Better Evidence and Translation for Calciphylaxis

BEAT-Calci is a randomized, adaptive, multi-center, platform trial that will evaluate multiple interventions, across several domains of therapeutic care. The objective of the study is to establish high-quality evidence on the effect of a range of interventions in patients with kidney failure and newly diagnosed calciphylaxis. Calciphylaxis is a rare disease affecting 1-2 people in 10,000.

The trial will commence with a Dialysis Membrane Domain and Pharmacotherapy Domain. The Pharmacotherapy Domain of BEAT-Calci is a placebo-controlled, double blind, response adaptive, randomised controlled trial that will investigate whether any of the pharmacotherapeutic agents is superior to placebo in improving outcomes. The Dialysis Membrane Domain of BEAT-Calci is an open-label, randomised controlled two-way comparison between two different dialysis technologies.

The BEAT-Calci Wound Assessment Scale (BCWAS) is the primary endpoint for the trial. It is an 8-point ordinal categorical scale of disease outcomes and will be used to determine each participant's outcome.

The trial will utilise a Bayesian adaptive sample size re-estimation approach for sample size calculations. The trial will continue to recruit until predefined superiority or futility rules are met. As the trial progresses, in response to information accumulating during the trial, there are various adaptations that can occur, including addition or removal of an intervention arm, response adaptive randomisation and addition of new therapeutic domains.

• BEAT-Calci Wound Assessment Scale - Baseline to Week 26 [Срок оценки: Week 26]
• Distribution of each of the individual components of the BCWAS, assessed at Weeks 4 [Срок оценки: Week 4]
• Distribution of each of the individual components of the BCWAS, assessed at Week 12 [Срок оценки: Week 12]
• Distribution of each of the individual components of the BCWAS, assessed at Week 26 [Срок оценки: Week 26]
• Bates-Jensen Wound Assessment Tool - from Baseline to Week 4 [Срок оценки: Week 4]
• Bates-Jensen Wound Assessment Tool - from Baseline to Week 12 [Срок оценки: Week 12]
• Bates-Jensen Wound Assessment Tool - from Baseline to Week 26 [Срок оценки: Week 26]
• Sentinel ulcer surface area - from Baseline, assessed at Week 4 [Срок оценки: Week 4]
• Sentinel ulcer surface area - from Baseline, assessed at Week 12 [Срок оценки: Week 12]
• Sentinel ulcer surface area - from Baseline, assessed at Week 26 [Срок оценки: Week 26]
• All ulcers total surface area - from Baseline, assessed at Week 4 [Срок оценки: Week 4]
• All ulcers total surface area - from Baseline, assessed at Week 12 [Срок оценки: Week 12]