Better Evidence and Translation for Calciphylaxis (BEAT-Calci)

University of Sydney

Status and phase

Enrolling

Phase 3

Conditions

Calciphylaxis

Treatments

Device: Medium Cut-off Dialyser

Drug: Magnesium citrate

Drug: Placebo capsule (Vitamin K1)

Drug: Vitamin K1

Drug: Placebo injection (normal saline)

Device: High Flux Dialyser

Drug: Sodium Thiosulfate

Drug: Placebo tablet (Magnesium citrate)

Study type

Interventional

Funder types

Other

NETWORK

Industry

Identifiers

NCT05018221

BEAT-Calci

Details and patient eligibility

About

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

Full 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.

Enrollment

350 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

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
Have a new calciphylaxis ulcer present for less than 10 weeks
Age ≥ 18 years
Eligible for randomisation in at least one recruiting domain
The participant and treating physician are willing and able to perform trial procedures

Exclusion criteria

Nil

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Sequential Assignment

Masking

Quadruple Blind

350 participants in 6 patient groups, including a placebo group

Placebo (Double-Blind Period)

Placebo Comparator group

Description:

Placebo Vitamin K1 Placebo Magnesium Citrate Placebo Sodium Thiosulphate

Treatment:

Drug: Placebo tablet (Magnesium citrate)

Drug: Placebo injection (normal saline)

Drug: Placebo capsule (Vitamin K1)

Vitamin K1 (Double-Blind Period)

Experimental group

Description:

Dose: 10mg Vitamin K1 capsules, administered 3 times per week following the subject's hemodialysis session. * Placebo Magnesium Citrate * Placebo Sodium Thiosulphate

Treatment:

Drug: Placebo tablet (Magnesium citrate)

Drug: Placebo injection (normal saline)

Drug: Vitamin K1

Magnesium Citrate (Double-Blind Period)

Experimental group

Description:

Dose: 150mg Magnesium Citrate tablets, administered 3 times per day. On dialysis days, administration of the middle daily dose should occur following the subject's hemodialysis session. * Placebo Vitamin K1 * Placebo Sodium Thiosulphate

Treatment:

Drug: Placebo injection (normal saline)

Drug: Placebo capsule (Vitamin K1)

Drug: Magnesium citrate

Sodium Thiosulfate (Double-Blind Period)

Experimental group

Description:

Dose: 25g Sodium Thiosulfate injection, administered intravenously 3 times per week, during the subject's last hour of hemodialysis. * Placebo Vitamin K1 * Placebo Magnesium Citrate

Treatment:

Drug: Placebo tablet (Magnesium citrate)

Drug: Sodium Thiosulfate

Drug: Placebo capsule (Vitamin K1)

High Flux Hemodialysis

Active Comparator group

Description:

Hemodialysis using a high flux dialyser

Treatment:

Device: High Flux Dialyser

Medium Cut-off Hemodialysis

Experimental group