JMIR Publications

ABSTRACT

Background:

Background:

Cladribine tablets (marketed as Mavenclad®) is a new oral therapy, which has recently been listed on the pharmaceuticals benefit scheme (PBS) in Australia for treatment of relapsing MS. The current dosing schedule is for 2 courses given a year apart, which has been shown to be effective for treatment of MS up to 4 years in 75% of patients (based on annualised relapse rate). However, re-initiation of therapy after year 4 has not been studied.

Objective:

Objective:

The Objective of this study is to

Methods:

Methods:

This will be a multicentre, 6-year, phase IV, low interventional observational study that incorporates clinical, haematological, biochemical, epigenetic, and radiological and cognitive biomarkers of disease. Subjects considered for treatment with cladribine as part of their routine clinical care, will be consented to take part in the study. They will be monitored at regular intervals during the initial course of medication administration in year 1 and year 2. After year 3, patients will have the option for re-dosing, if clinically indicated or to switch to another disease modifying therapy. Throughout the duration of the study, we will assess blood based biomarkers including lymphocyte subsets, serum neurofilament light chain, DNA methylation and RNA analysis as well as MRI findings (brain volume/ lesion load) and cognitive performance.

Results:

Results:

This study has been approved by the Hunter New England Local Health District Human Research Ethics Committee. Recruitment Started in Mach 2019.

Conclusions:

Conclusions:

This will be the first long-term efficacy trial of cladribine which offers re-initiation of therapy in the 3rd year, based on disease activity, after the initial two courses. We expect this study will indicate if any of the assessed biomarkers can be used to predict treatment efficacy or the need for future re-initiation of cladribine in MS patients. Clinical Trial: ANZCTR 376926