Clinical trial NCT05018221: Better Evidence and Translation for Calciphylaxis
Official title: Better Evidence and Translation for Calciphylaxis
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Calciphylaxis
Lead sponsor: University of Sydney
Sponsor class: OTHER
Collaborators: name: Australasian Kidney Trials Network
name: Northern Care Alliance NHS Foundation Trust
name: Waitemata District Health Board
First posted: 2021-08-24
Enrollment: 350
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: Adaptive, platform, randomized controlled trial, involving multiple interventions spanning several domains of therapeutic care.
Primary purpose: TREATMENT

Brief summary: This global platform study will evaluate multiple interventions, across several domains of therapeutic care, in adult patients with kidney failure and newly diagnosed calciphylaxis.

Detailed description: 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.

Eligibility criteria: Inclusion Criteria:

1. Currently receiving haemodialysis, or peritoneal dialysis that can be converted to haemodialysis, with planned ongoing haemodialysis a minimum of three times per week for at least the duration of the protocolised calciphylaxis treatments within this trial
2. Have a new calciphylaxis ulcer present for less than 10 weeks
3. Age ≥ 18 years
4. Eligible for randomisation in at least one recruiting domain
5. The participant and treating physician are willing and able to perform trial procedures

Exclusion Criteria:

Nil

Primary outcomes:
measure: BEAT-Calci Wound Assessment Scale (BCWAS) - Baseline to Week 12
timeFrame: Week 12
Secondary outcomes:
measure: BEAT-Calci Wound Assessment Scale - Baseline to Week 26
timeFrame: Week 26
measure: Distribution of each of the individual components of the BCWAS, assessed at Weeks 4
timeFrame: Week 4
measure: Distribution of each of the individual components of the BCWAS, assessed at Week 12
timeFrame: Week 12
measure: Distribution of each of the individual components of the BCWAS, assessed at Week 26
timeFrame: Week 26
measure: Bates-Jensen Wound Assessment Tool - from Baseline to Week 4
timeFrame: Week 4
measure: Bates-Jensen Wound Assessment Tool - from Baseline to Week 12
timeFrame: Week 12
measure: Bates-Jensen Wound Assessment Tool - from Baseline to Week 26
timeFrame: Week 26
measure: Sentinel ulcer surface area - from Baseline, assessed at Week 4
timeFrame: Week 4
measure: Sentinel ulcer surface area - from Baseline, assessed at Week 12
timeFrame: Week 12
measure: Sentinel ulcer surface area - from Baseline, assessed at Week 26
timeFrame: Week 26
measure: All ulcers total surface area - from Baseline, assessed at Week 4
timeFrame: Week 4
measure: All ulcers total surface area - from Baseline, assessed at Week 12
timeFrame: Week 12
measure: All ulcers total surface area - from Baseline, assessed at Week 26
timeFrame: Week 26
measure: Change over time of self-reported pain
timeFrame: Week 26
measure: Self-reported pain at week 12
timeFrame: Week 12
measure: Change over time of analgesic use
timeFrame: Week 26
measure: Analgesic use week 12
timeFrame: Week 12
measure: Composite self-reported pain and analgesic use over time
timeFrame: Week 26
measure: Composite self-reported pain and analgesic use at week 12
timeFrame: Week 12
measure: Change in self-reported quality of life from Baseline to Week 4
timeFrame: Week 4
measure: Change in self-reported quality of life from Baseline to Week 12
timeFrame: Week 12
measure: Change in self-reported quality of life from Baseline to Week 26
timeFrame: Week 26
measure: Time to first calciphylaxis-attributable infection from Baseline to Week 26
timeFrame: Week 26
measure: All-cause hospitalisation days
timeFrame: Weeks 0-26
measure: Mortality
timeFrame: Up to 5 years
measure: Kidney Transplantation
timeFrame: Up to 5 years
measure: Calciphylaxis recurrence
timeFrame: Up to 5 years
Other outcomes:
Locations:
city: Concord
country: Australia
facility: Concord Repatriation General Hospital
state: New South Wales
city: Kogarah
country: Australia
facility: St George Hospital
state: New South Wales
city: Birtinya
country: Australia
facility: Sunshine Coast Hospital and Health Service
state: Queensland
city: Brisbane
country: Australia
facility: Princess Alexandra Hospital
state: Queensland
city: Bundaberg
country: Australia
facility: Bundaberg Base Hospital
state: Queensland
city: Melbourne
country: Australia
facility: Royal Melbourne Hospital
state: Victoria
city: St Albans
country: Australia
facility: Sunshine Hospital (Western Health)
state: Victoria
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
city: Clayton
country: Australia
facility: Monash Medical Centre
city: Dunedin
country: New Zealand
facility: Dunedin Hospital
city: Grafton
country: New Zealand
facility: Auckland City Hospital (Auckland DHB)
city: Takapuna
country: New Zealand
facility: North Shore Hospital (Waitemata DHB)
city: Tauranga
country: New Zealand
facility: Tauranga Hospital
city: Whangarei
country: New Zealand
facility: Whangarei Hospital
Study officials:
affiliation: University of Sydney
name: Meg Jardine
role: STUDY_CHAIR
References: