Better Evidence And Translation in Calciphylaxis

Principal Investigators:  Meg Jardine
Clinical Project Manager: Donna Reidlinger (AKTN)
Clinical Research Associate: 
Trial Number: AKTN 17.01

Population:  Prevalent HD patients with presentations of calciphylaxis
Intervention: Thiosulphate, vit K2, magnesium, matching placebos
Follow-up:  6 years
Primary outcome: Composite of wound healing, avoidance of amputation/ development of new lesions/ death

Status: Recruitment to commence 2020
Target Recruitment: 264 participants across Australia and New Zealand, and the United Kingdom

Trial Summary

Calciphylaxis is a rare disease affecting 1-2 people in 10,000. The prognosis is bleak for those diagnosed with the painful condition, who have a mean 3 months life expectancy after diagnosis. The pathophysiology is complex and poorly understood, with no accepted disease-altering treatments. We propose the establishment of a novel and innovative SMART trial incorporating adaptive design features to fill this clear unmet therapeutic need.

The trial procedures closely mimic the approach of current practice and are designed to minimise barriers to participation. Important aspects of the study are:

  • Maximised exposure of participants to active treatments through design of interventions and adaptive randomisation
  • Sequential randomisation to additional active treatments in patients who are not responding
  • Facilitated access to treatments for participants
  • Formalised structures facilitate input on study design and conduct by consumers, nurses and clinicians and facilitate trial advocacy

The fundamental difference between the trial and current practice is the simple replacement of chaotic treatment selection with randomised selection.