, a selective inhibitor of vascular calcification, was well-tolerated and associated with improvement in wound healing, reduction of wound-related pain and improvement in wound-related quality of life (QoL). Those results informed the design of the CALCIPHYX trial, an ongoing, randomized, placebo-controlled, Phase 3 trial of SNF472 for treatment of calciphylaxis. Methods: In CALCIPHYX, 66 patients receiving haemodialysis who have an ulcerated calciphylaxis lesion will be randomized 1:1 to double-blind SNF472 (7 mg/kg intravenously) or placebo three times weekly for 12 weeks (Part 1), then receive open-label SNF472 for 12 weeks (Part 2). All patients will receive stable background care, which may include pain medications and sodium thiosulphate, in accordance with the clinical practices of each site. A statistically significant difference between the SNF472 and placebo groups for improvement of either primary endpoint at Week 12 will demonstrate efficacy of SNF472: change in Bates-Jensen Wound Assessment Tool-CUA (a quantitative wound assessment tool for evaluating calciphylaxis lesions) or change in pain visual analogue scale score. Additional endpoints will address wound-related QoL, qualitative changes in wounds, wound size, analgesic use and safety. Conclusions: This randomized, placebo-controlled Phase 3 clinical trial will examine the efficacy and safety of SNF472 in patients who have ulcerated calciphylaxis lesions. Patient recruitment is ongoing. Sinha S, Gould LJ, Nigwekar SU, Serena TE, Brandenburg V, Moe SM, et al. The CALCIPHYX study: a randomized, double-blind, placebo-controlled, Phase 3 clinical trial of SNF472 for the treatment of calciphylaxis. Clin Kidney J. 2022 Jan;15(1):136-44. 10.1093/ckj/sfab117 2048-8513 2048-8505 Clinical Kidney Journal https://hdl.handle.net/20.500.13003/19852 35035944 L2016946868 2-s2.0-85124975246 https://hdl.handle.net/20.500.12105/23537 743244500016 calciphylaxis controlled clinical trial design SNF472 rationale The CALCIPHYX study: a randomized, double-blind, placebo-controlled, Phase 3 clinical trial of SNF472 for the treatment of calciphylaxis