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Abstract: INFO07

Design of the CALCIPHYX Study, a Randomized, Double-Blind, Placebo-Controlled Phase 3 Study of SNF472 for Treating Calciphylaxis

Session Information

Category: Bone and Mineral Metabolism

  • No subcategory defined

Authors

  • Sinha, Smeeta, Salford Royal NHS Foundation Trust and The University of Manchester, Greater Manchester, United Kingdom
  • Gould, Lisa, South Shore Health System Center for Wound Healing, Weymouth, Massachusetts, United States
  • Nigwekar, Sagar U., Massachusetts General Hospital and Harvard Medical School, Boston, Massachusetts, United States
  • Moe, Sharon M., Indiana University School of Medicine, Indianapolis, Indiana, United States
  • Aronoff, George R., DaVita Kidney Care, Naples, Florida, United States
  • Hymes, Jeffrey L., Fresenius Kidney Care, Nashville, Tennessee, United States
  • Miller, Stephan, Sanifit Therapeutics, San Diego, California, United States
  • Padgett, Claire S., Sanifit Therapeutics, San Diego, California, United States
  • Gold, Alex, Sanifit Therapeutics, San Diego, California, United States
  • Perelló, Joan, Sanifit Therapeutics, Palma, Spain
  • Chertow, Glenn Matthew, Stanford University School of Medicine, Palo Alto, California, United States
Description

Calciphylaxis (calcific uremic arteriolopathy, CUA) is a severe form of vascular calcification characterized by painful necrotic skin ulcers and very high morbidity and mortality. No approved therapies are available. SNF472, an IV formulation of myo-inositol hexaphosphate, inhibits formation and growth of hydroxyapatite crystals, the final common step in the pathophysiology of vascular calcification. The objective of this ongoing Phase 3 randomized, double-blind, placebo-controlled study is to evaluate efficacy and safety of SNF472 for CUA. Efficacy outcomes include wound healing, pain, and wound-related quality of life (QoL). Standard safety assessments will be conducted.

Planned enrollment is approximately 66 adult patients on maintenance hemodialysis (HD) with a clinical diagnosis of CUA with wound ulceration and wound-related pain. Patients will be randomized 1:1 to SNF472 (7 mg/kg) or placebo 3x/week for 12 weeks during HD sessions, infused via the HD circuit. Following the blinded, 12-week randomized control period, all patients will receive SNF472 in a 12-week open-label period. Patients will also receive background care for CUA throughout the study, in accordance with the practices of each site.

Wound healing will be assessed with the Bates-Jensen Wound Assessment Tool (BWAT), BWAT-CUA, and qualitative review of wound images. All 13 items comprising the BWAT will be assessed: size, depth, edges, undermining, necrotic tissue type, necrotic tissue amount, exudate type, exudate amount, skin color surrounding the wound, peripheral tissue edema, peripheral tissue induration, granulation tissue, epithelialization. BWAT-CUA is an 8-item targeted modification of BWAT focused on prototypical features of CUA lesions including necrotic tissue type and amount, exudate type and amount, skin color surrounding the wound, peripheral tissue edema and induration, and granulation tissue. External, independent wound care experts blinded to treatment assignment will determine BWAT scores based on review of standardized images, measurements from imaging software, and assessments by site investigators. Pain will be assessed on a visual analog scale. QoL will be assessed with the Wound-QoL questionnaire.

Funding

  • Sanifit